Conditions: Limb-girdle Muscular Dystrophy Becker Muscular Dystrophy
Keywords: Steroids, Prednisone
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 20
Healthy volunteers: no

Brief summary:
The purpose of this study is to evaluate the safety and efficacy of oral weekly glucocorticoid steroids in patients with Becker Muscular Dystrophy (BMD), an inherited disorder in which patients experience weakness of the legs and pelvis, and Limb Girdle Muscular Dystrophy (LGMD), an inherited disorder in which patients experience progressive muscular weakness predominately in their hip and shoulders. The primary objective is safety which we the investigators will measure using laboratory testing and forced vital capacity (FVC), a breathing test that measures the strength of your lungs. The secondary objective is efficacy which will be measured by a change in MRI muscle mass, improved muscle performance, and quality of life.

The investigators hypothesize that patients who receive oral weekly glucocorticoid steroids will have improviements in strength and quality of life compared to their baseline. Furthermore, the investigators anticipate that oral weekly glucocorticoid steroids will not have significant adverse impact on patients.

Detailed description:
Glucocorticoid (GC) steroids are a mainstay of therapy for Duchenne Muscular Dystrophy, where they have been shown to prolong ambulation in for DMD in random clinical trials (Gloss et al., 2016). Dosing regimen vary for DMD, but most trials utilized oral daily dosing at 0.75- 1 mg/kg of prednisone or deflazacort (Birnkrant et al., 2018). The age at which to begin oral glucocorticoids and the age at which to cease steroid use are not well established by clinical trial investigation. High dose weekend dosing of oral glucocorticoid steroids has also been suggested to be noninferior to daily dosing when evaluated in a year-long study in DMD, and this approach is preferred in some settings since related to a reduced side effect profile, particularly with respect to behavioral changes which can occur with daily GC steroid dosing in children (Escolar et al., 2011). The use of GC steroids for other forms of muscular dystrophy, including Becker Muscular Dystrophy (BMD) and the Limb Girdle Muscular Dystrophies (LGMDs) is not considered standard of care and has insufficiently been investigated by randomized clinical trials (RCT). An RCT of GC steroids in LGMD 2B (DYSF mutations) was associated with unfavorable outcomes in the steroid treated group (Walter et al., 2013).

Recently, weekly steroid dosing was investigated in preclinical mouse models of muscular dystrophy, including the mdx mouse model of DMD/BMD and two models of LGMD, including LGMD 2B (DYSF) and 2C (SGCG) (Quattrocelli et al., 2017a; Quattrocelli et al., 2017b). All three models showed improved strength and reduced fibrosis with weekly GC steroid dosing. Moreover, in unpublished data, long term studies (24-52 weeks duration) in mice, showed favorable results with improved muscle strength in the mdx and DYSF models.

The investigators propose to carry out an open label safety and efficacy trial of oral weekly GC steroids in patients with BMD and LGMD subtypes. Subjects will be recruited based on age, molecular diagnosis of BMD and LGMD subtypes, and willingness to participate. Both ambulatory and nonambulatory subjects will be included. Subjects will be excluded if they have diabetes mellitus, full time ventilator use, or severely compromised cardiac function, including symptoms referable to heart failure. Subjects must provide consent.

Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM. Prior to initiation, subjects will provide a blood sample for baseline screening including serum chemistries, HgbA1-C, creatine kinase, and lipid panel (HDL, LDL, triglycerides, and total cholesterol) and for exploratory biomarkers. Subjects will also provide a urine sample to analyze changes in metabolic biomarkers that are excreted. Subjects will have a physical exam and medical record review. Subjects will have strength testing and complete 10 meter timed run test in addition to a 6 min walk test (if ambulatory). Subjects will be asked to complete quality of life questionnaire. At 6 months, subjects will be evaluated with a physical exam, strength testing, spirometry, 10 meter timed run test and 6 min walk test (if ambulatory), blood draw for serum chemistry, HgbA1-C, creatine kinase, lipid panel and for exploratory biomarkers. Subjects will also provide a urine specimen to be analyzed for any changes in excretion of metabolic markers as an exploratory endpoint. Subjects will be asked to complete a quality of life questionnaire. An MRI/ MRS will be performed before starting GC oral prednisone and at 6 months.

Arms:
arm 1: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM

Interventions: intervention 1: Prednisone
Intervention descriptions: intervention 1: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM

Protocol document:
Prot_001.pdf: 
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Weekly Steroids in Muscular Dystrophy 
 
NCT 04054375 
 
12 August 2019


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Weekly Steroids in Muscular Dystrophy 
 
PROTOCOL TITLE:  Open label safety and efficacy of once weekly steroid in patients with 
LGMD and Becker muscular dystrophy 
 
SHORT TITLE:  Weekly Steroids in Muscular Dystrophy (WSiMD) 
 
PRINCIPAL INVESTIGATOR: 
Senda Ajroud-Driss, MD 
Division of Neurology 
(312) 503-0671 
s-ajroud@northwestern.edu 
 
Co-Investigators: Elizabeth McNally, MD and Robert Sufit, MD 
 
Research Coordinator: Benjamin Joslin, Aaron Zelikovich 
 
VERSION NUMBER: 5.0 
 
VERSION DATE:  12Aug19 
  
STUDY SUMMARY: 
 
Investigational Agent(s)  
(Drugs or Devices) 
Oral prednisone 
IND / IDE / HDE #  N/A 
Indicate 
Special Population(s) 
Children  
 Children who are wards of the state  
 Adults Unable to Consent   
 Cognitively Impaired Adults  
 Neonates of Uncertain Viability  
 Pregnant Women  
 Prisoners (or other detained/paroled individuals)  
 Students/Employees  
Sample Size 30 
Funding Source Kurt+Peter Foundation 
Indicate the type of consent 
to be obtained 
Written  
Verbal/Waiver of Documentation of Informed Consent 
Waiver of HIPAA Authorization  
Waiver/Alteration of Consent Process   
Site 
 Lead Site ( For A Multiple Site Research Study) 
 Data Coordinating Center (DCC) 
Research Related 
Radiation Exposure 
Yes  
 No  
DSMB / DMC / IDMC 
Yes  
No 
 
OBJECTIVES: 
The purpose of this study is to evaluate the safety and efficacy of oral weekly glucocorticoid 
steroids in patients with Becker Muscular Dystrophy (BMD) and Limb Girdle Muscular Dystrophy 


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(LGMD). The primary objective is safety which we will measure using laboratory testing and 
forced vital capacity (FVC). The secondary objective is efficacy which will be measured by a 
change in MRI muscle mass, improved muscle performance, and quality of life. 
 
We hypothesize that patients who receive oral weekly glucocorticoid steroids will have clinically 
significant improvements in strength and quality of life compared to their baseline. Furthermore, 
we anticipate that oral weekly glucocorticoid steroids will not have a significant adverse impact 
on patients. 
 
BACKGROUND: 
Glucocorticoid (GC) steroids are a mainstay of therapy for Duchenne Muscular Dystrophy, 
where they have been shown to prolong ambulation in for DMD in random clinical trials (Gloss 
et al., 2016).  Dosing regimen vary for DMD, but most trials utilized oral daily dosing at 0.75- 1 
mg/kg of prednisone or deflazacort (Birnkrant et al., 2018).  The age at which to begin oral 
glucocorticoids and the age at which to cease steroid use are not well established by clinical 
trial investigation.  High dose weekend dosing of oral glucocorticoid steroids has also been 
suggested to be noninferior to daily dosing when evaluated in a year-long study in DMD, and 
this approach is preferred in some settings since related to a reduced side effect profile, 
particularly with respect to behavioral changes which can occur with daily GC steroid dosing in 
children (Escolar et al., 2011).  The use of GC steroids for other forms of muscular dystrophy, 
including Becker Muscular Dystrophy (BMD) and the Limb Girdle Muscular Dystrophies 
(LGMDs) is not considered standard of care and has insufficiently been investigated by RCTs.  
An RCT of GC steroids in LGMD 2B (DYSF mutations) was associated with unfavorable 
outcomes in the steroid treated group (Walter et al., 2013). 
 
