A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Participants 4 to 8 Years of Age With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 44
- 试验地点
- 16
- 主要终点
- Time to Complete 10 Meter Walk/Run[
研究概览
简要总结
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
详细描述
The main purpose of this study is to determine the effect of MNK-1411 on motor function in participants with Duchenne Muscular Dystrophy (DMD). Information is collected only from caretakers who are fluent in English, using the Pediatric Outcomes Data Collection Instrument (PODCI).
The PODCI is a validated 86-question instrument completed by the parent or legal guardian of children 2 to 10 years of age to assess a variety of health outcome measures (Uzark et al, 2012). This study will only collect information for the PODCI domains of sports and physical functioning and transfer/basic mobility.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
Double blind (Investigator/Participant) The Care Provider and Outcomes Assessor were also blinded, but it is a double-blind trial, followed by an open-label period.
入排标准
- 年龄范围
- 4 Years 至 8 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants must have a documented diagnosis of Duchenne Muscular Dystrophy (DMD) confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as "out of frame," or complete dystrophin gene sequencing consistent with DMD; AND in the opinion of the Investigator, a typical clinical profile consistent with DMD.
- •Participants taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (e.g., eteplirsen or ataluren) may be enrolled in the study if they have been on a stable dose for 30 days prior to the first dose of study drug, and plan to remain on that dose throughout the study.
排除标准
- •Participant has had previous systemic treatment with corticosteroids within 2 months prior to the Screening Visit. Exception: In subjects who were down-titrated to a physiological dose of corticosteroids (ie, 3mg/m2 of prednisone or deflazacort) a maximum of 1 month of no greater than a physiological dose followed by 1 month completely off corticosteroids prior to the Screening Visit will be acceptable for study entry. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. The use of topical or intra-articular corticosteroids is permitted during the study
- •Participant is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
- •Participant has Type 1 or Type 2 diabetes mellitus.
- •Participant has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
- •Participant has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
- •Participant has known immune compromised status (not related to disease/condition under study), including but not limited to, individuals who have undergone organ transplantation or who are known to be positive for the human immunodeficiency virus.
研究组 & 干预措施
Period 1: MNK-1411
Participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 1
干预措施: MNK-1411 (Drug)
Period 1: Placebo
Participants receive placebo at a volume appropriate to body weight during Period 1
干预措施: Placebo (Other)
Period 2: MNK-1411
All participants receive MNK-1411 at a dosing volume appropriate to body weight during Period 2
干预措施: MNK-1411 (Drug)
结局指标
主要结局
Time to Complete 10 Meter Walk/Run[
时间窗: Baseline, Week 24
10 Meter Walk/Run is a motor function test to measure the functional capability in patients with DMD.
次要结局
- Time to Climb 4 Standardized Stairs(Baseline, Week 24)
- Quantitative Muscle Testing Scores at Baseline(Baseline)
- North Star Ambulatory Assessment (NSAA) Score(Baseline, Week 24)
- Time to Stand From a Supine Position(Baseline, Week 24)
- Quantitative Muscle Testing Scores at Week 24(Week 24)
- Summary of Adverse Events in the Blinded Treatment Period(within 28 weeks)
- Summary of Adverse Events in the Open Label Period(within 28 weeks)