Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 450
- 试验地点
- 41
- 主要终点
- 10m walk/run
研究概览
简要总结
The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and reachable workspace, as well as optional muscle biopsy and wearable device (US participants only).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring.
排除标准
- •Unwilling or unable to provide informed consent.
- •Any other medical condition which in the opinion of the investigator would interfere with study participation.
结局指标
主要结局
10m walk/run
时间窗: Baseline - 5 years
The 10-meter walk/run (previously the 30 foot go) or gait speed task will be performed during study visits. This task tests a range of different abilities, from power, to endurance, and balance. Also, the 10 meter walk/run is a predictor of loss of ambulation in Duchenne Muscular Dystrophy.
Shoulder and Arm Range of Motion
时间窗: Baseline - 5 years
Range of motion tasks mimicking lifting or reaching up will be performed.
Shoulder and Arm Function
时间窗: Baseline - 5 years
Participants will be timed on stacking cans.
Spirometry (FVC, FEV1, PCF)
时间窗: Baseline - 5 years
Investigators will obtain forced vital capacity and forced expiratory volume in 1 second, both standardized outcomes used commonly in clinic and clinical trials. Also, for sites who routinely collect Peak Cough Flow this will also be obtained.
次要结局
- MIP/MEP and SNIP(Baseline - 5 years)
- 4 Stair Climb(Baseline - 5 years)
- Manual Muscle Testing (MMT)(Baseline - 5 years)
- Exercise and Pain Assessment(Baseline - 5 years)
- The Facial Disability Index (FDI)(Baseline - 5 years)
- Trunk Function(Baseline - 5 years)
- Hand Function(Baseline - 5 years)
- Timed Up and Go (TUG)(Baseline - 5 years)
- Saliva Methylation(Baseline (collected only once))
- FSHD Clinical Severity Scores(Baseline - 5 years)
- Patient-Reported Outcomes Measurement Information System-57 (PROMIS57)(Baseline - 5 years)
- The Upper Extremity Functional Index(Baseline - 5 years)
- FSHD Rasch-built overall disability scales (FSHD-RODS)(Baseline - 5 years)