Recently, weekly steroid dosing was investigated in preclinical mouse models of muscular 
dystrophy, including the mdx mouse model of DMD/BMD and two models of LGMD, including 
LGMD 2B (DYSF) and 2C (SGCG) (Quattrocelli et al., 2017a; Quattrocelli et al., 2017b).  All 
three models showed improved strength and reduced fibrosis with weekly GC steroid dosing.  
Moreover, in unpublished data, long term studies (24-52 weeks duration) in mice, showed 
favorable results with improved muscle strength in the mdx and DYSF models. 
 
We propose to carry out an open label safety and efficacy trial of oral weekly GC steroids in 
patients with BMD and LGMD subtypes.  Subjects will be recruited based on age, molecular 
diagnosis of BMD and LGMD subtypes, and willingness to participate.   Both ambulatory and 
nonambulatory subjects will be included.  Subjects will be excluded if they have diabetes 
mellitus, full time ventilator use, or severely compromised cardiac function, including symptoms 
referable to heart failure.   Subjects must provide consent.  
 
Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for 
patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more 
than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after 
their last meal between 7 and 9 PM. Prior to initiation, subjects will provide a blood sample for 
baseline screening including serum chemistries, HgbA1-C, creatine kinase, and lipid panel 
(HDL, LDL, triglycerides, and total cholesterol) and for exploratory biomarkers.  Subjects will 
also provide a urine sample to analyze changes in metabolic biomarkers that are excreted. 
Subjects will have a physical exam and medical record review.  Subjects will have strength 
testing and complete 10 meter timed run test in addition to a 6 min walk test (if ambulatory).   
Subjects will be asked to complete quality of life questionnaire.  At 6 months, subjects will be 
evaluated with physical exam, strength testing, spirometry, 10 meter timed run test and 6 min 


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walk test (if ambulatory), blood draw for serum chemistry, HgbA1-C, creatine kinase, lipid panel 
and for exploratory biomarkers.  Subjects will also provide a urine specimen to be analyzed for 
any changes in excretion of metabolic markers as an exploratory endpoint.  Subjects will be 
asked to complete a quality of life questionnaire. An MRI/ MRS will be performed before starting 
GC oral prednisone and at 6 months. 
 
STUDY ENDPOINTS: 
Primary Outcome: Safety 
 
Safety monitoring: 
• 
Comprehensive chemistry panel, hemoglobin A1C, CK, lipid panel at screening and 
after 6 months of GC steroid dosing 
• 
FVC (sitting, supine) at screening and at 6 months 
• 
Phone calls to each patient each month to determine their state of health and study 
drug compliance. 
 
Secondary Outcomes: Change in MRI muscle mass, improved muscle performance, improved 
quality of life, blood and urine metabolic biomarker changes. 
 
Functional Assessments 
• 
NSAD (Northstar Assessment for Dysferlinopathy)—AMB 
• 
6 min walk test and 10 meter timed run test, if patient ambulatory 
• 
PUL (Performance of Upper Limb) 
• 
Brooke Scale 
• 
Vignos Scale 
 
Strength assessments 
• 
MMT MRC 
 
Patient Reported Outcome Measures 
• 
PROM: ACTIVLM 
• 
Quality of life questionnaire 
 
Blood and urine for exploratory biomarkers 
 
Imaging 
• 
MRI/ MRS of leg and arm muscles to measure muscle volume, T2 value and fat 
fraction 
• 
DEXA scan at screening and at 6 months 
 
Outcome measures: 
• 
Comparing MRI/MRS changes the leg and arm muscles at screening and 6 months 
• 
Change in 6 min walk test and 10 meter timed run test for ambulatory patients 
• 
Change in strength assessment 
• 
Change in FVC measurements 
 
Strength assessment: 
• 
MRC scale:  Shoulder abduction, elbow flexion, knee extension and knee flexion, hip 
flexion, foot dorsiflexion. 
• 
Grip strength (Jamar dynamometer) 


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• 
Hand held dynamometry 
 
Functional testing: 
• 
Lift arm above head  
• 
Lift arm above head using 50g/100g/200g/500g weight 
• 
Bring arm to the mouth 
• 
Bring weight to mouth using 50g/100g/200g/500g weight 
 
STUDY INTERVENTION(S) / INVESTIGATIONAL AGENT(S): 
Oral prednisone is a synthetic glucocorticoid that is FDA-approved medication for the treatment 
of various inflammatory and allergic conditions. 
 
