A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Genethon
- 入组人数
- 220
- 试验地点
- 15
- 主要终点
- Pulmonary Function Test (PFT)
研究概览
简要总结
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.
详细描述
Study duration from FPFV: Q1 2019 to LPLV: Q4 2026
Primary Ojectives:
- To assess the natural disease course using standardized and disease appropriate evaluations in a cohort of young male subjects aged from 4 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD).
- To record a baseline period prior to rolling over into a gene therapy phase I/II/III clinical study.
Secondary Objectives:
- To identify clinical, imaging and/or laboratory parameters that could be predictive indicators of the disease course in DMD, within the selected range of age.
- To identify the best outcome measure(s) for further clinical trial assessments.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 4 Years 至 9 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •4 to 9 years old inclusive
- •Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).
- •Related to the DMD disease:
- •Diagnosis of DMD based upon documented gene testing with detailed genotyping
- •Able to achieve at inclusion and screening visits:
- •NSAA (North Star Ambulatory Assessment) scale > 18 or ≥ 16 if participant is between 4 and < 5 years old at screening and:
- •Gowers test < or = 7 sec and/or
- •6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
- •Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit
- •Related to the study protocol and ICH/GCP (Good Clinical Practice) requirements:
- •Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
- •Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)
排除标准
- •Subject will be excluded from enrolment into the study for any of the following reasons:
- •Related to the DMD disease severity:
- •Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%
- •Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
- •Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History
- •Related to specific assessments:
- •Muscle testing: inability to cooperate with
- •MRI: metal implants in regions of interest for the study
- •Related to the study protocol and ICH/GCP requirements:
- •Unwilling and/or unable to comply with all the study protocol requirements and/or procedures
- •Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months or IMP washout period (whichever is longer) prior to the screening visit of the study
- •Previously treated with a gene therapy drug for DMD, such as:
- •any AAV mediated gene transfer products or any gene editing products in a clinical trial or in a clinical setting,
- •if exons skipping drug was used, the last dose of exon skipping drug within 5 half-lives prior to the screening visit
- •Concomitant participation to any other interventional clinical trial
结局指标
主要结局
Pulmonary Function Test (PFT)
时间窗: Inclusion 36 months
Respiratory Function Assessment
ACTIVLIM
时间窗: Inclusion 36 months
Patient Reported Outcome
EQ-5D
时间窗: Inclusion 36 months
Questionnaire of Life
NSAA scale
时间窗: Screening 36 months
NSAA scale (age appropriate modified North Star Ambulatory Assessment)
10 Meter Walk/ Run test (10MW/RT)
时间窗: Screening 36 months
Time function Test
Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI)
时间窗: Inclusion 36 months
Muscle Imaging
Myoset : Myo-grip, -pinch
时间窗: Inclusion 36 months
Motor Function Measurement
ECG - Echocardiography
时间窗: Inclusion 36 months
Cardiac Function Assessment
Raise from floor (RFF)
时间窗: Screening - 36 months
Time function test
6 Minutes Walk Test (6 MWT)
时间窗: Inclusion 36 months
Motor Function Measurement
Stride velocity 95th centile (SV95c)
时间窗: Inclusion 36 months
Motor Function Measurement with wearable device (SYDE)
次要结局
未报告次要终点