Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults
Phase: NA
Conditions: Dystonia
Sponsor: University of California, San Francisco

Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults 
NCT03078816 
March 3, 2017 

Study Application (Version 1.11)
1.0
General Information
*Enter the full title of your study:
Thalamic DBS for Secondary Dystonia
*Enter the study number or study alias
DBSVop
* This field allows you to enter an abbreviated version of the Study Title to quickly identify this
study.
2.0
Add Department(s)
2.1 List the departments associated with this study. The Principal Investigator's department 
should be Primary.:
Primary 
Dept?
Department Name
UCSF - 
 - Neurosurgery
200044
UCSF - 
 - M_Neurology
140020
3.0
List the key study personnel: (Note: external and affiliated collaborators who 
are not in the UCSF directory can be identified later in the Qualifications of 
Key Study Personnel section at the end of the form)
3.1 *Please add a Principal Investigator for the study:
San Luciano Palenzuela, Marta M.D., MD
Select if applicable
Department Chair
Resident
Fellow
If the Principal Investigator is a Fellow, the name of the Faculty Advisor must be supplied below.
3.2 If applicable, please select the Research Staff personnel:
A) Additional Investigators
Bledsoe, Ian MD 
Other Investigator
Demopoulos, Carly PhD 
Other Investigator
Larson, Paul S, MD 
Other Investigator
Ostrem, Jill M.D. 
G

 Other Investigator
Racine, Caroline 
 Other Investigator
Starr, Philip MD, PhD 
 Other Investigator
Volz, Monica M 
 Study Clinician
Wang, Sarah S 
 Other Investigator
Watson, Christa L 
 Other Investigator
B) Research Support Staff
Gittings, Melissa L 
 Study Coordinator
Viser, Aaron 
 Study Coordinator
3.3 *Please add a Study Contact:
 
Wang, Sarah S 
The Study Contact(s) will receive all important system notifications along with the Principal 
Investigator. (e.g. The project contact(s) are typically either the Study Coordinator or the Principal 
Investigator themselves).
3.4 If applicable, please add a Faculty Advisor/Mentor:
 
3.5 If applicable, please select the Designated Department Approval(s):
 
Add the name of the individual authorized to approve and sign off on this protocol from your 
Department (e.g. the Department Chair or Dean).
4.0
 
Initial Screening Questions
Updated June 2017
4.1  
: 
* PROJECT SUMMARY
 
(REQUIRED) Give a brief overview of this project (250 words or less). Tell us 
what this study is about, who is being studied, and what it aims to achieve. If you have an NIH 
  
Abstract, paste it here:  Click on the orange question mark to the right for more detailed instructions.
Dystonia is a movement disorder seen in both children and adults that is characterized by “sustained or 
intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or 
both.”  Secondary dystonia has evolved to refer to dystonia resulting from damage to the nervous system 
or degenerative disease processes. While primary dystonia is generally thought to arise from genetic 
causes, secondary dystonias have a variety of causes including perinatal injuries (cerebral palsy), central 
nervous system infections, traumatic brain injuries, and many different metabolic, neurodegenerative, and 
mitochondrial conditions. Secondary dystonia is far more common in pediatric populations than primary 
dystonia, and far more recalcitrant to standard pharmacologic and surgical treatments including Deep 
Brain Stimulation. Given that most treatments for dystonia are developed for primary dystonia and then 
applied to secondary dystonia, it is not surprising that this effectiveness gap exists. Thus, there exists a 

large unmet need to develop new therapeutics, treatment strategies, and outcome measures for pediatric 
secondary dystonia .
2
Deep Brain Stimulation (DBS) is one such therapeutic intervention that has potential to improve 
secondary dystonia. DBS is a surgical treatment for several different movement disorders that evolved 
from functional stereotactic neurosurgery techniques initially used to lesion specific deep brain structures . 
3
While Essential Tremor and Idiopathic Parkinson’s Disease have predictable and consistent response rates 
to DBS in carefully selected patients, response rates of dystonia have been much more inconsistent. One 
predictor of success has been the presence of DYT-1 mutation, the most common known genetic cause of 
primary dystonia 
. Success rates in DYT-1 dystonia are consistently high with reductions in dystonia 
12
typically greater than 80%
 .  However, the results in secondary dystonia have been much more 
13–15
modest and inconsistent. A recent meta-analysis found that on average, dystonia symptoms as measured 
by common rating scales improve 23% following DBS for dystonic cerebral palsy (the most common cause 
of secondary dystonia), however there are frequent cases of non-responders
. Additionally, there have 
16
been very few examination, radiological or laboratory predictors of good response to DBS, except for 
genetic confirmation of DYT-1
. However, across both primary and secondary dystonia, younger age at 
17
the time of surgery (less than 21 years old) and shorter duration of symptoms (less than 15 years) have 
been shown to be the most likely predictive factors for a good postoperative outcome
.  This has led 
17
many to suggest that DBS should be offered earlier in the course of intractable dystonia, prior to the 
development of permanent complications such as orthopedic contractures.  Thus, we are setting an upper 
age limit of 25 to account for the concern that earlier implantation leads to improved outcomes. The lower 
age limit of 7 reflects the fact that the current humanitarian exemption for DBS for dystonia currently goes 
down to age 7. Thus, there exists a need to both improve patient selection as well as application of DBS 
for secondary dystonia in children. This study has been approved by the FDA under an investigational 
device exemption (IDE #G160233).
4.2  
: 
* HUD DEVICE (REQUIRED) Does this application involve a 
 (HUD):
Humanitarian Use Device
No 
Yes, and it includes a research component 
Yes, and it involves clinical care ONLY 
4.3  
: (Click the Help link for definitions and guidance): 
* TYPE OF RESEARCH
 
(REQUIRED)
Biomedical research 
Social, behavioral, educational, and/or public policy research 
Hybrid - includes aspects of BOTH types of research (check this option if your research is mainly social
/behavioral but also involves specimen collection or blood draws to look at biological measures) 
4.4  
 
* SUBJECT CONTACT:
 
(REQUIRED) Does this study involve 
 contact or interactions with 
ANY
participants:
Yes (including phone, email or web contact) 
No (limited to medical records review, biological specimen analysis, and/or data analysis) 
4.5  
Does your protocol involve any radiation exposure to patients/subjects 
* RADIATION EXPOSURE: 
 from 
 
for 
 purposes (e.g., x-rays, CT-scans, DEXA, CT-guided biopsy, 
EITHER
standard care
 
OR
research
radiation therapy, or nuclear medicine including PET, MUGA or bone scans): (REQUIRED)
  Yes   
  No
4.6  
 
* RISK LEVEL: 
 
(REQUIRED) What is your estimation of the risk level, including all screening 
procedures and study activities 
:
(Help Text updated 9/13)
Minimal risk 
Greater than minimal risk 
4.7  * REVIEW LEVEL: 
 
(REQUIRED) Requested review level (Click on the orange question mark to the 
right for definitions and guidance):

Full Committee 
Expedited 
Exempt 
4.11  
 
* CLINICAL TRIAL:
 
(REQUIRED)     Is this a clinical trial? According to The World Health Organization 
    
(WHO) and the International Committee of Medical Journal Editors (ICMJE) a 
is:
 
clinical trial
 
Any research study that prospectively assigns human participants or groups of humans to one 
or more health-related interventions to evaluate the effects on health outcomes.
      ICMJE requires 
 of a clinical trial in a public database (such as ClinicalTrials.gov) 
registration
    
prior to enrollment, for eventual publication of results in member biomedical journals.
Guidance: 
Public Law 110-85 requires that all investigators who perform an 
 must 
applicable clinical trial
        
ensure that the trial is registered on a government web site called 
.
ClinicalTrials.gov
 
The FDA 
   
requires registration for “applicable clinical trials,” defined as follows:
For any trials of drugs and biologics: controlled clinical investigations, other than Phase 1 
investigations, of a product subject to FDA regulation.
For trials of biomedical devices: controlled trials with health outcomes of devices subject to 
FDA regulation, other than small feasibility studies, and pediatric post-market surveillance.
    For additional information on the 
 registration process at UCSF and the definition 
ClinicalTrials.gov
of a clinical trial for purposes of registration, visit the ClinicalTrials.gov section of the UCSF Clinical 
 
  
  
Research Resource HUB.
 Yes   
 No
Clinical Trial Registration
  "NCT" number for this trial:
to be determined
If you don't yet have the NCT#, type 'Pending.' 
4.12  
 
* CLINICAL TRIAL PHASE (REQUIRED) Check the applicable phase(s) 
:
(Help Text updated 9/13)
Phase I
Phase II
Phase III
Phase IV
4.13  
: 
* INVESTIGATOR-INITIATED
 
(REQUIRED) Is this an investigator-initiated study:
 Yes   
 No
4.14  SCIENTIFIC REVIEW: If this study has undergone scientific or scholarly review, please indicate 
which entity performed the review (check all that apply):
Cancer Center Protocol Review Committee (PRC) (Full approval is required prior to final CHR approval 
for cancer-related protocols.)
CTSI Clinical Research Services (CRS) Advisory Committee
CTSI Consultation Services
Departmental scientific review
Other:
4.15  
 
* STEM CELLS: (REQUIRED) Does this study involve 
(including iPS cells and adult 
human stem cells 
stem cells), gametes or embryos:

No 
Yes, and requires CHR and GESCR review 
Yes, and requires GESCR review, but NOT CHR review 
4.16    
    
 
* FINANCIAL INTERESTS: (REQUIRED)
Do you or any other responsible personnel (or the spouse, 
registered domestic partner and/or dependent children thereof) have 
 related to 
financial interests
  
this study:  
  Yes   
  No
5.0
 Funding
5.1  
FEDERAL FUNDING: 
* 
(REQUIRED) Is this study currently supported in whole or in part by Federal 
funding, 
, OR has it received 
 Federal funding in the past:
even by a subcontract
ANY
  Yes   
  No
5.2  
Is this project linked in any way to the Department of Defense (DoD): 
* DoD INVOLVEMENT: 
(REQUIRED)
  Yes   
  No
5.3  
 
SPONSORS: Identify all sponsors and provide the funding details. If funding comes from a 
 
Subcontract, please list only the Prime Sponsor:
External Sponsors:
 
 
View 
Details
Sponsor Name
Sponsor Type
Awardee 
Institution
Contract 
Type:
UCSF RAS 
"P 
number" 
or 
eProposal 
number 
UCSF 
RAS 
System 
Award 
Number 
("A" + 
6 
digits) 
No Sponsor has been added to this IRB Study
If the funding is coming through UCSF and you don't know the A 
or P number,  you can search the eProposal side for the contract 
or grant (this does NOT replace adding the sponsor by name 
above 
 entering the A or P number):
AND
Project Status
Proposal Number
Project Title
Principal 
Investigator
No Projects are Linked to this IRB Study
Other Funding Sources and Unfunded Research - Gift, 
Program, or Internal Funding (check all that apply):
Funded by gift (specify source below)

Funded by UCSF or UC-wide program (specify source below)
Specific departmental funding (specify source below)
Unfunded (miscellaneous departmental funding)
Unfunded student project
6.0
 Sites, Programs, Resources, and External IRB Review
6.1  
 
 (check all that apply):
UCSF AND AFFILIATED SITES
UCSF (including Laurel Heights and all the other sites outside the main hospitals)
Parnassus
Mission Bay
China Basin
Mount Zion
Helen Diller Family Comprehensive Cancer Center
Langley Porter Psychiatric Institute
San Francisco General Hospital (SFGH)
SF VA Medical Center (SF VAMC)
Blood Centers of the Pacific (BCP)
Blood Systems Research Institute (BSRI)
Fresno Community Medical Center
Gallo
Gladstone
Jewish Home
Institute on Aging (IOA)
SF Dept of Public Health (DPH)
6.2  
At what locations will study visits and activities occur:
LOCATIONS: 
Outpatient visits will take place in the Neurology Clinic at 1635 Divisadero St., Suite 520 or if necessary, at 
the Pediatric Brain Center at Mission Bay. Surgery will take place at the UCSF Benioff Children's Hospital in 
San Francisco. On some occasions when patients may not be able to afford to travel to UCSF for some 
study visits after the immediate post-operative phase, visits may be conducted by video conferencing 
(such as Zoom).
6.3  
Will any study procedures or tests be conducted off-site by non-UCSF 
OFF-SITE PROCEDURES: 
personnel:
  Yes   
  No
6.4  RESEARCH PROGRAMS: Check any UCSF research programs this study is associated with:
Cancer Center
Center for AIDS Prevention Sciences (CAPS)
Global Health Sciences
Immune Tolerance Network (ITN)
Neurosciences Clinical Research Unit (NCRU)
Osher Center
Positive Health Program
6.5  * CTSI CRS SERVICES: (REQUIRED) Will this study be carried out at one of the UCSF Clinical Research 
 units or utilize 
:
Services (CRS)
CRS services
  Yes   
  No

6.6  * MULTI-CENTER TRIAL:
 
 (REQUIRED) Is this a multicenter research trial? By multi-center trial, we 
mean a study where the protocol is developed by an industry sponsor, consortium, a disease-group, 
etc., who then selects sites across the nation or in different countries to participate in the trial. The 
local sites do not have any control over the design of the protocol. 
  Yes   
  No
6.7  
Check all the other types of sites not affiliated with UCSF with which you are 
OTHER SITE TYPES: 
cooperating or collaborating on this project:   Do NOT check any boxes below if this is a 
multi-center clinical trial, UCSF is just one of the sites, and neither UCSF nor its 
affiliates are the coordinating center.
Other UC Campus
Other institution
Other community-based site
Foreign Country
Sovereign Native American nation (e.g. Navajo Nation, Oglala Sioux Tribe, Havasupai, etc.)
6.10  
Does this application include a request to rely on an a central IRB 
* 
: 
RELYING ON AN EXTERNAL IRB
(other than the NCI CIRB) or an external IRB (UC, commercial, or institutional): 
 
(REQUIRED)  
  Yes   
  No
7.0
 Research Plan and Procedures
7.1  This new consolidated section requests information about:
Hypothesis
Aims
Study Design
Background and Significance
Preliminary Studies
Procedures
Statistical Methods
References
Later sections include:
Drugs and Devices
Sample Size, Eligibility, and Subjects
Recruitment and Consent
Risks and Benefits
Data and Safety Monitoring Plan
Confidentiality, Privacy and Security
Financial Considerations
Qualifications of Personnel
Other Approval and Registrations
7.2  
Describe the hypothesis or what the study hopes to prove 
:
HYPOTHESIS: 
(Help Text updated 9/13)

Based on historical results of ventralis oral posterior (Vop) thalamotomy in secondary dystonia, Vop DBS 
will be safe and tolerable and will have improvements equal to or better than globus pallidal DBS in 
secondary dystonia in children and young adults.
7.3  
List the specific aims:
AIMS: 
Determine safety and tolerability of Vop stimulation in children and young adults with secondary 
dystonia
Assess preliminary efficacy of Vop stimulation for reducing dystonia in children and young adults 
with secondary dystonia
Assess preliminary effects of Vop stimulation on quality of life in children and young adults with 
secondary dystonia
7.4  
Briefly describe the study design (e.g., observational, interventional, randomized, placebo-
DESIGN: 
controlled, blinded, cross-over, cross-sectional, longitudinal, pharmacokinetic, etc.):  
This is a single-center, open-label, phase I clinical trial to estimate the treatment effect size and to assess 
preliminary safety and tolerability of Deep Brain Stimulation (DBS) of the ventralis oralis posterior nucleus 
of the thalamus in children and young adults with secondary dystonia. Patients will be recruited from two 
movement disorders centers: UCSF Center for the Surgical Treatment of Movement Disorders at Mount 
Zion and the Pediatric Brain Center at Mission Bay. The study will be discussed with all patients and their 
families/caregivers who meet inclusion and exclusion criteria, and written informed consent will be 
obtained from those patients who wish to enroll in the study.
 
A small cohort of 15 subjects will be implanted in the first two years. All participants will undergo a battery 
of clinical dystonia rating scales, measures of mobility, and quality of life measures performed 
prospectively pre- and post-DBS. Patients will also undergo a neuropsychological battery before surgery 
and 12 months post-operatively. The neuropsychological battery will be customized for each patient and his
/her expressive language abilities. The subjects will be examined every two months for one year following 
surgery and adjustments to the stimulation parameters will be made. The DBS stimulation parameters will 
be patient-specific and calibrated by the dystonia, mobility and quality of life rating scales.  The changes in 
dystonia and quality of life rating scales at the end of 1 year of treatment will be compared to historical 
results from published DBS of globus pallidus interna for secondary dystonia and used to design a future 
larger, randomized clinical trial. All dystonia scales measuring motor involvement will be videotaped so the 
tapes can later be reviewed if necessary.
 
 
7.5  
 Briefly provide the background and significance of this study (e.g. 
BACKGROUND AND SIGNIFICANCE:
 
why is this study needed) (space limit: one half page):
If this is a first in humans study, please summarize the safety data from the 
animal studies. For pediatric drug or device studies, please identify if this is the 
first study in pediatric populations.  
Dystonia is a movement disorder seen in both children and adults that is characterized by “sustained or 
intermittent muscle contractions causing abnormal, often repetitive, movements, postures, or both.”  
1
Secondary dystonia is far more common in pediatric populations than primary dystonia, and far more 
recalcitrant to standard pharmacologic and surgical treatments including Deep Brain Stimulation (DBS). 
There exists a large unmet need to develop new therapeutics, treatment strategies, and outcome 
measures for pediatric secondary dystonia .
2
We are proposing to investigate the ventralis oral posterior nucleus (Vop) of the thalamus as a new target 
for DBS in secondary dystonia. Prior to the development of DBS, the main surgical treatment of dystonia 
was thalamotomy . Although there were many different targets in the thalamus, often done in staged 
3
procedures, the most common and successful targeted nuclei was the Vop, which is traditionally thought to 
be the pallidal receiving area . Previous lesioning of Vop produced improvements in dystonia but 
4
intolerable side effects, especially when implanted bilaterally
. However, given that secondary dystonia 
4–6
patients were often reported to have superior results to primary dystonia
, it is reasonable to believe 
5,7
that if the side effects can be modulated, that targeting of the Vop nucleus with DBS could be a viable 

alternative to GPi. Given that DBS is a treatment that is inherently adjustable, it is conceivable that 
settings on the DBS could be adjusted to allow for clinical benefit with minimal side effects. Indeed, there 
have been several scattered successful case reports attesting to this possibility
.
8,9
7.6  
Briefly summarize any preliminary studies relevant to your proposed 
PRELIMINARY STUDIES: 
research 
:
(space limit: one half page)
none
7.7  
Is this a treatment study, i.e. does this study intend to provide treatment 
* TREATMENT PROTOCOL: 
to individuals with a medical or psychological condition: (REQUIRED) 
 Yes   
 No
7.8  
Types of research activities that will be carried out. Check all that 
 COMMON RESEARCH ACTIVITIES: 
*
apply and describe in more detail in the 'Procedures / Methods' section: (REQUIRED)
Interviews, questionnaires, surveys
Educational or cognitive tests
Focus groups
Observation
Non-invasive imaging or testing (MRI, EEG, pulse oximetry, etc.)
Administration of contrast agent
Imaging procedures or treatment procedures that involve radiation (x-rays, CT scans, CT-guided 
biopsies, DEXA scans, MUGA or PET scan)
Biopsy conducted solely for research purposes
Use of placebo
Sham surgical procedure
Collection of data from wearable tech such as Fitbit, Apple Watch, Garmin, motion actigraphs, etc.)
Fitness tests or other exertion activities
Use of mobile health apps or other apps
Social media-based research activities
None of the above
Please navigate back to section 4.0 Initial Screening Questions to change 
your answer to 4.4 about procedures involving radiation to 'Yes.' This will 
ensure that the Radiation Safety Committee receives a copy of your 
application when you first submit it to the IRB. Contact the Radiation 
Safety Commitee if you are not sure if radiation is involved.
7.9  
PROCEDURES / METHODS: 
* 
(REQUIRED)
For clinical research, list all study procedures, tests and treatments 
required for this study, including when and how often they will be 
performed. If there are no clinical procedures, describe the research 
activities.
 
If some of the activities would occur even if the person were not in the 
study, as in the case of treatment or tests performed for diagnostic 
purposes, clearly differentiate between those activities that will be 
done solely for research purposes and those that are happening as part 
of routine care.
 
Examples may include:

additional scans outside standard clinical diagnosis or monitoring
additional biopsies to collect tissue for research
extra clinic visits
extra lab tests not required for clinical care
If you have a procedure table, attach it to the submission with your other 
study documents.
Screening Visit (within 120 days of surgical date)
Clinical visit to determine initial eligibility for DBS therapy. This will include a clinical movement 
disorders neurology evaluation and/or a clinical surgical evalueation, including review of MRI brain.
 
Informed Consent
Written informed consent (and assent if indicated) will be obtained from all patients before enrollment in 
the study. All potential subjects and/or legal guardians will be properly informed as to the purpose of 
the study and the potential risks and benefits known or that can be reasonably predicted or expected 
and the option of undergoing DBS surgery outside of the study under an HDE. Key parts of the consent 
will be read out loud by a study investigator while the subject and/or guardian reads along and is asked 
if they understand the consent form. The Investigator will retain the original copy of the Informed 
Consent Form signed by the patient, and a duplicate will be provided to the patient.
 
Baseline Assessment
A baseline assessment will be completed prior to the DBS system implantation in all patients. Baseline 
evaluation will consist of reviewing/obtaining the following:
Inclusion/Exclusion criteria
Complete demographic information
Complete medical history and physical examination
Medication history (last 90 days)
Screening MRI of the brain
Clinical rating scales of motor function (videotaped):
Burke-Fahn Marsden Dystonia Rating Scale (BFMDRS)
Barry Albright Dystonia Scale (BADS)
Modified Ashworth Scale (MAS) (when possible)
Gross Motor Function Measure (GMFM) (when possible)
Modified UPDRS II
Caregiver administed rating scales
PEDI-CAT (Pediatric Evaluation of Disability Inventory Computer Admistrated Test)
PedsQL (Pediatric Quality of Life Inventory)
Neuropsychological Battery: tailored to subject's expressive language ability, but likely to include 
tests of:
non-verbal intellectual abilities (Matrix Reasoning, Picture Concepts, and Picture 
Completion [subtests of the Wechsler Intelligence Scale for Children, the Wechsler 
Abbreviated Scale of Intelligence, and the Wechsler Pre-school and Primary Scale of 
Intelligence)
verbal intellectual abilities (Vocabulary, Similarities, Information, and Picture Vocabulary 
[subtests of the Wechsler Intelligence Scale for Children])
memory (Memory for Faces, Dot Locations, and Digit Span [subtests of the 
Children’s Memory Scale])
mood/behavior (BASC-3: Self Report of Personality)
Formal measurements of speech and articulation abilities
Assessments for depression
Blood or urine pregnancy test to be done with standard pre-operative labs, up to 30 days before 
surgery.
This baseline evaluation for a non-study patient treated under the HDE would include all of the above 
except for the MAS, GMFM, Pedi-CAT, PedsQL, and Neuropsychological Testing.
 
DBS System Implantation
 
If blood or urine pregnancy test is not done as part of standard clinical preoperative labs, a test will 
be done on the day of surgery.
 

Implantation of the DBS system will be performed according to standard surgical procedures under 
general anesthesia. Patients will be given an intravenous dose of antibiotics perioperatively and for 
no less than 48 hours after surgery. Patients will be sent home with 10 days of antibiotics.
 
The DBS system implant procedure would be the same as for patients consented under the 
Humanitarian Device Exemption for clinical care of primary dystonia and typical off label use of DBS 
for secondary dystonia 
, except the electrodes will be placed in the thalamus 
(IRB# 10-01285)
instead of the globus pallidus. The infection protocol (IV antibiotics + 10 days of antibiotics post-op) 
is also different from usual clinical care.
 
Post-operative visit 1 and initiation of device use (12 days after surgery +/- 3 days)
Record any Adverse Experiences and/or Review subject diary for adverse experiences and 
dosing compliance.
Record changes to concomitant medications
Perform abbreviated physical examination
Perform and record vital signs
Initial activation of DBS device
Videotaped dystonia rating scales (BADS, BFMDRS) (prior to and following activation of DBS)
Once the patient’s system is programmed, the patient will receive a hand-held device to identify if 
the system is on or off and to slightly modify their settings if their symptoms necessitate. Patients 
will be seen every two months for programming adjustments, but may be seen at any time during 
the study to optimize programming.
 
All of the above is standard of care for post-operative visit 1 except the videotaping of dystonia 
rating scales.
 
Post-operative (months 2-11): Visits every 8 weeks +/- 14 days after initial programming 
appointment for adjustments to DBS settings
Record any Adverse Experiences and/or Review subject diary for adverse experiences and 
dosing compliance.
Record changes to concomitant medications.
Perform abbreviated physical examination.
Perform and record vital signs.
Dystonia rating scales (BFMDRS, BADS)
Optimize stimulator settings for best clinical benefit
At the Month 6 visit, we will administer the PedsQL, UPDRS II, and the Modified Ashworth Scale 
(when possible)
All of the above is consistent with standard of care, except the PedsQL and Modified Ashworth 
Scale at the 6 month visit
 
Post-operative month 12 visit: 52 weeks +/- 14 days from surgical date
Record any Adverse Experiences and/or Review subject diary for adverse experiences and 
exclusionary medication use.
Record changes to concomitant medications.
Perform complete physical examination.
Perform and record vital signs.
Clinical rating scales of motor function (videotaped):
Burke-Fahn Marsden Dystonia Rating Scale (BFMDRS)
Barry Albright Dystonia Scale (BADS)
Modified Ashworth Scale (MAS) (when possible)
Gross Motor Function Measure (GMFM) (when possible)
Modified UPDRS II
Caregiver administed rating scales 
PEDI-CAT (Pediatric Evaluation of Disability Inventory Computer Admistrated Test)
PedsQL (Pediatric Quality of Life Inventory)
Neuropsychological Battery: tailored to subject's expressive language ability, but likely to 
include tests of:
non-verbal intellectual abilities (Matrix Reasoning, Picture Concepts, and Picture 
Completion [subtests of the Wechsler Intelligence Scale for Children, the Wechsler 
Abbreviated Scale of Intelligence, and the Wechsler Pre-school and Primary Scale of 
Intelligence)
verbal intellectual abilities (Vocabulary, Similarities, Information, and Picture Vocabulary 
[subtests of the Wechsler Intelligence Scale for Children])
memory (Memory for Faces, Dot Locations, and Digit Span [subtests of the 
Children’s Memory Scale])

mood/behavior (BASC-3: Self Report of Personality)
This baseline evaluation for a non-study patient treated under the HDE would include all of the above 
except for the adverse event diary, MAS, GMFM, Pedi-CAT, PedsQL, and Neuropsychological Testing.
 
All of the above is consistent with standard of care except for adverse event diary, Modified Ashworth 
Scale, and PedsQL.
 
No additional post-op visits are included in this study compared to what is recommended for routine 
clinical care under the HDE. Follow-up evaluations with formal neurological examination and 
videotaping are important for the programming of the DBS devices. If the patient agrees to participate 
in research, the only additional step will be additional mobility and quality of life scales, 
neuropsychological testing and that the results of their formal neurological examinations (clinical rating 
scores and neuropsychological testing), will be pooled with those of other patients in this study, 
statistically analyzed, and the results will be published.
 
7.10  
: To what extent, if any, do the planned research procedures differ 
STANDARD CLINICAL PRACTICE
from the care that people would otherwise receive at this institution or the study site if not being 
done locally:
    
DBS is occasionally implanted in the globus pallidus interna (GPi) in patients with secondary dystonia as 
an off-label use of the device; the frequency of DBS use in secondary dystonia varies between institutions. 
In recent years, at our institution (UCSF), we have not frequently offered DBS for secondary dystonia 
given that stimulation in the globus pallidus has not led to a substantial enough benefit to warrant the 
risks and costs associated with the procedure. Given that historically thalamotomy for secondary dystonia 
has produced better results, we believe that thalamic DBS has a greater potential to produce substantial 
benefits than pallidal DBS.  Thus, for this study, the electrodes will be placed in the thalamus, not in the 
globus pallidus. Otherwise, the only differences from standard of care for DBS for dystonia will be 
neuropsychological testing at pre-operative visit and month 12 visit, as well as obtaining additional rating 
scales (PEDI-CAT, GMFM, PedsQL). The number of clinical visits remains the same.
7.11  
List all questionnaires, surveys, interview, or focus group guides that will be used for 
INSTRUMENTS: 
this study:
Clinical rating scales of motor function:
Validated
Burke-Fahn Marsden Dystonia Rating Scale (BFMDRS)
Barry Albright Dystonia Scale (BADS)
Modified Ashworth Scale (MAS)
Gross Motor Function Measure (GMFM)
Unvalidated
Modified UPDRS II for Secondary Dystonia (experimental)
Caregiver administered rating scales:
Validated
PEDI-CAT (Pediatric Evaluation of Disability Inventory Computer Administrated Test)
PedsQL (Pediatric Quality of Life Inventory)
Neuropsychological testing: Baseline and one year follow up neuropsychological evaluation will be 
performed using standardized tests when possible. The testing will be customized to each child or young 
adult according his or her age and ability to communicate and access the materials physically. Attempts 
will be made to include the following test battery or comparable tests: (1) non-verbal intellectual abilities 
(Matrix Reasoning, Picture Concepts, and Picture Completion [subtests of the Wechsler Intelligence Scale 
for Children, the Wechsler Abbreviated Scale of Intelligence, and the Wechsler Pre-school and Primary 
Scale of Intelligence)(2) verbal intellectual abilities (Vocabulary, Similarities, Information, and Picture 
Vocabulary [subtests of the Wechsler Intelligence Scale for Children]) (3) memory (Memory for Faces, Dot 
Locations, and Digit Span [subtests of the Children’s Memory Scale]) and (4) mood/behavior (BASC-3: Self 
Report of Personality)
Attach any unpublished instruments in the 'Other Study Documents' 
section of the Initial Review Submission Packet form after completing the 
study application. Published instruments should NOT be attached.

7.12  
Are you drawing any blood or collecting other biosamples (e.g. tissue, 
* BIOSPECIMEN COLLECTION: 
buccal swabs, urine, saliva, hair, etc.): (REQUIRED)
  Yes   
  No
7.25  
Briefly summarize the methods and types of analyses that will be 
STATISTICAL METHODS: 
performed:
All of the dystonia clinical rating scale measures will be measured pre-operatively and post-operatively 
after 1 year of continuous stimulation and the measures at the two time points will be compared using the 
Wilcoxon signed-rank test for matched pairs.
7.26  
(a separate bibliography can be attached 
REFERENCES: List only the 5-10 most relevant references 
for reference purposes if this study involves novel approaches, agents, or an emerging technology 
that the IRB may not be familiar with):
1.           Mink JW. Special concerns in defining, studying, and treating dystonia in children. 
. 2013;28:921-925. doi:10.1002/mds.25548.
Mov Disord
2.           Sironi VA. Origin and evolution of deep brain stimulation. 
. 
Front Integr Neurosci
2011;5:42. doi:10.3389/fnint.2011.00042.
3.           Franzini A, Cordella R, Messina G, et al. Targeting the brain: Considerations in 
332 consecutive patients treated by deep brain stimulation (DBS) for severe neurological 
diseases. 
. 2012;33(6):1285-1303. doi:10.1007/s10072-012-0937-9.
Neurol Sci
4.           Panov F, Gologorsky Y, Connors G, Tagliati M, Miravite J, Alterman RL. Deep brain 
stimulation in DYT1 dystonia: A 10-year experience. 
. 2013;73:86-93. doi:
Neurosurgery
10.1227/01.neu.0000429841.84083.c8.
5.           Cif L, Vasques X, Gonzalez V, et al. Long-term follow-up of DYT1 dystonia 
patients treated by deep brain stimulation: An open-label study. 
. 2010;25:289-
Mov Disord
299. doi:10.1002/mds.22802.
6.           Air EL, Ostrem JL, Sanger TD, Starr PA. Deep brain stimulation in children: 
experience and technical pearls. 
. 2011;8:566-574. doi:10.3171/2011.8.
J Neurosurg Pediatr
PEDS11153.
7.           Koy A, Hellmich M, Pauls KAM, et al. Effects of deep brain stimulation in 
dyskinetic cerebral palsy: A meta-analysis. 
. 2013;28:647-654. doi:10.1002
Mov Disord
/mds.25339.
8.           Vidailhet M, Jutras M-F, Grabli D, Roze E. Deep brain stimulation for dystonia. J 
. 2013;84:1029-1042. doi:10.1136/jnnp-2011-301714.
Neurol Neurosurg Psychiatry
9.           Hyam J a., Owen SLF, Kringelbach ML, et al. Contrasting connectivity of the 
ventralis intermedius and ventralis oralis posterior nuclei of the motor thalamus 
demonstrated by probabilistic tractography. 
. 2012;70(1):162-169. doi:
Neurosurgery
10.1227/NEU.0b013e3182262c9a.
10.        Andrew J, Fowler CJ, Harrison MJ. Stereotaxic thalamotomy in 55 cases of 
dystonia. 
. 1983;106 ( Pt 4:981-1000.
Brain
11.        Burchiel KJ. Thalamotomy for movement disorders. 
. 1995;6:
Neurosurg Clin N Am
55-71.
12.        Cardoso F, Jankovic J, Grossman RG, Hamilton WJ, Kelly PJ, Tasker RR. Outcome 
after stereotactic thalamotomy for dystonia and hemiballismus. 
. 1995;36:
Neurosurgery
501-508. doi:10.1227/00006123-199503000-00009.
13.        S. H, N. T, T. T. Safety and efficacy of stereotactic ventrooral-thalamotomy for 
musician’s dystonia. 
. 2014;29:S504.
Mov Disord
14.        Kim JP, Chang WS, Chang JW. Treatment of secondary dystonia with a combined 
stereotactic procedure: Long-term surgical outcomes. 
. 2011;153:
Acta Neurochir (Wien)
2319-2327. doi:10.1007/s00701-011-1147-6.
15.        Vidailhet M, Yelnik J, Lagrange C, et al. Bilateral pallidal deep brain stimulation for 
the treatment of patients with dystonia-choreoathetosis cerebral palsy: a prospective pilot 
study. 
. 2009;8(8):709-717. doi:10.1016/S1474-4422(09)70151-6.
Lancet Neurol
16.        Binder DK, Rau GM, Starr P a. Risk factors for hemorrhage during microelectrode-
guided deep brain stimulator implantation for movement disorders. 
. 2005;56
Neurosurgery
(4):722-728. doi:10.1227/01.NEU.0000156473.57196.7E.
17.        Sillay K, Larson P, Starr P. DEEP BRAIN STIMULATOR HARDWARE-RELATED 
INFECTIONS: INCIDENCE AND MANAGEMENT IN A LARGE SERIES. 
. 2008;62
Neurosurgery
(2):360-367. doi:10.1227/01.NEU.0000297020.60125.FC.
 