In this study, subjects will be asked to take weekly GC oral prednisone, dosed based on weight 
(1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who 
weigh more than 70 kg). Subjects will be instructed to take weekly prednisone on Mondays after 
their last meal between 7 and 9 PM. Study drug, oral prednisone, will be stored under the 
secure and controlled environment of the Investigational Drug Pharmacy at Northwestern 
Memorial Hospital. The Investigational Drug Pharmacy will follow its current SOP regarding the 
storage and handling of investigative drugs. The study drug will be dispensed from the 
Pharmacy and administered to subjects once they have signed informed consent, passed 
screening, on Day 0 of their treatment. Subjects will return home with 6 months of study drug 
treatment. They will be educated on dosing, storage, and return of unused study drug by the 
study staff. Subjects will be contacted on a monthly basis to assess medication and dosing 
adherence. They will be asked to confirm their medication and the dosing instructions and re-
educated if not adhering.   
 
PROCEDURES INVOLVED: 
This is an open label study that evaluates the safety and efficacy of oral weekly glucocorticoid 
steroids in patients with BMD and LGMD over 6 months. The study is comprised is three visits; 
screening to determine eligibility, initial dosing (Day 0), and final dosing (Month 6). 
 
Screening 
All subjects will be recruited to the study through the Muscular Dystrophy (MDA) Clinic at 
Northwestern Memorial Hospital. Prior to any study procedures, the study team will complete 
the full informed consent process with each patient. Upon obtaining informed consent, the study 
team will collect information from the patient including: demographics, vital signs, weight, height, 
medical history, concomitant medication, genetic mutation test results, EKG and 
Echocardiogram test results. If the subject does not have EKG or Echocardiogram results within 
the last 2 and 6 months, respectively, they will not be screened until they complete these two 
procedures as part of their standard of care. The principal investigator will review this 
information and determine whether the subject is eligible to participate in the study. If the 
subject is ineligible, he/she will be notified and continue to seek treatment through their treating 
physician.  
 
 
Data will be retained in paper source materials by members of the study team at Abbott Hall, 
1109, 710 N. Lake Shore Dr., Chicago, IL 60611. 
 
 
 


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Day 0 
Subjects will return to the MDA Clinic at Northwestern Memorial Hospital for the Day 0 
procedures. If on a separate day from the screening visit, the study team will review 
concomitant medications, medical history, and collect vital signs with height and weight. 
 
The following laboratory tests will be collected for safety purposes: A comprehensive chemistry 
panel, hemoglobin A1C, CK, and lipid panel.  A blood and urine sample will be taken for 
exploratory biomarkers. Samples will be collected by members of the study team and processed 
by the Northwestern Memorial Hospital Main Laboratory. Records will be populated in the 
patient’s medical charts and stored by members of the study team in the subject binders. 
 
A member of the study team will also collect forced vital capacity in a sitting and supine position. 
 
The subject will be asked to undergo an MRI through the study team at the Center for 
Translational Imaging at Northwestern University.  Before the study participant completes the 
MRI, he or she must answer questions about their medical and surgical history on a MRI safety 
questionnaire to determine whether it will be safe for them to complete the MRI.  If not, they will 
not be eligible to participate in this study. No contrast will be administered. The MRI images will 
be de-identified and electronically transmitted to a secure file transfer (SFT) system to the 
University of Florida for interpretation. 
 
Before dosing occurs, a member of the study team (physical or occupational therapist) will 
complete the following functional assessments: Northstar Assessment for Dysferlinopathy 
(NSAD) –AMB, 10 meter timed run test and 6-minute walk test if the patient is ambulatory, 
performance of upper limb (PUL) evaluation, Brookes and Vignos scales. A member of the 
study team will also collect the strength assessment, MMT MRC. 
 
Subjects will be asked to complete two questionnaires; ACTIVLM and a Quality of Life 
Questionnaire. 
 
The subject will also complete a bone density (DEXA) scan at Northwestern Memorial Hospital. 
 
Monthly Phone Calls 
Between Day 0 and Month 6, a member of the study team will call the subjects on a monthly 
basis to determine their state of health and whether they are taking the medication correctly. 
State of health will be determined by assessed by asking if the subject has experienced any 
changes to their physical health (adverse events).  He or she will also ask the subjects about 
how they are taking the medication and if you are doing so correctly. If the subjects are not, the 
study member will help the subjects by providing additional instructions on how to correctly take 
the study drug. 
 
Month 6 
After 6 months of treatment, subjects will return to the MDA Clinic at Northwestern Memorial 
Hospital. A member of the study team will collect vital signs, weight and height, medical history, 
concomitant medication information, and review adverse events. The subject will also provide a 
sample for safety laboratory analysis (Comprehensive chemistry panel, hemoglobin A1C, CK, 
and lipid panel).  A blood and urine sample will be taken for exploratory biomarkers. 
 
A member of the study team will also collect forced vital capacity in a sitting and supine position. 
 


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A member of the study team (physical or occupational therapist) will complete the following 
functional assessments: Northstar Assessment for Dysferlinopathy (NSAD) –AMB, 10 meter 
timed run test and 6-minute walk test if the patient is ambulatory, performance of upper limb 
(PUL) evaluation, Brookes and Vignos scales. A member of the study team will also collect the 
strength assessment, MMT MRC. 
 
Subjects will be asked to complete two questionnaires; ACTIVLM and a Quality of Life 
Questionnaire. 
 
The subject will also complete a bone density (DEXA) scan at Northwestern Memorial Hospital. 
 
An MRI will be conducted at the Center for Translational Imaging at Northwestern University 
within 2 weeks before or after the subject’s final visit.  No contrast will be administered. 
 
An overview of the study assessments may be found in Table 1.0 below: 
 
  
Screening (D0-2M) 
Day 0 
M6 +2 wks 
Informed Consent 
X 
  
  
Demographics 
X 
  
  
Vital Signs 
X 
X 
X 
Weight and Height 
X 
X 
X 
Medical History 
X 
X 
X 
Concomitant Medications 
X 
X 
X 
Genetic mutation 
Confirmation 
X 
  
  
Review EKG* 
X 
  
  
Review Echo** 
X 
  
  
Safety Monitoring Labs*** 
  
X 
X 
Forced Vital Capacity (FVC) 
  
X 
X 
NSAD 
  
X 
X 
6-min Walk Test / 10 m Run 
  
X 
X 
Performance of Upper Limb 
  
X 
X 
Brooke Scale 
 
X 
X 
Vignos Scale 
  
X 
X 
MMT MRC 
  
X 
X 
Grip Strength 
  
X 
X 
ACTIVLM 
  
X 
X 
Quality of Life Questionnaire 
(SF36) 
  
X 
X 
MRI/MRS 
 
X 
X 
DEXA Scan 
  
X 
X 
Study Drug Dispensation 
  
X 
  
Review of Adverse Events 
  
  
X 
Table 1.0:  Schedule of Assessments from Screening, Day 0, and Month 6 
* EKG – Results within 2 months of study initiation accepted 


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**Echo – Results within 6 month of study initiation accepted 
*** Comprehensive chemistry panel, hemoglobin A1C, CK, lipid panel 
 
SHARING RESULTS WITH PARTICIPANTS 
Results of investigational diagnostic tests, and incidental findings, will be shared with the 
subjects through the MyChart patient portal. Once the laboratory samples are drawn and 
analyzed by the Northwestern Memorial Hospital Main Laboratory, the results will be posted to 
MyChart. If the results are abnormal and clinically significant, the Principal Investigator will notify 
the subject about further testing and treatment. Furthermore, the Principal Investigator will 
contact and inform the subject’s primary care physician. 
 
