Cerebral PalsyPhase 2NCT07434492
Smart AFO and 5-Azacitidine to Enhance Mobility in Children With Cerebral Palsy
A Phase 2 study testing Smart Ankle-Foot Orthosis and 5-Azacitidine 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 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. Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Smart Ankle-Foot Orthosis and 5-Azacitidine
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 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 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
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- 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
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
About this study
Official title: 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.
Read the 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.
- Conditions
- Cerebral Palsy
- Being tested
- Smart Ankle-Foot Orthosis, 5-Azacitidine, Placebo
- Keywords
- Pediatric
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Smart Ankle-Foot OrthosisDevice
Also called Biomotum Ambulo AFO
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-AzacitidineDrug
Also called VIDAZA
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.
PlaceboDrug
Also called Mannitol
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Device: Smart Ankle-Foot Orthosis |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Follow-Up, approximately 13 weeks
Secondary outcomes (8)
10 Meter Walking Test
Participants will walk 10 meters to assess gait speed.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to Follow-Up, approximately 13 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 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.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shirley Ryan AbilityLab
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Megan O'Brien, Shirley Ryan AbilityLab
- Investigators
- Megan O'Brien, PhD, Principal investigator, Shirley Ryan AbilityLab
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07434492
- Sponsor's protocol ID
- STU00225131
- Status last verified
- May 2026
- First submitted
- 11 Feb 2026
- Last updated
- 1 Jun 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 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Smart Ankle-Foot Orthosis and 5-Azacitidine to Enhance Gait and Community Mobility for Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT07434492
- Acronym / sponsor ID
- STU00225131
- Conditions
- Cerebral Palsy
- Keywords
- Pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 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 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.
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 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 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.
| Arm | Type | Receives |
|---|---|---|
| Smart AFO & Home Intervention Training + 5-Azacitidine | Experimental | Device: Smart Ankle-Foot Orthosis, Drug: 5-AzacitidineParticipants 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. |
| Smart AFO & Home Intervention Training + Mannitol (Placebo) | Active comparator | Device: Smart Ankle-Foot Orthosis, Drug: PlaceboParticipants 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. |
| Smart AFO & Home Intervention Training | Active comparator | Device: Smart Ankle-Foot OrthosisParticipants 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. |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (11 listed)
- 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
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 |
|---|---|---|
| Smart Ankle-Foot OrthosisAlso: Biomotum Ambulo AFOThe 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. | Device | Smart AFO & Home Intervention Training, Smart AFO & Home Intervention Training + 5-Azacitidine, Smart AFO & Home Intervention Training + Mannitol (Placebo) |
| 5-AzacitidineAlso: VIDAZAA 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. | Drug | Smart AFO & Home Intervention Training + 5-Azacitidine |
| PlaceboAlso: MannitolA 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. | Drug | Smart AFO & Home Intervention Training + Mannitol (Placebo) |
Primary outcome
6 Minute Walking Test
Time frame: Baseline to Follow-Up, approximately 13 weeks
Participants will complete a 6 minute walking test to measure the total distance they can walk in a 6 minute period.
8 secondary outcomes
10 Meter Walking Test
Time frame: Baseline to Follow-Up, approximately 13 weeks
Gross Motor Function Measure 88 (GMFM-88)
Time frame: Baseline to Follow-Up, approximately 13 weeks
Selective Control Assessment of the Lower Extremity (SCALE)
Time frame: Baseline to Post-Intervention, approximately 7 weeks
Pediatric Berg Balance Scale (BBS)
Time frame: Baseline to Follow-Up, approximately 13 weeks
Modified Tardieu Scale (MTS)
Time frame: Baseline to Follow-Up, approximately 13 weeks
Wong-Baker FACES Pain Rating Scale
Time frame: Baseline to Follow-Up, approximately 13 weeks
Pictorial Children's Effort Rating Table (PCERT)
Time frame: Baseline to Follow-Up, approximately 13 weeks
Cerebral Palsy Quality of Life Questionnaire (CP-QOL)
Time frame: Baseline to Follow-Up, approximately 13 weeks
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 1 Jun 2026.
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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.
Megan O'Brien, 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 Smart Ankle-Foot Orthosis and 5-Azacitidine 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 11 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 1 Jun 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?
6 Minute Walking Test (time frame: Baseline to Follow-Up, approximately 13 weeks). 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 1 Jun 2026.