Cerebral Palsy
Conditions
Keywords
Pediatric
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.
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.
Interventions
DEVICESmart 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.
DRUG5-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.
DRUGPlacebo
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.
Sponsors
Shirley Ryan AbilityLab
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
TRIPLE (Subject, Caregiver, Outcomes Assessor)
Masking description
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.
Intervention 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.
Eligibility
Sex/Gender
ALL
Age
8 Years to 17 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 6 Minute Walking Test | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 10 Meter Walking Test | Baseline to Follow-Up, approximately 13 weeks | Participants will walk 10 meters to assess gait speed. |
| Gross Motor Function Measure 88 (GMFM-88) | Baseline to Follow-Up, approximately 13 weeks | GMFM-88 will be used to measure changes in gross motor function over time. |
| Selective Control Assessment of the Lower Extremity (SCALE) | Baseline to Post-Intervention, approximately 7 weeks | The SCALE is a clinical assessment tool used to evaluate selective voluntary motor control of the lower limbs. |
| Pediatric Berg Balance Scale (BBS) | Baseline to Follow-Up, approximately 13 weeks | The Pediatric BBS will be used to assess static balance and fall risk. |
| Modified Tardieu Scale (MTS) | Baseline to Follow-Up, approximately 13 weeks | Modified Tardieu Scale will be completed to assess the muscle's response to stretch at given velocities. |
| Cerebral Palsy Questionnaire (CPCHILD) | Baseline to Follow-Up, approximately 13 weeks | CPCHILD will be administered to capture the child's and family's perspective on well-being and participation. |
| Wong-Baker FACES Pain Rating Scale | Baseline to Follow-Up, approximately 13 weeks | The Wong-Baker FACES Pain Rating Scale will be used for children to self-report their level of pain. |
| Pictorial Children's Effort Rating Table (PCERT) | Baseline to Follow-Up, approximately 13 weeks | The PCERT will be used for children to self-assess and communicate their perceived exertion during physical activity or exercise. |
Countries
United States
Contacts
CONTACTAudrey Wiesner, BS
CONTACTJacklyn Stoller, PT, DPT
PRINCIPAL_INVESTIGATORMegan O'Brien, PhD
Shirley Ryan AbilityLab
Outcome results
None listed