8.0
Drugs and Devices
8.1  
Are you 
 any drugs and/or biologics that are either 
* DRUGS AND/OR BIOLOGICS: 
STUDYING
approved or unapproved: (REQUIRED)
  Yes   
  No
 
Note: This question is frequently answered incorrectly. If any drugs or 
biologics, approved or unapproved, are being administered under this 
protocol, you should check 'Yes' unless you are 
 sure that NONE 
absolutely
of the drugs are part of the research protocol. Tip: Ask the PI or the 
sponsor if you are not sure how to answer this question.
8.3  
: Are you 
 any medical devices, in vitro diagnostics, or assays that are 
* MEDICAL DEVICES
STUDYING
 
either approved or unapproved:(REQUIRED)
 Yes   
 No
8.4     
 Are you requesting a Non-Significant Risk (NSR) determination for an investigational device: 
* NSR:
        
(REQUIRED)
 
Note: an 
 is different from an Investigational Device Exemption 
NSR determination
(IDE). Check the Help link for more guidance on what types of devices can qualify for an NSR 
  
determination.  
  Yes   
  No
8.5  
List the medical devices or in vitro diagnostics to be studied or used. In the device 
LIST THE DEVICES: 
details screen you will be asked questions such as:   
Whether the device is FDA approved or investigational
Medicare device category
If the device will be provided at no cost
If an IDE is necessary, the IDE number, and who holds the IDE
Risk category of the device
FDA status of the device
    Please see the 
 for more details about the use of devices in research, including 
UCSF IRB website
the Investigator Checklist for Significant Risk, Non-Significant Risk, and/or IDE Exempt Device Studies
        
 
 If the sponsor’s protocol does not list the IDE number, you must 
Verification of IDE numbers:
submit documentation from the sponsor or FDA identifying the IDE number for this study. Attach this 
        
documentation in the Other Study Documents section of the Initial Review Submission Packet.
 
  
If you have any correspondence from the FDA or sponsor regarding this device, 
     
  
please attach it to the application.
 
View 
Details
Device Name
Is the Device FDA 
Approved
Is this a new 
device or a new 
use of an already 
approved device
IDE Number
Activa PC Primary Cell 
Neurostimulator 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 

HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
Activa SC Single Cell 
Neurostimulator 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
Yes 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
No Significant Risk  
Neurostimulator 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  

DBS Lead 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
Activa SC Single Cell 
Neurostimulator 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
N/A  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
Medtronic DBS extension 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 

Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
DBS Extension 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
Patient Programmer 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
Test Stimulator 
Yes 
Yes 
G160233 

Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
N'Vision Clinician Programmer 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  
Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
N'Vision Software Application 
Card 
Yes 
Yes 
G160233 
Manufacturer/Supplier of Device
Medtronic 
Medicare Category
A
B
Where will the Devices Be Stored
 
Will Devices be supplied at no Cost
No 
Is this a HUD (HDE)
No 
HDE Number
 
Is the Device FDA Approved
Yes 
Is this a new device or a new use 
of an already approved device
Yes  

Is an IDE necessary
Yes  
IDE Number
G160233  
Who holds the IDE
PI holds the IDE  
IDE Details
 
In the opinion of the sponsor, 
select the level of risk associated 
with this device
Significant Risk  
8.6  
Is this an expanded access or compassionate use protocol, meaning the primary purpose is to 
* 
diagnose, monitor or treat a patient's condition, rather than the collection of safety and efficacy data 
of the experimental agent: (REQUIRED) 
  Yes   
  No
9.0
 Sample Size and Eligibility Criteria
9.1  
 
How many people will you enroll:
ENROLLMENT TARGET: 
17
 If there are multiple participant groups, indicate how many people will be in each 
group:
9.3  
Explain how and why the number of people was chosen. For multi-site 
SAMPLE SIZE JUSTIFICATION: 
studies, this is referring to the number that will be enrolled across all sites:
This is a phase I pilot study of 15 participants to determine safety and tolerability as well as 
estimate effect size to inform planning of future larger trials that will test 
effectiveness. There is not sufficient data on the efficacy outcome measures to calculate 
sample size using statistical methods, thus the number of subjects was estimated based on a 
similar prospective study of DBS of globus pallidus (GPi) in adults with secondary dystonia 
in which a significant improvement was found using 13 subjects. We will screen up to 17 
patients to achieve a total of 15 implanted.
9.4  
Eligible age ranges: 
* 
 AGE RANGE: 
PARTICIPANT
(REQUIRED)
0-6 years
7-12 years
13-17 years
18-64 years
65+
9.5  
 Data will be collected from or about the following types of people (check all 
STUDY POPULATIONS:
* 
 
that apply): (REQUIRED)
Inpatients
Outpatients
Family members or caregivers
Providers
People who have a condition but who are not being seen as patients
Healthy volunteers
Students

1.  
2.  
3.  
Staff of UCSF or affiliated institutions
None of the above
9.6  
Check the populations that may be enrolled: 
SPECIAL SUBJECT GROUPS: 
* 
(REQUIRED)
Children / Minors
Subjects unable to consent for themselves
Subjects unable to consent for themselves (emergency setting)
Subjects with diminished capacity to consent
Subjects unable to read, speak or understand English
Pregnant women
Fetuses
Neonates
Prisoners
Economically or educationally disadvantaged persons
None of the above
If not already addressed in the Background and Significance questions in the 
Research Plan section or elsewhere, explain why it is appropriate to include 
the types of subjects checked above in this particular study:
The intervention is aimed at children and young adults because data from Deep Brain Stimulation (DBS) in 
other sites in the brain (globus pallidus) suggests that the earlier in the disease course DBS if performed 
the better the outcome. DBS of globus pallidus has been shown to be safe and effective in children as 
young as 7 years of age.
Describe the additional safeguards that have been included in the study to protect 
the rights and welfare of these subjects and minimize coercion or undue influence:
Here are some examples:
evaluating capacity to consent for individuals who may be decisionally 
impaired (specify how)
calibrating payment amounts to be non-coercive for the financially 
disadvantaged
conducting more in-depth evaluations of subjects’ understanding of the study 
and the voluntary nature of participation
involving advocates in the consent process
More information and other safeguards are described here: Vulnerable Subject 
 and 
.
Populations
Recruiting Staff and Students
Children and adolescents ages 7-17 will be asked to give verbal assent to the procedure. Subjects age 18 
and over who are unable to give informed consent will have a surrogate give informed consent on their 
behalf.
9.7  
 Briefly describe the population(s) that will be involved in this study. Include 
INCLUSION CRITERIA: 
anyone that data will be collected from or about (e.g. patients, healthy controls, caregivers, providers, 
administrators, students, parents, family members, etc.):
Male or female between 7-25 years of age at Visit 2 (day of surgery).
Documentation of a secondary dystonia diagnosis as determined by a 
fellowship trained movement disorders specialist, including either 
abnormality on brain MRI or history of hypoxic-ischemic brain injury 
preceding onset of dystonia symptoms.  
Symptoms present for at least 6 months.

4.  
5.  
6.  
7.  
8.  
9.  
10.  
11.  
Written informed consent and assent (when applicable) obtained from 
participant or participant’s legal representative and ability for participant 
to comply with the requirements of the study.
Dystonia symptoms that are sufficiently severe, in spite of best medical 
therapy, to warrant surgical implantation of deep brain stimulators in the 
clinical opinion of the treating physician
Minimum score of Burke-Fahn-Marsden Rating Scale and/or Barry-Albright 
Dystonia Rating Scale of 6.
Stable doses of anti-dystonia medications (such as levodopa, baclofen, or 
diazepam) for at least 30 days prior to baseline assessment
If participant receives botulinum toxin injections, patient should be on a 
stable injection regimen (as defined as no changes in past 4 months)
Intact thalamic anatomy as determined by standard clinical MRI in the 
past 2 years. 
Patient has been clinically determined to be a good candidate for deep 
brain stimulation.
A caregiver willing to administer rating scales related to the study
9.8  
 List any exclusion criteria (e.g. reasons why someone would not be included in 
EXCLUSION CRITERIA:
the study):
Pregnancy or breast feeding
Major comorbidity increasing the risk of surgery (severe hypertension, severe diabetes, or need for 
chronic anticoagulation other than aspirin)
Inability to comply with study protocols or follow-up visits
Uncontrolled epilepsy (more than 1 seizure in the past 6 months)
Has an active infection
 Requires diathermy, electroconvulsive therapy (ECT) or transcranial magnetic stimulation (TMS) to 
treat a chronic condition
 Has an 
implanted  neurostimulator, cardiac pacemaker, drug pumps, defibrillators, or 
active 
metallic implant in head.
 Dystonia caused by known genetic mutation in any DYT genes
  Subjects with secondary dystonias 
 by tardive dyskinesia associated with psychotropic 
caused
medications or an ongoing inflammatory or infectious process.
 Subjects with cognitive impairment or dementia as defined by an estimated IQ of less than 70 
on  nonverbal IQ measures.
 Subjects with uncontrolled depression as diagnosed by board certified psychiatrist
 Subjects with uncontrolled co-existing medical conditions: e.g., uncontrolled systemic hypertension 
with values above 170/100; active heart disease needing immediate intervention; active respiratory 
disease needing immediate intervention; uncorrected coagulation abnormalities;
 Contraindication to undergo a brain MRI
 Subjects who are immunocompromised, are on anticoagulation therapy that would preclude their 
ability to undergo the implant procedure, and are allergic or have shown hypersensitivity to any 
materials of the neurostimulation system which come into contact in the body
9.9    
Do any study activities take place on patient 
* RESEARCH CONDUCTED ON PATIENT CARE WARDS: 
  
care units at UCSF medical facilities: (REQUIRED)  
 Yes   
 No
Attach a letter of acknowledgement for the study from the involved 
patient care manager. If you don't know who the patient care manager is, 
click 
 to send an email to the nursing group.
here

10.0
 Inclusion of Minors in Research
10.1  
Select all the 
 that apply:
REGULATORY CATEGORIES OF RESEARCH: 
regulatory categories
No greater than minimal risk (45 CFR 46.404, 21 CFR 50.51)
Greater than minimal risk but presenting prospect of direct benefit (45 CFR 46.405, 21 CFR 50.52)
Greater than minimal risk (though only a minor increase over minimal risk) and no prospect of direct 
benefit but likely to yield generalizable knowledge about the subjects disorder or condition (45 CFR 
46.406, 21 CFR 50.53)
Research not otherwise approvable which presents an opportunity to understand, prevent, or alleviate 
a serious problem affecting the health or welfare of children (45 CFR 46.407, 21 CFR 50.54)
Explain why the research in this study falls under the above category or categories:
The study involves brain surgery that carries significant risk, but there is a good chance of substantial 
(>25%) reduction in dystonia symptoms with the intervention in the study.
10.2  
MINORS CONSENTING: Will this study enroll minors who can 
 (as in 
legally consent for themselves
the case of emancipated minors or minors being treated for pregnancy or drug use without their 
parents knowing). This is different from agreeing to be in the study even when their parents are the 
ones providing 'official' consent, which we refer to as 'providing assent':
    
