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

NCT07433023 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STU00225218
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Cerebral Palsy

Keywords

Pediatric; Transcutaneous Spinal Stimulation

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline to Follow-Up, approximately 22 weeks

Study Arms (3)

tSCS + Functional Activity Training

ACTIVE COMPARATOR

Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate.

Other: tSCS; Other: Functional Activity Training

tSCS + Functional Activity Training + 5-Azacitidine

EXPERIMENTAL

Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate. A subset of opt-in participants will also be randomized to receive either 5-Azacitidine (VIDAZA) or placebo (Mannitol) at midpoint. A registered nurse will deliver a single subcutaneous injection of either AZA or placebo at a dose of 75 mg/m² to the participants lower abdomen or medial thigh.

Other: tSCS; Drug: 5-Azacitidine; Other: Functional Activity Training

tSCS + Functional Activity Training + Mannitol (Placebo)

ACTIVE COMPARATOR

Transcutaneous spinal cord stimulation will be paired with training based on Aim. Participants in Aim 1(GMFCS levels 1-3) will engage in three sessions a week for 16 weeks of treadmill and overground gait training using tSCS. Participants in Aim 2(GMFCS levels 4-5) will engage in three sessions a week for 16 weeks of functional activity based training as appropriate. A subset of opt-in participants will also be randomized to receive either 5-Azacitidine (VIDAZA) or placebo (Mannitol) at midpoint. A registered nurse will deliver a single subcutaneous injection of either AZA or placebo at a dose of 75 mg/m² to the participants lower abdomen or medial thigh.

Other: tSCS; Drug: Mannitol; Other: Functional Activity Training

Interventions

tSCS OTHER

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.

Also known as: Transcutaneous Spinal Stimulation

tSCS + Functional Activity Training; tSCS + Functional Activity Training + 5-Azacitidine; tSCS + Functional Activity Training + Mannitol (Placebo)

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

Also known as: VIDAZA

tSCS + Functional Activity Training + 5-Azacitidine

Mannitol DRUG

A subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m².

Also known as: Placebo

tSCS + Functional Activity Training + Mannitol (Placebo)

Functional Activity Training OTHER

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.

tSCS + Functional Activity Training; tSCS + Functional Activity Training + 5-Azacitidine; tSCS + Functional Activity Training + Mannitol (Placebo)

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Adequate caregiver support to be able to participate in training and assessment sessions for the duration of the study, at the discretion of the Investigator.
• Capable of performing simple cued motor tasks and can follow 2-3 step commands.
• Capable of communicating an accurate yes or no answer to questions according to parent or guardian.
• Able to localize pain/ discomfort.
• Physician approval for participation.
• Parent/ guardian permission.

You may not qualify if:

• 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
• Implanted stimulator (e.g., epidural stimulator, vagus nerve stimulator, pacemaker, cochlear implant) or drug delivery device (e.g., baclofen pump)
• Dependence on an electro-magnetic medical implant (e.g., cardiac pacemaker, implanted drug pump), ventilation support, or another external device.
• History of uncontrolled seizures
• Unexplained presence of persistent complaints of pain of any kind
• Unable to localize pain/discomfort
• Severe cortico-visual impairment
• Active pressure sores
• Active urinary tract infection
• Active cancer or cancer in remission less than 5 years
• Clinically significant depression, psychiatric disorders, or ongoing drug abuse
• Immunodeficiency or hematologic condition

Sponsors & Collaborators

• Shirley Ryan AbilityLab: lead

Study Sites (1)

Shirley Ryan AbilityLab

Chicago, Illinois, 60611, United States

RECRUITING

MeSH Terms

Conditions

Cerebral Palsy

Interventions

Azacitidine; Mannitol

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Sugar Alcohols; Alcohols; Carbohydrates

Study Officials

• Arun Jayaraman, PhD

  Shirley Ryan AbilityLab

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Audrey Wiesner, BS

CONTACT

3122388435; awiesner@sralab.org

Jacklyn Stoller, PT, DPT

CONTACT

3122387620; jstoller@sralab.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Masking Details: If a participant chooses to opt-in at consent to receive the injection, participants and clinicians will be blinded to their randomization. Caregivers will also be blinded to their child's randomization. Randomization will occur prior to the midpoint assessment.
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Scientific Chair, Technology & Innovation

Model Details: This is a parallel group study design in which all participants will engage in the primary intervention of training combined with tSCS. Participants will also have the option to opt-in at consent to be randomized to receive either an injection of 5-Azacitidine or placebo.

Study Record Dates

• First Submitted: February 5, 2026
• First Posted: February 25, 2026
• Study Start: May 28, 2026
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2029
• Last Updated: May 29, 2026

Record last verified: 2026-05

Locations

US (1)