tSCS and 5-Azacitidine for Enhanced Motor Outcomes in Cerebral Palsy

ConditionCerebral Palsy

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age4-17

SponsorShirley Ryan AbilityLab

About this trial

This is an intervention study to investigate the impact of spinal stimulation on mobility outcomes in children with Cerebral Palsy. Participants will complete a 16-week training program with weekly sessions of spinal stimulation and walking or activity-based training. Participants will also have the option to participate in a randomized control trial to investigate the impact of 5-Azacitidine combined with the spinal stimulation to further affect mobility outcomes.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of cerebral palsy (CP) classified as Gross Motor Function Classification System (GMFCS) Levels I-V.

Between 4 and 17 years old at the time of enrollment/consent.

Diagnosis of spastic CP hemiplegia, diplegia, or quadriplegia.

Stable medical condition as determined by the investigator.

Disqualifiers

Concurrent neurological disease affecting the central nervous system.

Cardiovascular or musculoskeletal disease or injury that would prevent full participation in physical therapy intervention

Orthopedic dysfunction, injury, or surgery that would impact an individual's ability to use the upper and/or lower extremity

Unhealed fracture or other musculoskeletal impairment that might interfere with upper or lower extremity rehabilitation or testing activities

Trial design

Design model

Parallel

Treatments tested in this trial

tSCS
Other intervention
Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation and activity based training. Stimulation intensity will be based on involuntary motor threshold, determined during a MEPs assessment.
5-Azacitidine
Drug
A subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m²
Mannitol
Drug
A subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m².
Functional Activity Training
Other intervention
All participants will engage in functional activity training based on Aim. Participants will participate in three 45 minute sessions per week for a total of 16 weeks involving transcutaneous spinal stimulation (tSCS) and activity based training. Participants in Aim 1 will complete gait training. Participants in Aim 2 will complete activity-based training.

Treatment groups

80 Participants

are divided into 3 treatment groups

Group A: tSCS + Functional Activity TrainingActive comparator 2 interventions

Group B: tSCS + Functional Activity Training + 5-AzacitidineExperimental treatment 3 interventions

Group C: tSCS + Functional Activity Training + Mannitol (Placebo)Active comparator 3 interventions

Trial outcomes

Primary outcomes

Spatiotemporal Gait Symmetry via Instrumented Gait-Mat

For Aim 1 participants (GMFCS levels 1-3), an instrumented gait-mat will be used to assess spatiotemporal gait symmetry.

Time frame

Baseline to Follow-Up, approximately 22 weeks

Modified Tardieu Scale

For Aim 2 participants (GMFCS levels 4-5), muscle spasticity will be assessed using the Modified Tardieu Scale.

Time frame

Baseline to Follow-Up, approximately 22 weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Shirley Ryan AbilityLab

Lead sponsor

Source ClinicalTrials.gov

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