 
Note: This is very rare and the answer is usually 'No.'
  Yes   
  No
10.3  
PARENTAL PERMISSION VS. WAIVER: Please review the 
 to see under what circumstances 
guidance
the IRB can waive parental permission.
Parental permission will be obtained
Waiver of parental permission is requested: The waiver meets the provisions for a waiver of consent (i.
e., the research poses minimal risk, it could not practicably be carried out without the waiver of 
parental permission, AND the waiver will not adversely affect the rights and welfare of the minor 
participants (45 CFR 46.116(d))
Waiver of parental permission is requested: Parental permission is not a reasonable requirement to 
protect the minor (e.g. neglected or abused children) or parental knowledge of the study may 
endanger the health or welfare of the minor (45 CRF 46.408(c))
Provide details on the other protections that will be in place:
10.4  
 Please review the 
 to see under what circumstances the 
ASSENT OF MINORS OR WAIVER:
guidance
IRB can waive assent. 
Assent of children developmentally and psychologically able to provide assent will be obtained
Waiver of assent is requested: The capability of some or all of the children is so limited that they 
cannot reasonably be consulted
Waiver of assent is requested: The research holds out a prospect of direct benefit that is important to 
the health or well-being of the children and is available only in the context of the research
Waiver of assent is requested: The activities involving the minor are limited to chart review or the 
something equally innocuous
Waiver of assent is requested: It is not culturally appropriate to involve the minor in the decision to 
participate (e.g. some foreign research)
Provide a brief justification for the waiver:

The disease processes that cause secondary dystonia can also impair children's ability to speak and 
provide assent, and thus we are requesting this waiver only for patients who are impaired.
10.5  
(select all that will be used):
DOCUMENTATION OF PERMISSION AND ASSENT: 
Permission form addressed to the parents
Simplified assent form addressed to the child, 7-12 years old (parents get separate form)
Assent form addressed to the child, 13 years and older (for subjects and parents)
Assent form addressed to the child, 13 years and older (parents get separate form)
Check one:
One parent's signature will be obtained 
Two parents' signatures will be obtained 
If this study is approvable under regulatory category .405 and you plan to get permission from only one 
parent, explain why you think one parent's permission is sufficient:
10.6  
Might this study enroll wards of the state:
WARDS OF THE STATE: 
  Yes   
  No
11.0
 Recruitment and Consent
11.1  
 
What kinds of methods will be used to identify potential participants for 
* RECRUITMENT METHODS: 
recruitment (check all that apply): (REQUIRED)
Medical records review
Recruitment registry
Re-contact of participants from the investigators' previous studies
Referrals from colleagues (attach the 'Dear Colleague' letter or other recruitment materials you will 
provide to colleagues)
Referrals from the community / word of mouth
Advertisements (flyers, brochures, radio or t.v. ads, posting on clinical research sites or social media, 
presentation of the study at community events/media, etc.)
Online recruiting tool such as TrialSpark
CTSI Recruitment Services unit
Other method (describe below)
11.2  * SEARCHING OF MEDICAL RECORDS: (REQUIRED)
Whose patients are they:
Investigators' own patients or patients seen within the same practice
Patients not under the care of the investigators
How and by whom will records be accessed and searched (check all that apply):
Self-search in APeX or other medical records source
Self-search using UCSF's Research Cohort Selection Tool
CTSI Consultation Service Recruitment Services
UCSF Academic Research Services (ARS)
University of California Research Exchange (UC ReX)
Other method (describe below)

11.3  
How, when, and by whom will eligibility for recruitment be 
DETERMINATION OF ELIGIBILITY: 
determined:
Eligibility will be determined by the Investigator. After the Investigator determines that the patient and 
caregivers understand the risks and benefits, comprehend the consent, and have received satisfactory 
answers to all questions and signed the consent, the Investigator will determine eligibility based upon a 
complete history and physical, evaluation of screening assessment tools, and determination as to whether 
the patient meets all of the inclusion criteria and none of the exclusion criteria.
11.4  
Who initiates contact (check all that apply): 
INITIATION OF CONTACT: 
* 
(REQUIRED)
Investigators/study team
UCSF recruitment unit (e.g. CTSI Consultation Services)
Potential participant
Other (explain below)
11.5  
 (check all that apply): 
HOW IS CONTACT INITIATED:
* 
(REQUIRED)
In person
Phone
Letter / email
Website or app
Other (explain below)
11.6  
Based on the checkboxes you chose above, please provide a narrative 
RECRUITMENT PLAN: 
describing your recruitment plan. We want to know:
Who is conducting the search for potential participants, and how?
How are potential subjects being approached for recruitment? By whom, and when?
If there will be more than one participant group (e.g. patients, healthy controls, caregivers, family 
members, providers, etc.), provide details about the recruitment plans for each group. 
(Recommended length - 100-250 words)
Patients referred to the UCSF movement disorders surgery clinics for surgical treatment of dystonia will be 
offered participation in this study, by Dr. Viehoever, or Dr. Starr, if they are appropriate candidates for 
surgical treatment of dystonia.
11.7  
How will permission to participate (i.e., informed consent) be obtained from 
CONSENT METHODS: 
* 
each potential participant. If there will be multiple groups and different plans for consenting each, 
  
check all that apply. See the orange Help bubble to the right for more detailed guidance.  
 
Participants will (check all that apply): (REQUIRED)
Sign a consent form at the end of the consent discussion (signed consent)
Provide online 'eConsent' using DocuSign or another E-Signature system
Click through a link in a survey or email after reading about the study and then complete the study 
online (electronic consent)
Be told about the study and be given a handout/information sheet and be asked if they agree to 
participate (verbal consent)
Complete the study activities and turn in materials, as in the case of a completed survey that is placed 
in a drop box or mailed to the study team (implied consent)
Not be able to provide consent and will have a family member consent for them, as in the case of a 
critically ill or unconscious patient (surrogate consent)
Not be able to provide consent (emergency waiver of consent - allowed for minimal risk research or 
greater than minimal risk research with an approved community consultation plan)
Not know about the study, as in the case of chart reviews or observations of public behavior (waiver of 
consent)
Other method (describe below)

Attach your consent form, information sheet, or electronic consent text in 
the Informed Consent Documents section of the Initial Review Submission 
Packet Form.
11.8  
Describe the process for obtaining informed consent, including details such as 
CONSENT PROCESS: 
* 
who will have the consent discussion and when participants will be asked to sign the consent form in 
relation to finding out about the study: (REQUIRED)        
 
We encourage researchers to review our 
   
.
guidance on obtaining and documenting informed consent
If there are multiple groups being consented differently, provide details about the consent 
process for each group.
If you are relying on 
, provide details about how that will happen.
verbal or implied consent
For studies using online recruitment and consent or consent via mail, provide details here.
Consent will be obtained by Dr. Amy Viehoever, Dr. Philip Starr, study nurse or coordinator certified to 
obtain consent in the surgical movement disorders clinic at UCSF. Patients who are eligible for this 
research study may be offered the choice of having DBS surgery under this research protocol or continue 
with standard medical treatment. The consent form will be reviewed and signed following a detailed 
verbal discussion about the risks and benefits of the procedure. The assent form will be reviewed with 
the child and signed following a detailed discussion. The discussion will occur after review of history and 
physical exam shows the patient to be an appropriate candidate for DBS. Patients and caregivers will 
have an open-ended amount of time to review the consent form and sign it.
 
 
 It is important that the people obtaining consent are qualified to do so. Briefly 
*
describe the training and experience these individuals have in obtaining informed 
consent: (REQUIRED)
Either the Principal Investigator, Dr. Amy Viehoever, the Study Nurse, Monica Volz, or the Study 
Coordinator, Kristen Dodenhoff, will obtain the consent forms.
 
Dr. Viehoever is an Assistant Professor in the Divisions of Child Neurology and Surgical Movement 
Disorders at UCSF. She sees patients at the UCSF Pediatric Brain Center at Benioff Children's Hospital and 
Mt. Zion Campus. Dr. Viehoever's research focuses on improving the use of Deep Brain Stimulation (DBS) 
for pediatric primary and secondary dystonia, including Cerebral Palsy. She also uses advanced MRI and 
optical imaging to improve our understanding of how DBS improves brain function in both adult and 
pediatric patients. She also is a member of the Wolfram Study Group, a multidisciplinary collaboration to 
study the natural history of Wolfram Syndrome. She is an expert in optical imaging and using functional 
imaging to study neurologic disorders.
 
Monica Volz, MS, RN, is a clinical nurse specialist with 10 years of experience managing clinical trials and 
movement disorders patients treated with deep brain stimulation.
 
Kristen Dodenhoff is a clinical research coordinator with experience coordinating and obtaining consent for 
multiple studies being conducted within the Movement Disorders and Neuromodulation Center.
 
 
11.9  
Indicate how the study team will assess and enhance the subjects' 
CONSENT COMPREHENSION: 
* 
understanding of study procedures, risks, and benefits prior to signing the consent form (check all 
that apply): (REQUIRED)        
 
Tip: Review the Consent Comprehension - Learning Notes in the Help 
bubble at the right for specific questions that can be asked to assess comprehension, consider using 
the UCSF Decision-Making Capacity Assesment Tool, and review our guidance on obtaining written or 
 
verbal informed consent for more detail on how to conduct the assessment.
The study team will engage the potential participant in a dialogue, using open-ended questions about 
the nature of the study or the experimental treatment, the risks and benefits of participating, and the 
voluntary nature of participation
Potential participants will be asked or shown a series of questions to assess their understanding of the 
study purpose, procedures, risks and benefits, as well as the voluntary nature of participation 
(especially appropriate when the consent process happens online or through a mobile health app)
Other method (describe below):

Provide details of the other approaches that will be used, if using another method 
to assess comprehension:
Investigator(s) will meet with the subject to review the consent form and answer all questions. If the 
Investigator determines that the subject and parent or legal guardian does not fully understand or is 
hesitant, he/she will not be enrolled.
11.11    
: Indicate which 
 you will use to consent non-
NON-ENGLISH CONSENT METHOD
* 
method(s)
  
English speaking subjects: (REQUIRED)  
Preferred Method—Consent form and other study documents will be available in the subject’s primary 
language Personnel able to discuss participation in the patient’s language will be present for the 
consent process.
Short-Form—A qualified interpreter will translate the consent form verbally, and subjects will be given 
the Experimental Subject’s Bill of Rights in their primary language, following instructions in Those Who 
do not Read, Speak or Understand English for required witnessing and signatures
Explain how you will maintain the ability to communicate with non-English 
* 
speakers throughout their participation in the study: (REQUIRED)
If a member of the study staff speaks the primary language, we will communicate with the participant 
directly throughout their participation. If there is no member of the study staff who speaks the 
participant's primary language, a qualified interpreter will be hired for all visits. Qualified interpreters will 
be present for the informed consent process for all patients. 
11.13  
What is the estimated time commitment for participants (per visit and in total):
TIME: 
Participation in the study will take a total of about 48 hours for the surgery and the related ICU stay 
(patient will be discharged from the ICU) , and 24 hours over a period of 12 months for outpatient study 
visits.
IMPORTANT TIP: Ensure this information is consistent with the 
information provided in the consent form.
11.14  
Is there a standard of care (SOC) or usual care that would be offered 
ALTERNATIVES: 
to prospective participants at UCSF (or the study site) if they did not participate in this research 
study:
 Yes   
 No
Describe the care that patients would ordinarily receive at the medical center if 
they did not participate in this study (provide details, assuming that some of the 
IRB members are not specialists in this field):
Other treatments for dystonia include medications and injections into the muscles of botulinum toxin. All 
patients being offered DBS surgery have already tried the appropriate treatments and have received 
inadequate benefit. Thus, the only alternative to DBS surgery is to continue to derive as much benefit as 
possible from medications and botulinum toxin injections.
11.15  
Is the study drug or treatment available off-study:
OFF-STUDY TREATMENT: 
Yes 
No  
Not applicable 
12.0
 Waiver of Consent/Authorization for Recruitment 
Purposes