Results from questionnaires, functional assessments, blood / urine exploratory 
biomarkers, Dexa, and MRI imaging will not be shared with the patient. 
 
STUDY TIMELINES 
The study team will screen and enroll subjects for a period no longer than 1 year from IRB 
approval. 
 
Screening will occur at one clinic visit that will last no more than 2 hours.  If subjects have not 
had a MRI within the previous 6 months, they will return for a MRI (1 hour).  Similarly, if subjects 
have not had a DEXA scan within the previous 6 months, they will return for a DEXA scan (1 
hour).  
 
Enrolled subjects will participate in the study for 6 months from their first dosing (Day 0). 
Subjects will schedule their MRI and DEXA scan on the final day of the study. If subjects are 
unable to complete a MRI or Dexa on that day, they will return to complete the required 
procedure within the next 2 weeks. 
 
The investigators estimate the study’s primary analyses will be completed by December 2019. 
 
INCLUSION AND EXCLUSION CRITERIA 
Inclusion criteria (Collected at screening): 
1. Patients with Becker muscular dystrophy or LGMD2A (CAPN3), LGMD 2B (DYSF), 
LGMD 2C (SGCG), LGMD2E (SGCB), LGMD2F (SGCD), LGMD 2I (FKRP), LGMD 
(ANO5).  Genetic mutation or muscle biopsy staining required to confirm genetic subtype 
2. Ages 18-65 years 
3. EKG without evidence of prior infarct or atrial fibrillation done within 2 months of study 
initiation. 
4. Echocardiogram with LVEF >25% done within 6 months of study initiation. 
5. Stable medications (same medication and dose) for the previous 3 months 
6. Stable pulmonary status for the previous 6 months (No change in FVC by more than 
20% in the past 6-months) 
Exclusion criteria (Collected at screening): 
1. Diabetes  
2. BMI>35 kg/m2  
3. Cardiac transplantation 
4. Myocardia Infarct in the past 2-years from screening 
5. Any history of tuberculosis 
6. Untreated or uncontrolled (medication and/or dose change in previous month from 
screening) hypertension   


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7. A diagnosis of congestive heart failure 
8. A diagnosis of chronic kidney disease 
9. A diagnosis of untreated hypothyroidism 
10. The patient is believed to be at high risk of osteoporosis by the primary investigator 
11. Inability to provide consent  
12. Prior oral glucocorticoid steroid use for > than one month during a subject’s life.  
13. Full time ventilator dependency  
14. Heart failure symptoms or LVEF <25%  
15. Orthopedic surgery within the prior year or upcoming elective orthopedic surgery within 
the 6-months from Day 0. 
16. Inability to complete MRI (claustrophobia, metal implants) 
17. Pregnant women at screening, women seeking to become pregnant, or men seeking to 
father a child within 6-months from Day 0 should not participate in this study. 
 
PARTICIPANT POPULATION(S) 
Accrual 
Number: 
Category/Group: 
(Adults) 
Consented: 
Maximum Number to be 
Consented or 
Reviewed/Collected/Screened 
Enrolled: 
Number to Complete 
the Study or Needed 
to Address the 
Research Question 
Total: 
50 adults 
40 adults 
30 adults 
 
 
RECRUITMENT METHODS 
All subjects will be recruited through the Muscular Dystrophy (MDA) clinic at Northwestern 
Memorial Hospital. The MDA clinic is located in the Lavin Family Pavilion, 259 E. Erie St., Suite 
1900, Chicago, IL 60611 and is held on Wednesday mornings from 08:00-12:00.   
 
Participants will be selected by the neurologists in the MDA clinic, primarily the Principal 
Investigator. They will be selected for screening based on their clinical exam, medical history, 
and willingness to participate in the study. The Principal Investigator will review the patient’s 
medical chart to determine pre-eligibility. A member of the study team will conduct the informed 
consent process once pre-eligibility is confirmed by the Principal Investigator. 
 
No external methods will be used for recruitment purposes. 
 
