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

Smart Ankle-Foot Orthosis and 5-Azacitidine to Enhance Gait and Community Mobility for Children With Cerebral Palsy

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.

Study Overview

• Status: Recruiting
• Conditions: Cerebral Palsy
• Intervention / Treatment: Device: Smart Ankle-Foot Orthosis; Drug: 5-Azacitidine; Drug: Placebo

Detailed Description

The goal of this project is to investigate the impact of the Biomotum Ambulo ankle-foot orthosis on gait outcomes in children with Cerebral Palsy. The device will be tested for its orthotic benefits when providing mechanical ankle assistance and its therapeutic benefits when providing progressive, resistance-based ankle exercise. Through a 1:1 randomization, a subset of opt-in participants will also receive a dose of 5-azacitidine (AZA), an FDA-approved drug for pediatric oncology, or a placebo. Research in muscle fibers has 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 smart AFO 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 investigate the potential genetic and epigenetic correlations with the severity and trajectory of musculoskeletal impairments and with responses to the smart AFO and AZA interventions.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Audrey Wiesner, BS; Phone Number: 3122388435; Email: awiesner@sralab.org
• Study Contact Backup: Name: Jacklyn Stoller, PT, DPT; Phone Number: 3122387620; Email: jstoller@sralab.org

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Shirley Ryan Abilitylab

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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: 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); Monitoring Heart Rate; 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; 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.; Other Names: Biomotum Ambulo AFO; Drug: 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.; Other Names: VIDAZA
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: 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); Monitoring Heart Rate; 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; 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.; Other Names: Biomotum Ambulo AFO; Drug: 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.; Other Names: Mannitol
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: 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); Monitoring Heart Rate; Caregiver filling out brief log to track training session details.	Device: 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.; Other Names: Biomotum Ambulo AFO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
10 Meter Walking Test	Participants will walk 10 meters to assess gait speed.	Baseline to Follow-Up, approximately 13 weeks
Gross Motor Function Measure 88 (GMFM-88)	GMFM-88 will be used to measure changes in gross motor function over time.	Baseline to Follow-Up, approximately 13 weeks
Selective Control Assessment of the Lower Extremity (SCALE)	The SCALE is a clinical assessment tool used to evaluate selective voluntary motor control of the lower limbs.	Baseline to Post-Intervention, approximately 7 weeks
Pediatric Berg Balance Scale (BBS)	The Pediatric BBS will be used to assess static balance and fall risk.	Baseline to Follow-Up, approximately 13 weeks
Modified Tardieu Scale (MTS)	Modified Tardieu Scale will be completed to assess the muscle's response to stretch at given velocities.	Baseline to Follow-Up, approximately 13 weeks
Wong-Baker FACES Pain Rating Scale	The Wong-Baker FACES Pain Rating Scale will be used for children to self-report their level of pain.	Baseline to Follow-Up, approximately 13 weeks
Pictorial Children's Effort Rating Table (PCERT)	The PCERT will be used for children to self-assess and communicate their perceived exertion during physical activity or exercise.	Baseline to Follow-Up, approximately 13 weeks
Cerebral Palsy Quality of Life Questionnaire (CP-QOL)	CP-QOL will be administered to capture the child's and family's perspective on well-being and quality of life.	Baseline to Follow-Up, approximately 13 weeks

Collaborators and Investigators

• Sponsor: Shirley Ryan AbilityLab
• Investigators: Principal Investigator: Megan O'Brien, PhD, Shirley Ryan Abilitylab

Study record dates

Study Major Dates

• Study Start (Actual): May 28, 2026
• Primary Completion (Estimated): August 31, 2030
• Study Completion (Estimated): August 31, 2030

Study Registration Dates

• First Submitted: February 11, 2026
• First Submitted That Met QC Criteria: February 20, 2026
• First Posted (Actual): February 25, 2026

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 28, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Carbohydrates; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Alcohols; Aza Compounds; Nucleosides; Ribonucleosides; Sugar Alcohols; Azacitidine; Mannitol
• Other Study ID Numbers: STU00225131

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No