Recruiting
Phase 2

Smart AFO & 5-Azacitidine

Sponsor:

Shirley Ryan AbilityLab

Code:

NCT07434492

Conditions

Cerebral Palsy

Eligibility Criteria

Sex: All

Age: 8 - 17

Healthy Volunteers: Not accepted

Interventions

Smart Ankle-Foot Orthosis

5-Azacitidine

Placebo

Study Details

Brief summary:

This is an intervention study to investigate the impact of a smart ankle-foot orthosis (AFO) on mobility outcomes in children with Cerebral Palsy. Participants will complete a three phase protocol including 6-weeks of at home training with the smart AFO. Participants will also have the option to participate in a randomized control trial to investigate the impact of 5-Azacitidine combined with the smart AFO to further effect mobility outcomes.

Conditions

Cerebral Palsy

Study ID

NCT07434492

Start date

May 28, 2026

Status verified date

May, 2026

Completion date

Aug 31, 2030

Anticipated

Primary completion date

Aug 31, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 8 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosis of cerebral palsy classified as Gross Motor Function Classification System (GMFCS) levels I to III
  • Between 8 and 17 years and 8 months of age at the time of enrollment/consent. Participants that will turn 18 during the course of the study will not be included as pausing the at-home protocol until re-consenting is possible could interfere with intervention results.
  • 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 PI.
  • Height/weight/BMI between the 5th - 95th percentile of children with cerebral palsy
  • Able to walk for at least 6 minutes (assisted or unassisted)
  • Able to understand and follow simple directions
  • Able to safely fit into a device configuration and tolerate assistance without knee hyperextension while walking
  • At least 20 degrees of passive ankle plantar flexion range of motion
  • Physician approval for participation

Exclusion Criteria:

  • Knee extension or ankle dorsiflexion contractures greater than 15 degrees
  • 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 5
  • Pregnancy
  • Orthopedic surgery on the lower limbs completed in the prior 12 months
  • New implanted device (e.g., baclofen pump) and/or active intrathecal medication titration that may affect muscle spasticity
  • Botulinum toxin treatment within 3 months preceding enrollment unless given approval by the study physician
  • Severe osteoporosis unless given approval by the study 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 PI or study physician

Study Design

Enrollment

60 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Other

Interventions and Outcome Measures

Arms

experimental: Smart AFO & Home Intervention Training + 5-Azacitidine

Participants will receive one Smart AFO device for use during the at-home intervention phase of the study. Participants will be asked to complete and log at least three 30-minute gait training sessions per week at home with the device.

Each session will involve:

1. Walking for 5-10 minutes intervals for 3 trials, with the goal of performing at a 7/10 on the Pictorial Children's Effort Rating Table (PCERT)
2. Monitoring Heart Rate
3. Caregiver filling out brief log to track training session details.

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.

active comparator: Smart AFO & Home Intervention Training + Mannitol (Placebo)

Participants will receive one Smart AFO device for use during the at-home intervention phase of the study. Participants will be asked to complete and log at least three 30-minute gait training sessions per week at home with the device.

Each session will involve:

1. Walking for 5-10 minutes intervals for 3 trials, with the goal of performing at a 7/10 on the Pictorial Children's Effort Rating Table (PCERT)
2. Monitoring Heart Rate
3. Caregiver filling out brief log to track training session details.

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.

active comparator: Smart AFO & Home Intervention Training

Participants will receive one Smart AFO device for use during the at-home intervention phase of the study. Participants will be asked to complete and log at least three 30-minute gait training sessions per week at home with the device.

Each session will involve:

1. Walking for 5-10 minutes intervals for 3 trials, with the goal of performing at a 7/10 on the Pictorial Children's Effort Rating Table (PCERT)
2. Monitoring Heart Rate
3. Caregiver filling out brief log to track training session details.

Interventions

Smart Ankle-Foot Orthosis

The Biomotum Ambulo is an adjustable smart AFO device featuring two adjustable spring settings and provides real-time biofeedback using footplate sensors and progressive goal parameters based on individual gait.

5-Azacitidine

A subset of opt-in participants will receive a single dose (75mg/m²) of 5-Azacitidine (AZA), an FDA-approved drug for pediatric oncology. A registered nurse will deliver a single subcutaneous injection at midpoint.

Placebo

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

Primary outcome measure

  • 6 Minute Walking Test [ Time Frame: Baseline to Follow-Up, approximately 13 weeks ]

Central Contacts and Locations

Central contacts

Audrey Wiesner, BS

3122388435awiesner@sralab.org

Jacklyn Stoller, PT, DPT

3122387620jstoller@sralab.org

Locations

Shirley Ryan AbilityLab

Recruiting

Chicago, Illinois, United States, 60611

More Information

Sponsor

Shirley Ryan AbilityLab

Last update posted

Jun 1, 2026

Last verified

May, 2026

Keywords

  • Pediatric

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Shirley Ryan AbilityLab on 2026-06-01.