COMPENSATION FOR PARTICIPATION IN RESEARCH ACTIVITIES 
Subjects will not be compensated for participating in this study. Furthermore, subjects will not be 
reimbursed for expenses related to travel, lodging, or accommodation.  
 
If patients become ill or are injured as a result of this study (medications, devices or 
procedures), they should seek medical treatment through their doctor or treatment center of 
choice. The subject should promptly tell his/her study doctor about any illness or injury.   
 
The hospital [university, researchers] will not pay for medical care required because of a bad 
outcome resulting from your participation in this research study. This does not keep you from 
seeking to be paid back for care required because of a bad outcome. 
 


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WITHDRAWAL OF PARTICIPANTS 
Subjects may be withdrawn from the study if the Principal Investigator believes that participating 
places them at risk of harm. Subjects may also be withdrawn if it is found that they are not 
complying with the study’s required dosing instructions. 
 
In the event that a subject is to be withdrawn, the Principal Investigator will inform the subject 
regarding the reason for withdrawal. The Principal Investigator will make recommendations for 
seeking additional testing and prompt medical care, if necessary. A member of the study team 
will indicate that the subject was withdrawn in the subject binder. 
 
RISKS TO PARTICIPANTS 
Participating in this study places subjects at minimal risk of the following: 
 
Risks from glucocorticoid steroids 
• 
The risks associated with glucocorticoid steroids include: glaucoma (elevated pressure in 
the eyes), edema (fluid retention in lower legs), hypertension (high blood pressure), weight 
gain, problems with mood and memory, alterations in endocrine function, increased risks 
related to infections, decrease in bone density, and possible damage to your vision.  These 
risks are seen when people take glucocorticoid steroids daily for extended periods of time.  
Based on preclinical studies in animals and limited studies in humans, it is expected these 
risks are much less when taking glucocorticoids steroids once per week.  
 
Since the subjects will be taking glucocorticoid steroids once weekly, for 6 months, their risk of 
side effects is low, with short duration, and reversible. 
 
Risk from blood draws 
The risks of taking blood include pain, a bruise at the point where the blood is taken, redness 
and swelling of the vein and infection, and a rare risk of fainting. 
 
Risk from MRI 
Some people cannot have an MRI because they have certain types of metal in their body.  For 
instance, if you have a heart pacemaker, artificial heart valves, metal implants such as metal ear 
implants, bullet pieces, chemotherapy or insulin pumps or any other metal such as metal clips or 
rings, you cannot have an MRI.  During this test, subjects will lie in a small closed area inside a 
large magnetic tube. Some people are scared or anxious in small places (claustrophobic).  The 
MRI scanner makes loud banging noises while taking a measurement, so either ear plugs or 
specially designed headphones will be used to reduce the noise. 
 
Risks from DEXA bone scans 
Procedures such as CT scans, X-rays and/or radioactive drugs will be used during this research 
study to see how subjects are doing. The cumulative radiation exposure from these tests is 
considered small and is not likely to adversely affect subjects or their disease. However, the 
effects of radiation add up over a lifetime.  It is possible that having several of these tests may 
add to subjects’ risk of injury or disease. When deciding to enter this study, subjects will think 
about their past and future contact with radiation. Examples of contact with radiation include x-
rays taken for any reason or radiation therapy for cancer treatment. 
 
Background radiation is present on Earth (soil, air, water, food) and also includes cosmic 
radiation. Exposure to radiation from natural sources is a feature of everyday life. Your 


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participation in this study will involve additional exposure to radiation. The radiation dose 
received for one DEXA scan is approximately equal to 4.5 days of background radiation. 
 
The effective dose for one DEXA scan (lumbar spine and total hip) is 4 mrem. The total effective 
dose of all research related radiation procedures is 8 mrem.  
 
POTENTIAL BENEFITS TO PARTICIPANTS 
There may be benefit from participating in this research.  Potential benefit may include 
increased strength and muscle mass.   
 