This section is required when medical records may be reviewed to 
determine eligibility for recruitment.
12.1  
Study personnel need to access 
* PRACTICABILITY OF OBTAINING CONSENT PRIOR TO ACCESS: 
protected health information (PHI) during the recruitment process and it is not practicable to obtain 
informed consent until potential subjects have been identified: (REQUIRED)
Yes 
If 
, a waiver of consent/authorization is NOT needed.
no
12.2  
 
A waiver for screening of health records to identify potential subjects poses no 
* RISK TO PRIVACY: 
more than minimal risk to privacy for participants:
Yes 
If 
, a waiver of authorization can NOT be granted.
no
12.3  
Screening health records prior to obtaining consent will not adversely affect 
* RIGHTS/WELFARE: 
subjects' rights and welfare:
Yes 
If 
, a waiver of authorization can NOT be granted.
no
12.4  
Check all the identifiers that will be collected prior to obtaining informed consent:
* IDENTIFIERS: 
Names
Dates
Postal addresses
Phone numbers
Fax numbers
Email addresses
Social Security Numbers*
Medical record numbers
Health plan numbers
Account numbers
License or certificate numbers
Vehicle ID numbers
Device identifiers or serial numbers
Web URLs
IP address numbers
Biometric identifiers
Facial photos or other identifiable images
Any other unique identifier
None
Note: HIPAA rules require that you collect the minimum necessary.
12.5  
 
Describe any health information that will be collected prior to obtaining 
* HEALTH INFORMATION: 
informed consent:
Patients recruited for this study will be clinical patients referred to the care of Dr. Viehoever for DBS 
evaluation. Health information that is collected prior the obtaining informed consent will be information 

necessary for the standard clinical care of patients including the identifiers indicated above. Any research-
related information will be collected after obtaining informed consent.
Note: HIPAA requires that you collect the minimum necessary.
12.6  
Describe your plan to destroy any identifiable data 
* DATA RETENTION/DESTRUCTION PLAN: 
collected to determine eligibility for recruitment. This should be done at the earliest opportunity. If 
you plan to retain identifiable recruitment data, provide the justification for doing so: 
As  long  as  the  study  is  in  progress  patient  names  and  medical  records  numbers  will  be   retained  in  th
e  research  database.    At  the  time  the  study  is  completed or a patient is deamed ineligible for the 
study,  UCSF  patient   names, medical  records  numbers, and health 
information  will  be  deleted  from  the  research  database.  
13.0
 Surrogate Consent
13.1  
Are any subjects inpatients on a psychiatric ward or mental health facility, or 
PSYCHIATRIC SCREEN: 
on psychiatric hold:
No 
If Yes, use of surrogate consent for research is NOT allowed in California.
13.2  
Is this study related to the cognitive impairment, lack of capacity, or serious 
AREAS OF RESEARCH: 
or life-threatening diseases and conditions of the research subjects: 
Yes 
13.3  
Explain why use of surrogates is necessary for completion of this study:
JUSTIFICATION: 
Secondary dystonia often causes significant problems with expressive language and speech as well as 
writing, and thus some subjects may not be able to give verbal or written consent.
13.4  
 Describe the plans for assessing the decision-making capacity of 
COGNITIVE ASSESSMENT: 
prospective subjects:
Baseline Neuropsychological testing will be performed as will make use of non-verbal testing methods to 
assess intellectual capacity and function of each subject. We will also rely on caregiver reports and the 
individual education plan (IEP) that is used by the schools to provide child specific instruction to fully 
understand each subjects abilities and deficits. 
13.5  
Describe the plans for obtaining consent from subjects who 
POST-ENROLLMENT CONSENT PLANS: 
regain ability to consent after a surrogate has given initial consent:
It is possible that the treatment with DBS will improve motor functioning enough to allow for a subject to 
provide either verbal or written consent during the post-operative follow-up. If this happens, we will 
reconsent the subject under his/her own capacity.
13.6  
SURROGATE CONSENT REQUIREMENTS: Check to acknowledge:
Research takes place in California. All surrogates will complete the “Self-Certification of Surrogate 
Decision Makers for Participation in Research” form.
Conscious subjects will be notified of the decision to contact a surrogate. If subjects object to study 

participation, they will be excluded even if their surrogate has given consent.
Surrogates will not receive any financial compensation for providing consent.
If a higher-ranking surrogate is identified at any time, the investigators will defer to the higher-ranking 
surrogate’s decision regarding the subject’s participation in the research.
For research taking place outside of California, explain how investigators will 
confirm that surrogates are legally authorized representatives:
N/A
14.0
 Risks and Benefits
14.1  
Check if your study involves any of these specific research-related risks 
RESEARCH-RELATED RISKS: 
to participants that may need to be disclosed in the consent form:
For interventional studies, risk that the regimen may be more harmful or less effective than other 
available interventions
Risks associated with radiation exposure for imaging studies specifically for research purposes
Risks associated with the administration of contrast agent for imaging studies
Risks associated with withholding of treatment or discontinuation of current treatment (e.g., washout 
period is required by the study protocol)
For randomized, placebo-controlled trials, possible temporary or permanent health consequences from 
the deprivation of effective therapies during the placebo administration period
For studies involving a sham surgical procedure, the risk that participants may experience increased 
morbidity without the possibility of benefit
Risks associated with modification or extension of a surgical procedure primarily for research purposes 
(e.g. risks associated with prolonging anesthesia, time in the operating room, etc.)
Risk of pain or physical discomfort caused by the research intervention
Possible personal discomfort due to sensitive topics (stress, embarassment, trauma)
For any boxes checked above, describe how you will minimize these risks and 
* 
discomforts, e.g., adding or increasing the frequency of monitoring, additional 
screening to identify and exclude people with diminished kidney or liver function, 
or modification of procedures such as changing imaging studies to avoid giving 
contrast agent to people who are more likely to suffer side effects from it, etc.: 
(REQUIRED)
Weight gain or cognitive impairment. This is a theoretical risk based on side effects reported from 
thalamotomies in prior research reports
Stimulation induced dysarthria or weakness. This is a theoretical risk based on side effects reported from 
thalamatomies in this patient population. If these side effects occur, decreasing or turning off stimulation 
should eliminate them.
Infection: We anticipate that the infection rate in this patient population may be higher than the infection 
rate in Parkinson’s disease, Essential Tremor, and primary dystonia. This is due to known higher 
infection rates in children compared with adults and the presence of a mixed movement disorder with a 
greater amount of hyperkinetic movements as compared with other movement disorders patients.
Patient growth necessitating lead/extension length revision
Patient brain/skull growth leading to lead migration: In cases where growth of the brain and/or skull is not 
complete at time of implant, the distance from the lead anchor point (burr hole) to the target site 
increases with time and growth of the individual. As a result, lead migration relative to the target site may 
occur.
Children should be advised to avoid games, sports and other pastimes where a strain to the lead
/connector assembly or a percussive injury to system components may be likely to occur (e.g., soccer, 
football/rugby).
Intracranial hemorrhage

Hemiplegia/Hemiparesis
Paresis/asthenia
Worsening of Motor Impairment
Trouble swallowing
Sensory Impairment (e.g., pain, paresthesia, sensory disturbance, hypesthesia, hearing/tinnitus)
Headache
Speech/Language disturbances (e.g., dysarthria, hypophonia)
Subcutaneous Hemorrhage/Seroma
Cerebral Spinal Fluid Abnormality
Eye visual disturbances (e.g., diplopia, abnormal vision, visual field defect) and eye disorders (e.g., 
twitching)
Cognitive disturbances (e.g., thinking abnormal, confusion, alteration of mentation) and 
Neuropsychological disturbances (e.g., depression, anxiety)
14.2  
Describe any anticipated risks and discomforts not listed above:
RISKS: 
Potential Risks
General risks of DBS surgery (risks incurred by any patient having DBS implantation surgery, within or outside of 
this protocol)
 
a
General Anesthesi
There are always risks with general anesthesia. This may include a reaction to medications, myocardial 
infarction, or even death. There may be unknown risks.
Risks of the surgical procedure
Implanting the neurostimulator system carries the same risks associated with any other brain surgery. Risks may 
include:
Paralysis, coma, and/or death
Bleeding inside the brain (stroke)
Leaking of fluid surrounding the brain
Air embolism (air bubble that enters an exposed blood vessel on the surface of the brain or in the skull 
bone, which can travel to the heart and possibly lung, a potentially life-threatening condition)
Seizures
Infection
Allergic response to implanted materials
Temporary or permanent neurological complications, including but not limited to decline in mental status, 
problems with speech, problems with movement or coordination
Confusion or attention problems
Pain at the surgery sites
Headache

Changes in hearing
Possible Device Complications
There may be pain, lack of healing, or infection where the brain stimulation system parts are implanted. If 
this happens, further surgery or removal of part of the system may be necessary.
The brain’s stimulation system parts may wear through the skin which can cause an infection or scarring.
The brain leads or lead/extension connectors may move. Further surgery to re-adjust the location may 
be needed.
Components or parts of the brain stimulation system may suffer mechanical breakage resulting in loss of 
therapy. Further surgery to replace the system parts may be needed.
The brain stimulation system could stop because of an electrical or software malfunction, which could 
require further surgery if noninvasive attempts to restore the software did not succeed.
Battery in the pulse generator could be prematurely depleted. This would require further surgery. Pulse 
generator battery service life depends on individual use, but for most patients this should be > 2 years. 
There may be an allergic reaction to the brain stimulation system. The system materials coming in 
contact with the tissues include titanium, polyurethane, silicone, and nylon. The body could also reject 
the system (as a foreign body).
There is the possibility of tissue damage resulting from the programming parameters or a malfunction of 
one of the parts of the brain stimulation system. 
14.3  
MINIMIZING RISKS: Describe the steps you have taken to minimize the risks/discomforts to 
subjects. Examples include:
designing the study to make use of procedures involving less risk when appropriate
minimizing study procedures by taking advantage of clinical procedures conducted on the 
study participants
mitigating risks by planning special monitoring or conducting supportive interventions for the 
study
having a plan for evaluation and possible referral of subjects who report suicidal ideation
a. An attending pediatric anesthesiologist will administer and monitor the patient during the 
General Anesthesi
administration of all anesthesia and will be present during surgery.
Surgical risks will be minimized by utilizing a highly skilled surgical team that 
Risks of the surgical procedure. 
has performed over 1500 DBS device implants over the last 17 years. All procedures will be performed by a 
neurosurgeon specializing in DBS surgery who has personally performed over 1000 of these operations, at least 
100 of which have been in children, which is one of the largest pediatric cohorts in the world. The study will 
exclude participants with major comorbidity increasing the risk of surgery (prior stroke, severe hypertension, 
severe diabetes, or need for chronic anticoagulation other than aspirin). After surgery, the patients will be 
admitted to a pediatric intensive care unit or neurosurgical transitional care unit and monitored for any 
postoperative complications. These specialized units have managed over 1000 postoperative DBS patients. All 
patients will be monitored for bleeding and other neurological changes related to the device or the surgical 
procedure.
The UCSF Surgical Movement Disorder Center has published 
Possible hardware related complications. 
extensively on risk avoidance, and risk management in patients undergoing DBS hardware implantation.
 
