Deze pagina is automatisch vertaald en de nauwkeurigheid van de vertaling kan niet worden gegarandeerd. Raadpleeg de Engelse versie voor een brontekst.

tSCS en 5-Azacitidine voor Verbeterde Motorische Uitkomsten bij Cerebrale Parese

3 november 2026 bijgewerkt door: Arun Jayaraman, PT, PhD, Shirley Ryan AbilityLab

Vooruitgang in Neurorevalidatie: Evaluatie van Transcutane Spinale Stimulatie en 5-Azacitidine voor Verbeterde Motorische Uitkomsten bij Cerebrale Parese

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.

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

80

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

  • Verenigde Staten
    • Illinois
      • Chicago, Illinois, Verenigde Staten, 60611
        • Werving
        • Shirley Ryan AbilityLab
        • Hoofdonderzoeker:
          • Arun Jayaraman, PhD

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

4 jaar tot 17 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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 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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Ander
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verdrievoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Actieve vergelijker: tSCS + Functional Activity Training

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.
Ander: 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.

Andere namen:
  • Transcutane spinale stimulatie
Ander: 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.
Experimenteel: tSCS + Functional Activity Training + 5-Azacitidine

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.
Ander: 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.

Andere namen:
  • Transcutane spinale stimulatie
Ander: 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.

Geneesmiddel: 5-Azacitidine

A subset of opt-in participants will receive a single subcutaneous injection of 5-Azacitidine at a dose of 75 mg/m²

Andere namen:
  • VIDAZA
Actieve vergelijker: tSCS + Functional Activity Training + Mannitol (Placebo)

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.
Ander: 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.

Andere namen:
  • Transcutane spinale stimulatie
Ander: 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.

Geneesmiddel: Mannitol

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

Andere namen:
  • Placebo

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Spatiotemporal Gait Symmetry via Instrumented Gait-Mat
Tijdsspanne: 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.
Baseline to Follow-Up, approximately 22 weeks
Modified Tardieu Scale
Tijdsspanne: 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.
Baseline to Follow-Up, approximately 22 weeks

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Shirley Ryan AbilityLab

Onderzoekers

  • Hoofdonderzoeker: Arun Jayaraman, PhD, Shirley Ryan AbilityLab

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

28 mei 2026

Primaire voltooiing (Geschat)

31 augustus 2030

Studie voltooiing (Geschat)

31 augustus 2030

Studieregistratiedata

Eerst ingediend

5 februari 2026

Eerst ingediend dat voldeed aan de QC-criteria

18 februari 2026

Eerst geplaatst (Werkelijk)

25 februari 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

5 november 2026

Laatste update ingediend die voldeed aan QC-criteria

3 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • STU00225218

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

product vervaardigd in en geëxporteerd uit de V.S.

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

Abonneren