DATA MANAGEMENT AND CONFIDENTIALITY 
This is a pilot study to determine safety and tolerability of the intervention.  All subjects will 
receive once weekly prednisone, and no placebo arm is planned for this study.  Data will be 
compared between study entry and 6 months of once weekly steroids.  The data from this study 
will be used to plan for a larger trial.  In preclinical animal models, a 25% improvement in 
muscle strength and function was observed.   
 
During the trial, study team members with access to Northwestern Memorial Hospital’s 
electronic medical records System, Epic, will have access to the subject’s medical chart. The 
study team will print, redact, and store all study-specific test results in the subject binders. All 
medical records will be de-identified and labeled with the subject identification number. The 
tests obtained from Epic include: genetic test results, medical history, concomitant medications, 
safety laboratory results, EKG results, Echo results, DEXA results, and MRI results. The 
remaining tests will be performed during the study visit and documented on case report forms 
(source documentation) located in the subject binders. 
 
MRI images that are collected as part of this research study will be obtained at the Center for 
Translational Imaging and Northwestern University. The study team will obtain a de-identified 
copy of the MRI images for all study participants that contains the participant’s study number. A 
member of the study team will transmit the images to the University of Florida through a secure 
file transfer (SFT) system. Copies of the images will also be secured by the study team in the 
subject binders. 
 
The Principal Investigator and study team will have access to the data and be responsible for 
data transmission and storage. 
 
The study binders will be secured in Abbott Hall 1109, 710 N. Lake Shore Dr., Chicago, IL 
60611 and, upon completion of the trial, O’Hare Storage Facility. Study data will be stored for a 
period no longer than 5 years. After this period, unless otherwise notified by the study team, all 
study records will be shredded or disposed through HIPAA-compliant document bins. 
 
 
PROVISIONS TO PROTECT THE PRIVACY INTERESTS OF PARTICIPANTS 
The Principal Investigator and study team will make every effort to ensure that the subjects’ 
privacy is protected. As mentioned above, subjects will only share personal information with the 
Principal Investigator and members of the study team. The study team will be permitted to 
access sources of information about the participant through the consent.  During the consent 
process, and throughout the study, subjects will be given the opportunity to ask questions about 
the study and voice their concerns. Contact information will be provided to the subjects in the 
consent. 


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COMPENSATION FOR RESEARCH-RELATED INJURY 
Subjects will not be compensated for research-related injury. 
 
ECONOMIC BURDEN TO PARTICIPANTS 
This study does not pose a large economic burden to participants because they will not have to 
pay for the study treatment and study procedures. Furthermore, subjects will be seen during 
their routine, standard-of-care visits to minimize additional costs of travel. Patients will not be 
seen until an EKG and ECHO are collected as part of their standard of care to prevent these 
costs being transferred to the patient. 
  
CONSENT PROCESS 
The consent process will take place in the MDA clinic at Northwestern Memorial Hospital, 259 
E. Erie St., 1900, Chicago, IL 60611. Subjects will be given time to read the informed consent 
form, ask questions, and have them answered satisfactorily (sufficient time to consult with family 
members and outside physician will be granted). A member of the study team will perform the 
consent process as follows: 
• 
Read the Informed Consent Guidelines 
• 
Ensures that consent form given to subject is the current version with approval stamp 
• 
Administers the informed consent form explaining the following: 
o Anticipated benefits of the trial  
o Attendant discomforts and risks  
o Appropriate alternative procedures that might be advantageous for the subject  
o The extent to which confidentiality of records identifying the participant will be 
maintained  
o Compensation 
o Contacts for questions or in case of injury 
o Voluntary participation 
o Summary of the study including interventions and assessment visits 
o Specific assessments scheduled for the current visit  
• 
Optional elements 
o Changes to protocol (and consent document) since last visit 
• 
Allows time for Subject to read the consent 
• 
Asks if Subject has any questions, answers adequately 
• 
Collects completed consent from Subject 
• 
Does not begin visit until consent is obtained from Subject and photocopy returned to 
Subject 
• 
Files original copy of consent in Subject’s personal information file. 
 
The Consent Process will take place in the Neurology Clinic.  
Will be following “SOP: Informed Consent Process for Research (HRP-090)