16,17
These include having participants wash with an antibacterial soap the evening prior to surgery, administration of 
an intravenous anti-staphylococcal cephalosporin with good skin penetration in the hour prior to incision, use of 
hair clippers only rather than razors, use of appropriate device anchoring techniques to underlying bone and 
fascia, creation of bone troughs to lower device profile in patients with thin skin, use of incisions that do not 
overlie the largest hardware diameter, multilayer closure, use of occlusive dressings, and wound inspection
/staple removal by our own team, rather than an outside physician unfamiliar with device surgery, 10-14 days 
post-procedure.

Stimulation induced dysarthria or weakness: This is a theoretical risk based on side effects reported from 
thalamatomies in this patient population. If these side effects occur, decreasing or turning off stimulation should 
eliminate them.
Infection: We plan to increase the peri-operative antibiotic coverage to include methylcillin-resistance 
staphylococcus. Additionally, we will add a 10 day post-operative course of oral antibiotics to our infection 
prevention regimen as this has been suggested to improve post-operative infection rates in these children.
Depression: As part of screening and recording of adverse events at each interim visit, we will ask the patient 
and/or caregiver if the patient has experienced mood changes. In the event of severe worsening of mood and/or 
suicidal ideation, the study doctor will refer to appropriate psychiatry services, urgently consult with the medical 
safety monitor and log as an SAE.
Speech: As part of screening and recording of adverse events at each interim visit, the subject and caregiver will 
be asked about speech changes. Speech will also be assessed as part of dystonia rating scales by the study 
team. If there are negative changes detected on either AE recording or dystonia rating scales, the DBS will be 
turned off briefly to determine if these changes are stimulation induced. If these changes are not resolved with 
programming adjustment, the formal speech testing done initially on neuropsychological testing will be repeated, 
and this will be recorded as an AE.
Cognitive Changes: As part of screening and recording of adverse events at each interim visit, if the patient or 
caregiver endorses subjective cognitive changes, neuropsychological testing will be repeated. If there are 
negative changes on neuropsychological testing that cannot be resolved by stimulation changes, the study 
doctor will consult with the medical safety monitor.
Weight gain: Body mass index (BMI) will be measured at every visit and monitored for changes. Changes to BMI 
that are not attributable to improvement in the patient’s condition or normal growth will be logged as an adverse 
event.
Additional time: Study visits will be conducted in conjunction with usual clinical visits, when possible, to minimize 
the amount of extra time needed to participate in the study
14.4  
Describe the resources in place to conduct this study in a way that assures protection 
RESOURCES: 
of the rights and welfare of participants:   These resources typically include appropriately trained 
and qualified personnel (in terms availability, number, expertise and experience), funding, space, 
  
equipment, and time to devote to study activities.  Depending on the nature of the research study, 
investigators should consider the proximity or availability of critical resources that may be essential 
to the safety and welfare of participants, such as
the proximity of an emergency facility for care of participant injury
availability of psychological support after participation
resources for participant communication, such as language translation services
Both Investigators, study coordinators and clinical study staff are experienced in the conduct of human 
research and adept at maintaining safety, confidentiality and clinical care for patients enrolled in clinical 
trials. Each study role has back-up staff to insure availability and continuity of care.
14.5  
BENEFITS: 
* 
(REQUIRED) Note: These are the benefits that the IRB will consider during their review. 
They are not necessarily appropriate to include in the consent form.
Possible immediate and/or direct benefits to participants and society at 
large (check all that apply):
Positive health outcome (e.g. improvement of condition, relief of pain, increased mobility, etc.)
Closer follow-up than standard care may lead to improved outcomes or patient engagement
Health and lifestyle changes may occur as a result of participation
Knowledge may be gained about their health and health conditions
Feeling of contribution to knowledge in the health or social sciences field
The research presents a reasonable opportunity to further the understanding, prevention, or alleviation 
of a serious problem affecting the health or welfare of children
Other benefit (describe below)

None
14.6  
Explain why the risks to subjects are reasonable in relation to anticipated 
RISK TO BENEFIT RATIO: 
benefits, if any, to the participant or society:
There are very few options for treating secondary dystonia, and most of the options are medications that 
carry very significant side effects, especially sedation. The potential of both reducing medications, 
improving quality of life, improving ability to function independently is worth the signficant risk that this 
study proposes.
15.0
 
Data and Safety Monitoring Plan
15.2  
DATA AND SAFETY MONITORING PLAN: 
* 
(REQUIRED)
All greater than minimal risk studies are required to provide a plan. Lack 
of an adequate plan is one of the most common reasons why IRB approval 
is delayed.
 
Instructions:
Describe the plan for monitoring data quality and participant safety. Key areas that 
should be included in the plan are:
An explanation of the plan to monitor data collection, study progress, and 
safety
A description of who will perform the monitoring and at what frequency (e.g., 
the PI only, a contract research organization, a Data and Safety Monitoring 
Board or Data Monitoring Committee, etc.)
The type of data and events that will be reviewed (e.g., adverse events, 
breaches of confidentiality, unanticipated problems involving risk to 
participants or others, unblinded efficacy data, etc.)
Procedures and timeline for communicating monitoring results to the UCSF 
IRB, the study sponsor, and other appropriate entities
Assurance that the research team will adhere to the UCSF IRB reporting 
requirements
As appropriate:
A plan for conducting and reporting interim analysis
Clearly defined stopping rules
Clearly defined rules for withdrawing participants from study interventions
The study participants will be followed bi-monthly for the first year and then as frequently as clinically 
indicated for the second year. Safety assessments will be done at every visit including vital signs, limited 
physical exam, safety check of the DBS device. Any Adverse Experience will be recorded and the subject 
diary will be reviewed to detect any adverse experiences. The Investigator will maintain constant vigilance 
over the study and review the data no less than on a monthly basis. The data will be presented and 
reviewed by the MSM at least every 6 months. Data will be presented and reviewed by the IRB annually.
Study stopping criteria are two or more patients with study related serious adverse events i.e. 
symptomatic intracranial hemorrhage, subdural hematoma, seizures, worsening neurological status, or 
death.

Subject stopping criteria would be (1) greater than 40% worsening on the Burke-Fahn-Marsden dystonia rating 
scale (BFMDRS) or the Barry Albright dystonia scale (BADS) that does not improve with programming changes 
(2) Depression that does not improve with programming changes or suicidiality. Any patient that is withdrawn 
from the study will be followed as part of intention to treat analysis.
15.3  
Will a Data and Safety Monitoring Board (DSMB) 
DATA AND SAFETY MONITORING BOARD (DSMB): 
* 
be established: (REQUIRED)
Yes 
No 
15.4  
Provide details from the DSMB's charter, including meeting frequency, and 
DSMB DETAILS: 
affiliations and qualifications of members:   If the DSMB has not yet been established, submit these 
details to us as they become available.
All AEs will also be reported to the, medical safety monitor (MSM) who is a neurosurgeon (Dr. Paul 
Larson) within the institution with no direct involvement in this study but who have expertise in 
implantable devices. Treatment related adverse events assessed as definitely, probably, or possibly related 
to study procedures and either serious or unexpected, noted by any study personnel will be reported 
within 5 working days of their knowledge of the event to the MSM.  The MSM will then advise the PI on 
potential changes in procedures to improve safety, or, in the event of multiple serious adverse events, 
may invoke stopping rules.
16.0
 Confidentiality, Privacy, and Data Security
16.1  
Indicate how subject privacy will be protected:
PROTECTING PRIVACY: 
Conduct conversations about the research in a private room
Ask the subject how they wish to be communicated with – what phone numbers can be called, can 
messages be left, can they receive mail about the study at home, etc.
Take special measures to ensure that data collected about sensitive issues do not get added to their 
medical records or shared with others without the subject’s permission
Other methods (describe below)
16.2  
Do any of the instruments ask about illegal or stigmatized behavior:
SENSITIVE DATA: 
  Yes   
  No
16.3  
Could a breach of privacy or 
CONSEQUENCES OF A LOSS OF PRIVACY OR CONFIDENTIALITY: 
confidentiality  result in any significant consequences to participants, such as criminal or civil 
liability, loss of state or federal benefits, or be damaging to the participant's financial standing, 
employability, or reputation:
  Yes   
  No
16.4  
Explain any extra steps that will be taken to assure 
EXTRA CONFIDENTIALITY MEASURES: 
confidentiality and protect identifiable information from improper use and disclosure, if any:
Data will be stored in a computerized spreadsheet with identifiers removed and information anonymized 
when entered into the file. The link between identifying information (i.e., subject’s full name) and the 
research code will be stored in an encrypted database. Computer-based files will only be made available 
to personnel involved the study through the use of access privileges and passwords.
 
The database portal can be accessed only from the UCSF Surgical Movement Disorders offices and from 
other approved locations with VPN access and authenticated by the database manager.  
 

De-identified patient data may be transmitted and received via secure network with other movement 
disorders centers.  Patient data will include demographics, clinical history, surgical data, clinical scales (for 
example, Burke-Fahn-Marsden and Barry-Albright Dystonia scales), surgical complications, medication 
information, and programming data.
 
16.5  
Do you anticipate that this study may collect information that State or Federal 
 REPORTABILITY: 
*
law requires to be reported to other officials, such as elder abuse, child abuse, or threat to self or 
others: (REQUIRED)
  Yes   
  No
16.6  
Will this study obain a Certificate of Confidentiality:
CERTIFICATE OF CONFIDENTIALITY: 
  Yes   
  No
16.7  
Will there be any sharing of 
 research test results 
SHARING OF RESEARCH RESULTS: 
EXPERIMENTAL
with subjects or their care providers:
 Yes   
 No
Note: This is unusual and not recommended, particularly in cases where 
the tests are carried out in a non-CLIA certified laboratory, the results are 
of unproven clinical significance, or where there are not known 
preventative strategies and/or treatments. If these are the most likely 
scenarios for your study, you should check 'No.'  
 
If you have an incidental finding of clear clinical significance, call the 
HRPP QIU at 415-476-1814 for a consult. 
Explain under what circumstances research results may be shared:
Results of Neuropsychological testing will be shared with the subject and caregivers and they will be 
counseled on the results.
16.8  
Will any personal identifiers be collected: 
IDENTIFIERS: 
* 
(REQUIRED) 
 Yes   
 No
Check all the identifiers that may be included:
Names
Dates
Postal addresses
Phone numbers
Fax numbers
Email addresses
Social Security Numbers*
Medical record numbers
Health plan numbers
Account numbers
License or certificate numbers
Vehicle ID numbers
Device identifiers or serial numbers
Web URLs

IP address numbers
Biometric identifiers
Facial photos or other identifiable images
Any other unique identifier
If publications from this study may include ANY photos or images of 
patients - even without faces - either collected for research or from the 
medical records, you are required to have each patient sign the 'Consent 
for Photography / Authorization for Publication' form prior to submittal 
for publication. Failure to obtain consent for publication may result in a 
finding of Serious Non-compliance by the IRB and civil and criminal 
penalties, including fines up to $1.5 million dollars for violation of the 
HIPAA privacy protections if a participant complains.  
Could study records include 
 photos or images (even 'unidentifiable' ones): 
* 
ANY
(REQUIRED)
 Yes   
 No
16.9  
 
Will identifiable information be shared with outside groups:
DATA DISCLOSURE: 
  Yes   
  No
16.11  
(check all that apply): 
* DATA COLLECTION AND STORAGE: 
(REQUIRED)
Collection methods:
Paper-based (surveys, logs, diaries, etc.)
Electronic case report forms (CRFs), such as OnCore or another clinical trial management portal
Web-based online surveys or computer-assisted interview tool
Mobile applications (mobile or tablet-based)
Wearable devices
Audio/video recordings
Other:
Data will be collected/stored in systems owned by (check all that apply): 
* 
(REQUIRED)
UCSF
SF VAMC
Amazon (Amazon Cloud)
Other academic institution
3rd party vendor (business entity)
Other (explain below)
16.12  
Indicate how data are kept secure and protected from improper use and 
DATA SECURITY: 
disclosure (check all that apply):   NOTE: Whenever possible, do not store subject identifiers on 
laptops, PDAs, or other portable devices. If you collect subject identifiers on portable devices, 
you MUST encrypt the devices.
Data are stored securely in My Research
Data are coded; data key is destroyed at end of study
Data are coded; data key is kept separately and securely
Data are kept in a locked file cabinet
Data are kept in a locked office or suite

Electronic data are protected with a password
Data are stored on a secure network
Data are collected/stored using REDCap or REDCap Survey
Data are securely stored in OnCore
16.13  
  
Confirm below that you will keep data confidential: 
* DATA SECURITY: 
(REQUIRED)  I will keep 
any data sets that include identifiers secure and protected from improper use and disclosure by 
using methods such as:
Physical Security – Keeping data in locked file cabinets, locked offices, locked suites, and 
physically securing computers and servers.
Electronic Security – Following UCSF minimum security standards for electronic information 
, which includes (but is not limited to): not storing identifiers on portable devices 
resources
like laptops or flash drives if they are unencrypted, encrypting portable devices, and storing 
data in password-protected files and on secure networks.
 
