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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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:
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. |
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| 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:
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. |
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| Smart AFO & Home Intervention Training | Active Comparator |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| 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 |
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Audrey Wiesner, BS | Contact | 3122388435 | awiesner@sralab.org | |
| Jacklyn Stoller, PT, DPT | Contact | 3122387620 | jstoller@sralab.org |
| Name | Affiliation | Role |
|---|---|---|
| Megan O'Brien, PhD | Shirley Ryan AbilityLab | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Shirley Ryan AbilityLab | Recruiting | Chicago | Illinois | 60611 | United States |
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| ID | Term |
|---|---|
| D002547 | Cerebral Palsy |
| ID | Term |
|---|---|
| D001925 | Brain Damage, Chronic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D001374 | Azacitidine |
| D008353 | Mannitol |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
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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.
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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.
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:
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|
| 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. |
|
|
| 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. |
|
|
| 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 |
| D011743 |
| Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |
| D013402 | Sugar Alcohols |
| D000438 | Alcohols |
| D002241 | Carbohydrates |