Cerebral PalsyPhase 2NCT07433023
tSCS and 5-Azacitidine for Enhanced Motor Outcomes in Cerebral Palsy
A Phase 2 study testing tSCS, 5-Azacitidine and Mannitol in people with Cerebral Palsy, run by Shirley Ryan AbilityLab at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 17 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- tSCS, 5-Azacitidine and Mannitol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteUnited States
- Study dates
- 28 May 2026to 31 Aug 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Show all 10Show fewer
- 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.
Who cannot join
- 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.
Show all 22Show fewer
- 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 cancer or cancer in remission less than 5 years
- Clinically significant depression, psychiatric disorders, or ongoing drug abuse
- Immunodeficiency or hematologic condition
- Allergy to AZA or mannitol
- Plans to receive a vaccine within 30 days of the planned AZA or placebo injection to take place at week 10
- Pregnancy
- Orthopedic surgery completed in the prior 12 months
- Intrathecal medication titration that may affect muscle spasticity
- Botulinum toxin treatment within 3 months preceding enrollment unless given approval by the treating physician
- Current enrollment in a conflicting research study
- Any other health condition or diagnosis not listed above that may put the participant at risk as determined by the investigator or treating physician
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Shirley Ryan AbilityLab
Chicago, Illinois, 60611
Arun Jayaraman, PhD
About this study
Official title: Advancing Neurorehabilitation: Evaluating Transcutaneous Spinal Stimulation and 5-Azacitidine for Enhanced Motor Outcomes in Cerebral Palsy
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.
Read the 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
- Being tested
- tSCS, 5-Azacitidine, Mannitol, Functional Activity Training
- Keywords
- Pediatric, Transcutaneous Spinal Stimulation
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tSCSOther
Also called Transcutaneous Spinal Stimulation
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-AzacitidineDrug
Also called VIDAZA
A subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m²
MannitolDrug
Also called Placebo
A subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m².
Functional Activity TrainingOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Other: tSCS; Drug: Mannitol; Other: Functional Activity Training |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline to Follow-Up, approximately 22 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2026
- Study start28 May 2026
- Primary completion31 Aug 2030 (estimated)
- Study completion31 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Shirley Ryan AbilityLabThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shirley Ryan AbilityLab
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Arun Jayaraman, PT, PhD, Shirley Ryan AbilityLab
- Investigators
- Arun Jayaraman, PhD, Principal investigator, Shirley Ryan AbilityLab
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07433023
- Sponsor's protocol ID
- STU00225218
- Status last verified
- Nov 2026
- First submitted
- 5 Feb 2026
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Advancing Neurorehabilitation: Evaluating Transcutaneous Spinal Stimulation and 5-Azacitidine for Enhanced Motor Outcomes in Cerebral Palsy
- ClinicalTrials.gov ID
- NCT07433023
- Acronym / sponsor ID
- STU00225218
- Conditions
- Cerebral Palsy
- Keywords
- Pediatric, Transcutaneous Spinal Stimulation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Shirley Ryan AbilityLabUniversity, hospital or other
- Study start
- 28 May 2026 (actual)
- Primary completion
- 31 Aug 2030 (estimated)
- Study completion
- 31 Aug 2030 (estimated)
- First posted
- 25 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 3
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| tSCS + Functional Activity Training | Active comparator | Other: tSCS, Other: Functional Activity TrainingTranscutaneous 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. |
| tSCS + Functional Activity Training + 5-Azacitidine | Experimental | Other: tSCS, Drug: 5-Azacitidine, Other: Functional Activity TrainingTranscutaneous 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. |
| tSCS + Functional Activity Training + Mannitol (Placebo) | Active comparator | Other: tSCS, Drug: Mannitol, Other: Functional Activity TrainingTranscutaneous 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. |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (22 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tSCSAlso: Transcutaneous Spinal StimulationParticipants 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. | Other | tSCS + Functional Activity Training, tSCS + Functional Activity Training + 5-Azacitidine, tSCS + Functional Activity Training + Mannitol (Placebo) |
| 5-AzacitidineAlso: VIDAZAA subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m² | Drug | tSCS + Functional Activity Training + 5-Azacitidine |
| MannitolAlso: PlaceboA subset of opt-in participants will receive a single subcutaneous injection of Mannitol (placebo) at a dose of 75 mg/m². | Drug | tSCS + Functional Activity Training + Mannitol (Placebo) |
| Functional Activity TrainingAll 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. | Other | tSCS + Functional Activity Training, tSCS + Functional Activity Training + 5-Azacitidine, tSCS + Functional Activity Training + Mannitol (Placebo) |
Primary outcome
Spatiotemporal Gait Symmetry via Instrumented Gait-Mat
Time frame: Baseline to Follow-Up, approximately 22 weeks
For Aim 1 participants (GMFCS levels 1-3), an instrumented gait-mat will be used to assess spatiotemporal gait symmetry.
Modified Tardieu Scale
Time frame: Baseline to Follow-Up, approximately 22 weeks
For Aim 2 participants (GMFCS levels 4-5), muscle spasticity will be assessed using the Modified Tardieu Scale.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
Related trials for Cerebral Palsy
Not yet recruiting
Feasibility of a Robot-Guided Active Play Intervention for Children With Cerebral Palsy
Cerebral Palsy
Recruiting
Additional Effects on tDCS With AOT on Lower Limb Functions on Children With Diplegic CP
Diplegic Cerebral Palsy
Recruiting
Sensory Integration Therapy vs Conventional Physical Therapy in Children With Mixed Cerebral Palsy
Cerebral Palsy
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Arun Jayaraman, PhD principal investigator
Shirley Ryan AbilityLab
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing tSCS, 5-Azacitidine and Mannitol in people with Cerebral Palsy, run by Shirley Ryan AbilityLab at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Audrey Wiesner, BS (3122388435, awiesner@sralab.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Spatiotemporal Gait Symmetry via Instrumented Gait-Mat (time frame: Baseline to Follow-Up, approximately 22 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.