Smart Ankle-Foot Orthosis and 5-Azacitidine to Enhance Gait and Community Mobility for Children With Cerebral Palsy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- 6 Minute Walking Test
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- Triple (Participant, Care Provider, Outcomes Assessor)
盲法说明
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.
入排标准
- 年龄范围
- 8 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Smart AFO & Home Intervention Training + 5-Azacitidine
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:
- 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)
- Monitoring Heart Rate
- 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.
干预措施: 5-Azacitidine (Drug)
Smart AFO & Home Intervention Training + 5-Azacitidine
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:
- 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)
- Monitoring Heart Rate
- 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.
干预措施: Smart Ankle-Foot Orthosis (Device)
Smart AFO & Home Intervention Training + Mannitol (Placebo)
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:
- 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)
- Monitoring Heart Rate
- 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.
干预措施: Smart Ankle-Foot Orthosis (Device)
Smart AFO & Home Intervention Training + Mannitol (Placebo)
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:
- 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)
- Monitoring Heart Rate
- 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.
干预措施: Placebo (Drug)
Smart AFO & Home Intervention Training
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:
- 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)
- Monitoring Heart Rate
- Caregiver filling out brief log to track training session details.
干预措施: Smart Ankle-Foot Orthosis (Device)
结局指标
主要结局
6 Minute Walking Test
时间窗: 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.
次要结局
- Cerebral Palsy Quality of Life Questionnaire (CP-QOL)(Baseline to Follow-Up, approximately 13 weeks)
- 10 Meter Walking Test(Baseline to Follow-Up, approximately 13 weeks)
- Gross Motor Function Measure 88 (GMFM-88)(Baseline to Follow-Up, approximately 13 weeks)
- Selective Control Assessment of the Lower Extremity (SCALE)(Baseline to Post-Intervention, approximately 7 weeks)
- Pediatric Berg Balance Scale (BBS)(Baseline to Follow-Up, approximately 13 weeks)
- Modified Tardieu Scale (MTS)(Baseline to Follow-Up, approximately 13 weeks)
- Cerebral Palsy Questionnaire (CPCHILD)(Baseline to Follow-Up, approximately 13 weeks)
- Wong-Baker FACES Pain Rating Scale(Baseline to Follow-Up, approximately 13 weeks)
- Pictorial Children's Effort Rating Table (PCERT)(Baseline to Follow-Up, approximately 13 weeks)
研究者
Megan O'Brien
Associate Director, Technology & Innovation
Shirley Ryan AbilityLab