Yes 
16.15  
Study data will be:
HIPAA APPLICABILITY: 
 
Derived from the Integrated Data Repository (IDR) or The Health Record Data Service (THREDS) at 
SFGH
Derived from a medical record (e.g. APeX, OnCore, etc. Identify source below)
Added to the hospital or clinical medical record
Created or collected as part of health care
Obtained from the subject, including interviews, questionnaires
Obtained ONLY from a foreign country or countries
Obtained ONLY from records open to the public
Obtained from existing research records
None of the above
Unless a waiver of Authorization is granted, in addition to the consent 
 
form, participants will need to sign UCSF Research Subject Authorization 
Form (HIPAA Form). NEW REQUIREMENT - This form should be uploaded 
in the Other Study Documents section of the Initial Review Submission 
Failure to obtain HIPAA Authorization for research is one of 
Packet Form. 
the most common findings from QIU Routine Site Visits. Your IRB 
approval letter will include instructions about HIPAA requirements 
specific to your study.
If derived from a medical record, identify source:
APEX, records sent from outside providers
16.16  
Does the research require access to any of 
HIPAA - PERMISSION TO ACCESS SENSITIVE DATA: 
* 
the following types of health information from the medical record: (check all that apply) 
(REQUIRED)
Drug or alcohol abuse, diagnosis or treatment
HIV/AIDS testing information
Genetic testing information
Mental health diagnosis or treatment
None of the above

17.0
 Financial Considerations
17.1  
Will subjects be paid for participation, reimbursed for time or expenses, or receive any 
* PAYMENT: 
other kind of compensation: (REQUIRED)
 Yes   
 No
17.2  
Subjects payment or compensation method (check all that apply):
PAYMENT METHODS: 
Payments will be (check all that apply):
Cash
Check
Gift card
Debit card
UCSF Research Subject Payment Card
Reimbursement for parking and other expenses
Other:
17.3  
Describe the schedule and amounts of payments, including the total subjects 
PAYMENT SCHEDULE: 
can receive for completing the study: 
If there are multiple visits over time, explain how payments will be prorated for partial 
completion
If deviating from recommendations in Subject Payment Guidelines, include specific 
justification below
17.4  
Will subjects or their insurance be charged for any study activities:
COSTS TO SUBJECTS: 
 Yes   
 No
Describe the costs that may be incurred by subjects or 3rd party payers as a result 
of participation:
Explain why it is appropriate to charge those costs to the subjects
ompare subjects’ costs to the charges that 
If this is a therapeutic study, c
would typically be associated with receiving care off-study (e.g. is it more 
expensive to participate in this study than to receive care off-study?)
Although the goals of the study are to assess safety and tolerability, the secondary are to treat the 
subject's dystonia with an approved medical device that we are using off-label (with an IDE) for this 
patient population. Inclusion criteria specify that there are no other treatment options available to the 
participants. When we obtain authorization from the subjects insurance carrier for implantation of the DBS 
device, we will also verify that the subject's insurance will also provide coverage for any possible 
explantation of the device.
18.0
 Qualifications of Key Study Personnel
18.1          
 
  NOTE: This information is required and your application will be 
considered incomplete without it. If this study involves invasive or risky 
procedures, or procedures requiring special training or certification, please 
identify who will be conducting these procedures and provide details 
about their qualifications and training. Also identify each person who will be 

involved in the consent process. Click the orange question mark for more 
    
   
information and examples.
Under qualifications, please include:
Academic Title
Institutional Affiliation (UCSF, SFGH, VAMC, etc.)
Department
Certifications
        
  
  
 
November, 2015 - NEW Definition of Key Study Personnel and 
    
CITI Training Requirements:
  
  
include the Principal Investigator, other 
UCSF Key Study Personnel 
investigators and research personnel who are directly involved in conducting 
research with study participants or who are directly involved in using study 
participants’ identifiable private information during the course of the research. 
Key Personnel also include faculty mentors/advisors who provide direct 
oversight to Postdoctoral Fellows, Residents and Clinical Fellows serving as PI 
        
on the IRB application.
 
The IRB requires that all Key Study Personnel 
complete Human Subjects Protection Training through 
 
CITI prior to approval of 
a new study, or a modification in which KSP are being added. More information 
  
  
on the CITI training requirement can be found on our website.  
      
KSP Name
Description of Study 
Responsibilities - Briefly 
describe what will each person 
be doing on the study. If there 
are procedures requiring special 
expertise or certification, 
identify who will be carrying 
these out. Also identify who will 
be obtaining informed consent.
Qualifications, Licensure, and 
Training
Dr. Ostrem, Jill M.D.
As study investigator, Dr. 
Ostrem will help interpret and 
analyze the data.
Dr. Ostrem is fellowship 
trained in movement 
disorders, board certified in 
Neurology and an Associate 
Professor of
Neurology at UCSF. She has
extensive clinical and research 
experience in the area of 
medical and surgical 
movement disorders.
Dr. Starr, Philip MD, PhD
Dr. Starr will perform study 
neurosurgical procedures and 
participate in evaluation of 
study data.
Dr. Starr is board certified in 
neurosurgery, fellowship 
trained in movement disorders 
surgery, board certified in 
neurosurgery, and a Professor 
of Neurosurgery at UCSF. He 
has performed >800 deep 
brain stimulator implants for 
movement disorders and is 
one of the most experienced 
functional neurosurgeons in 
the world. He has published 
extensively on deep brain 
stimulation and dystonia 
physiology.
Volz, Monica M
As study nurse, Ms. Volz will 
consent patients, manage and 
program study patients and 
record data.
Monica Volz, MS, RN, is a 
clinical nurse specialist with 10 
years of experience managing 
clinical trials and movement 
disorders patients treated with 
deep brain stimulation.

Dr. Demopoulos, Carly PhD
As study neuropsychologist, 
Dr. Demopoulous will 
administer neuropsychological 
testing and help interpret 
results.
Dr. Demopoulous has a Ph.D. 
in neuropsychology with 
fellowship training. She has 
special expertise in testing 
children with communication 
difficulties.
Racine, Caroline
As a study investigator and 
neuropsychologist, Dr. Racine 
will help interpret with results 
of neuropsychological testing.
Dr. Racine is an experienced 
clinical neuropsychologist at 
UCSF with special expertise in 
movement disorders.
Dr. San Luciano Palenzuela, 
Marta M.D., MD
As study P.I., Dr. San Luciano 
will be responsible for 
overseeing the study, 
recruiting subjects, evaluating 
and examination of subjects, 
recording data, and data 
analysis and interpretation.
Dr. San Luciano is an 
Assistant Clinical Professor of 
Neurology at UCSF and is 
fellowship- trained in 
movement disorders and has 
experience in DBS.
Larson, Paul S, MD
Dr. Larsen will serve as the 
neurosurgeon on the Data 
Safety Monitoring Committee
Dr. Larsen is board certified in 
neurosurgery, board certified 
in neurosurgery, and a 
Professor of Neurosurgery at 
UCSF. He is one of the most 
experienced functional 
neurosurgeons in the world. 
He has published extensively 
on deep brain stimulation.
Wang, Sarah S
Dr. Wang will help with project 
logistics and help provide 
regulatory oversight and CHR 
requirements.
Dr. Wang is a Senior Research 
Coordinator in the UCSF 
Surgical Movement Disorders 
Center.
Watson, Christa L
As study neuropsychologist, 
Dr. Watson will administer 
neuropsychological testing and 
help interpret results.
Dr. Watson has a Psy D and is 
a post doctoral fellow at the 
Dyslexia Center at UCSF. She 
has special expertise in 
working with pediatric patients.
Gittings, Melissa L
As a research coordinator, 
Melissa will work on data 
entry, data reporting, and 
other regulatory 
responsibilities.
Melissa is a clinical research 
coordinator in the UCSF 
Movement Disorders and 
Neuromodulation Center.
Dr. Bledsoe, Ian MD
As study investigator, Dr. 
Bledsoe will help interpret and 
analyze the data.
Dr. Bledsoe is an Assistant 
Clinical Professor of Neurology 
at UCSF and is fellowship- 
trained in movement disorders 
and has experience in DBS.
Viser, Aaron
As a research coordinator, 
Aaron will work on data 
collection through chart 
review, data verification, and 
manuscript preparation.
Aaron is a clinical research 
coordinator in the UCSF 
Movement Disorders and 
Neuromodulation Center.
19.0
 Other Approvals and Registrations
19.1  
 
Does this study involve administration of vaccines 
* ADMINISTRATION OF RECOMBINANT DNA: 
produced using recombinant DNA technologies to human subjects 
: 
(Help Link added Aug '15)

(REQUIRED)
  Yes   
  No
19.2  
Does this study involve human gene transfer (NOTE: Requires NIH 
* HUMAN GENE TRANSFER: 
Recombinant DNA Advisory Committee (RAC) review prior to IRB approval): (REQUIRED) 
  Yes   
  No
19.4  
Indicate if this study involves other regulated materials and requires approval 
OTHER APPROVALS: 
and/or authorization from the following regulatory committees:
Institutional Biological Safety Committee (IBC)
Specify BUA #:
Institutional Animal Care and Use Committee (IACUC)
Specify IACUC #:
Controlled Substances
20.0
 End of Study Application
20.1  End of Study Application Form        
 
  To continue working on the Study 
: Click on the section you need to edit in the left-hand menu. Remember to save 
Application
        
through the entire Study Application after making changes.
 
If you are done working on 
        
:
the Study Application
 
Before proceeding, please go back to Section 
Important: 
4.0 Initial Screening Questions and Save and Continue through the form to make sure all the 
relevant sections and questions have been included. If you've changed any answers since 
you started, the branching may have changed. Your application will be incomplete and it will 
        
have to be returned for corrections. 
 
Once you are sure the form is complete, click 
Save and Continue. If this is a new study, you will automatically enter the Initial Review 
Submission Packet form, where you can attach consent forms or other study documents. 
        
Review the 
 for a list of required attachments.
Initial Review Submission Checklist
 
  
       
Answer all questions and attach all required documents to speed up your approval.
 
       
  
              
 
 
 
 
 
The UCSF IRB wants your feedback about this new form. Please click 
the link to take a 
 about the new application form.
brief survey