NCT07433023

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

Advancing Neurorehabilitation: Evaluating Transcutaneous Spinal Stimulation and 5-Azacitidine for Enhanced Motor Outcomes in Cerebral Palsy

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: Shirley Ryan AbilityLab · Academic / Other
• Sex: All
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This project will investigate the impact of transcutaneous spinal cord stimulation (tSCS) on gait and activity-based training in children with Cerebral Palsy. Through a 1:1 randomization, a subset of participants will also receive a dose of 5-azacitidine (AZA), an FDA-approved drug for pediatric oncology, or a placebo. Research in muscle fibers as shown that AZA aids in muscle growth and regeneration. The use of AZA in this study is an off-label application, employed together with the tSCS intervention to investigate the impact of targeting both muscular and mechanical deficits related to CP. This project will also collect blood samples of participants to perform genetic and epigenetic profiling in order to test for correlation with the severity and trajectory of musculoskeletal impairments and with responses to the tSCS and AZA interventions. The study team hypothesizes that personalized tSCS combined with targeted gait or activity based training will improve motor outcomes in children with spastic CP by reducing muscle tone, improving gait symmetry and neuromotor function, and increasing functional mobility. Furthermore, the addition of a repurposed anabolic agent (AZA) will augment these gains compared to tSCS and training alone. Children with CP across GMFCS levels will participate in a three-phase protocol, including: (1) a two week baseline phase, (2) a sixteen week intervention phase during which participants will receive tSCS and training interventions, and (3) a four-week post-intervention phase to examine long term outcomes. At the midpoint, participants will receive a single dose of AZA or a single dose of a placebo through a 1:1 randomization. Participants will have a blood draw in phase 1 for genetic and pre-intervention epigenetic profiling and will have a second blood draw in phase 3 for post-intervention epigenetic profiling.

Conditions

• Cerebral Palsy

Interventions

Type	Name	Description
OTHER	tSCS	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.
DRUG	5-Azacitidine	A subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m²
DRUG	Mannitol	A subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m².
OTHER	Functional Activity Training	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.

Timeline

• Start date: 2026-03-01
• Primary completion: 2029-01-01
• Completion: 2029-01-01
• First posted: 2026-02-25
• Last updated: 2026-02-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study