Clinical trial NCT06122675: Cerebellar Deep Brain Stimulation for Movement Disorders in Cerebral Palsy in Children and Young Adults
Official title: Cerebellar Deep Brain Stimulation for Severe Combined Movement Disorders and Spasticity in Children and Young Adults with Cerebral Palsy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Dyskinetic Cerebral Palsy
Dystonic Cerebral Palsy
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
Collaborators: name: National Institutes of Health (NIH)
name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2023-11-08
Enrollment: 10
Sex: ALL
Minimum age (years): 7
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: The differences in primary outcome scores are calculated while on intervention (effective stimulation) versus placebo (sham stimulation), using an N-of-1 trial design. Each N-of-1 trial consists of a baseline assessment prior to DBS surgery (4 weeks), DBS surgery and open label phase to determine optimal stimulation settings (20 weeks), followed by a randomized three-cycle sequence of paired 8-week exposure periods, with each pair including intervention then placebo or vice versa (24 weeks). The DBS intervention exposure will be at the personalized optimal stimulation settings determined during the open-label phase.
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to test the safety of placing Deep Brain Stimulators (DBS) in a part of the brain called the cerebellum and using electrical stimulation of that part of the brain to treat movement symptoms related to cerebral palsy. Ten children and young adults with dyskinetic cerebral palsy will be implanted with a Medtronic Percept Primary Cell Neurostimulator. We will pilot videotaped automated movement recognition techniques and formal gait analysis, as well as collect and characterize each subject's physiological and neuroimaging markers that may predict hyperkinetic pathological states and their response to therapeutic DBS.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of DCP (dystonic and/or choreoathetotic cerebral palsy) with or without comorbid spasticity, with a clear history of hypoxic ischemic brain injury preceding motor symptoms made by a pediatric neurologist, with supporting MRI findings.
* Age 7-25 at the time of surgery.
* Gross Motor Function Classification System (GMFCS) Levels II-V.
* History of appropriate therapy with oral medications with inadequate relief as determined by a movement disorders or pediatric neurologist. Prior history of selective dorsal rhizotomy is allowed.
* Patient and family have requested surgical intervention with DBS for their movement disorder.
* No gross cerebellar abnormalities observed and reported on structural MRI.
* Written informed consent and written/verbal assent for those younger than 18 years of age.
* Ability to comply with study follow-up visits for brain recordings, neuroimaging and testing of sham and effective stimulation and clinical assessments.

Exclusion Criteria:

* Coagulopathy, uncontrolled epilepsy, severe cardiopulmonary or gastrointestinal conditions, or other medical conditions considered to place the patient at elevated risk for surgical complications.
* Pregnancy: all women of child-bearing potential will be required to have a negative urine pregnancy test prior to undergoing their surgical procedure.
* Exclusion of genetic mimics of cerebral palsy: exclusion of conditions that manifest with a clinical syndrome similar to CP, in the absence of documented risk factors or neuroimaging findings consistent with a history of brain injury or congenital cerebral malformation. Work up may include comparative genomic hybridization (CGH) microarray and multi-gene panel and/or whole genome or whole exome sequencing.)
* Severe fixed contractions and skeletal deformities that would preclude determination of improvement.
* Traumatic brain injury (i.e., non-accidental trauma) or history of infectious or autoimmune encephalitis.
* Requirement of diathermy, electroconvulsive therapy or transcranial magnetic stimulation.

Primary outcomes:
measure: Average total (global index) Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18-R) scores
timeFrame: Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).
measure: Average total standardized Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) scores
timeFrame: Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
Study officials:
affiliation: University of California, San Francisco
name: Marta San Luciano Palenzuela, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: