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

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Устройство 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.
• Препарат 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.
• Препарат 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.

Первичные конечные точки

• 6 Minute Walking Test [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]

Вторичные конечные точки (8)

• 10 Meter Walking Test [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Gross Motor Function Measure 88 (GMFM-88) [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Selective Control Assessment of the Lower Extremity (SCALE) [Срок оценки: Baseline to Post-Intervention, approximately 7 weeks]
• Pediatric Berg Balance Scale (BBS) [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Modified Tardieu Scale (MTS) [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Wong-Baker FACES Pain Rating Scale [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Pictorial Children's Effort Rating Table (PCERT) [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]
• Cerebral Palsy Quality of Life Questionnaire (CP-QOL) [Срок оценки: Baseline to Follow-Up, approximately 13 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Другое

Центры проведения

США · 1 центр

• Shirley Ryan AbilityLab — Chicago

Идентификаторы

NCT: NCT07434492 · STU00225131

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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