Smart AFO and 5-Azacitidine to Enhance Mobility in Children With Cerebral Palsy

NCT07434492 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STU00225131
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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

Keywords

Pediatric

Outcome Measures

Primary Outcomes (1)

• 6 Minute Walking Test

  Participants will complete a 6 minute walking test to measure the total distance they can walk in a 6 minute period.

  Baseline to Follow-Up, approximately 13 weeks

Secondary Outcomes (8)

• 10 Meter Walking Test

  Baseline to Follow-Up, approximately 13 weeks

• Gross Motor Function Measure 88 (GMFM-88)

  Baseline to Follow-Up, approximately 13 weeks

• Selective Control Assessment of the Lower Extremity (SCALE)

  Baseline to Post-Intervention, approximately 7 weeks

• Pediatric Berg Balance Scale (BBS)

  Baseline to Follow-Up, approximately 13 weeks

• Modified Tardieu Scale (MTS)

  Baseline to Follow-Up, approximately 13 weeks

Study Arms (3)

Smart AFO & Home Intervention Training + 5-Azacitidine

EXPERIMENTAL

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.

Device: Smart Ankle-Foot Orthosis; Drug: 5-Azacitidine

Smart AFO & Home Intervention Training + Mannitol (Placebo)

ACTIVE COMPARATOR

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.

Device: Smart Ankle-Foot Orthosis; Drug: Placebo

Smart AFO & Home Intervention Training

ACTIVE COMPARATOR

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.

Device: Smart Ankle-Foot Orthosis

Interventions

Smart Ankle-Foot Orthosis DEVICE

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.

Also known as: Biomotum Ambulo AFO

Smart AFO & Home Intervention Training; Smart AFO & Home Intervention Training + 5-Azacitidine; Smart AFO & Home Intervention Training + Mannitol (Placebo)

5-Azacitidine DRUG

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.

Also known as: VIDAZA

Smart AFO & Home Intervention Training + 5-Azacitidine

Placebo DRUG

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.

Also known as: Mannitol

Smart AFO & Home Intervention Training + Mannitol (Placebo)

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Shirley Ryan AbilityLab: lead

Study Sites (1)

Shirley Ryan AbilityLab

Chicago, Illinois, 60611, United States

Location

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

• Megan O'Brien, 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: Participants who opt in at consent to receive the injection of 5-azacitidine or placebo will be blinded to their randomization. Caregivers will also be blinded to their child's randomization. Randomization will take place prior to the midpoint assessment.
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Director, Technology & Innovation

Model Details: This is a parallel group study in which all participants will receive the primary intervention consisting of at home training three times per week using a smart AFO device. In addition to the primary intervention, participants have the option at consent to opt in to receive a single dose of 5-Azacitidine or placebo, and/or to complete two blood draws at pre-test and post-test.

Study Record Dates

• First Submitted: February 11, 2026
• First Posted: February 25, 2026
• Study Start: April 20, 2026
• Primary Completion (Estimated): August 31, 2030
• Study Completion (Estimated): August 31, 2030
• Last Updated: April 20, 2026

Record last verified: 2026-04

Locations

US (1)