Cerebellar Deep Brain Stimulation for Movement Disorders in Cerebral Palsy in Children and Young Adults

Dystonic Cerebral Palsy Clinical Trial

Official title:

Cerebellar Deep Brain Stimulation for Severe Combined Movement Disorders and Spasticity in Children and Young Adults With Cerebral Palsy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06122675
• Other study ID #: 22-37182
• Secondary ID: UH3NS128297-01A1
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 1, 2024
• Est. completion date: July 1, 2029

Study information

• Verified date: May 2024
• Source: University of California, San Francisco
• Contact: Marta San Luciano Palenzuela, MD, MS
• Phone: 4153532311
• Email: Marta.SanLucianoPalenzuela[@]ucsf.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: July 1, 2029
• Est. primary completion date: July 1, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 25 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cerebral Palsy
• Dystonic Cerebral Palsy
• Movement Disorders
• Paralysis

Intervention

Device:
• DBS
Implanted in the cerebellum.

Locations

Country	Name	City	State
United States	University of California, San Francisco	San Francisco	California

Sponsors (3)

Lead Sponsor	Collaborator
University of California, San Francisco	National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Average total (global index) Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18-R) scores	This is the primary motor outcome measure. It is the Movement Disorder-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18 R). The MD-CRS 4-18 R is a validated tool aimed to evaluate movement disorders in developmental age. It is particularly useful for rating the severity of movement disorders in dyskinetic cerebral palsy. It scored based on two parts. Part I: General Assessment has a minimum total score of 0 (no impairment) and a maximum total score of 60 (maximal impairment). Part II: MD Assessment has a minimum total score of 0 (movement disorder is absent) and a maximum total score of 28 (maximal movement disorder presence).	Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).
Primary	Average total standardized Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) scores	This is the primary Quality of Life (QOL) outcome measure. It is the Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) scale. The CPCHILD is a reliable and valid measure of caregivers' perspectives on health status, functional limitations, and well-being of patients with severe CP, including those individuals who are non-verbal and non-ambulatory. Because many children suffering with CP are severely affected in many areas of their lives including activities of daily living, communication, mobility and overall health, there is an imperative to evaluate new interventions, especially invasive and resource-intensive ones such as DBS, using outcomes that are more meaningful to patients and their caregivers. Scores for each domain and for the total survey are standardized and range from 0 (worst) to 100 (best).	Evaluated weekly during randomized blinded phase (total of 3 months in each condition, sham stimulation and effective stimulation).