Smart AFO and 5-Azacitidine to Enhance Mobility in Children With Cerebral Palsy
ConditionCerebral Palsy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age8-17
SponsorShirley Ryan AbilityLab
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
Smart Ankle-Foot Orthosis
DeviceThe 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
DrugA 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
DrugA 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.
Treatment groups
60 Participants
are divided into 3 treatment groupsGroup A: Smart AFO & Home Intervention Training + 5-AzacitidineExperimental treatment 2 interventions
Group B: Smart AFO & Home Intervention Training + Mannitol (Placebo)Active comparator 2 interventions
Group C: Smart AFO & Home Intervention TrainingActive comparator 1 intervention
Trial outcomes
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.
Time frame
Baseline to Follow-Up, approximately 13 weeksSecondary outcomes
1
10 Meter Walking Test
Participants will walk 10 meters to assess gait speed.
Time frame
Baseline to Follow-Up, approximately 13 weeks2
Gross Motor Function Measure 88 (GMFM-88)
GMFM-88 will be used to measure changes in gross motor function over time.
Time frame
Baseline to Follow-Up, approximately 13 weeks3
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.
Time frame
Baseline to Post-Intervention, approximately 7 weeks4
Pediatric Berg Balance Scale (BBS)
The Pediatric BBS will be used to assess static balance and fall risk.
Time frame
Baseline to Follow-Up, approximately 13 weeksSponsors and contacts
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Source ClinicalTrials.gov