NCT01239758终止2 期
An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA4 个研究点 分布在 1 个国家目标入组 11 人开始时间: 2010年10月1日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 11
- 试验地点
- 4
- 主要终点
- Number of patients with adverse events.
研究概览
简要总结
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.]
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completion of participation in Study A031-03 and Investigator approval
- •Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
排除标准
- •Participation in any other therapeutic clinical trial
- •Plans to have surgery during the course of the study
结局指标
主要结局
Number of patients with adverse events.
时间窗: From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.
时间窗: Baseline to End-of-Study Visit, approximately 24 weeks later.
次要结局
- Change in pulmonary function tests.(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- Percent change in total lean body mass by DXA scan.(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- Percent change in total body and lumbar spine bone mineral density by DXA scan.(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- Percent change in muscle strength score by hand-held myometry.(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).(Baseline to End-of-Study Visit, approximately 24 weeks later.)
- Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).(Baseline to End-of-Study Visit, approximately 24 weeks later.)
研究者
研究点 (4)
Loading locations...
相似试验
终止
3 期
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)Duchenne Muscular DystrophyNCT03603288Santhera Pharmaceuticals161
终止
1 期
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)Muscular Dystrophy, DuchenneNCT03675126Sarepta Therapeutics, Inc.15
已完成
2 期
A Study to Evaluate the Safety, Tolerability and Efficacy of AIN457 in Patients With Relapsing-remitting Multiple SclerosisMultiple SclerosisNCT01433250Novartis Pharmaceuticals39
已完成
2 期
A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Nocturnal HemoglobinuriaNCT03181633Alexion Pharmaceuticals, Inc.8
终止
3 期
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)Distal Myopathy With Rimmed VacuolesDistal Myopathy, Nonaka TypeGNE MyopathyQuadriceps Sparing MyopathyHereditary Inclusion Body MyopathyNCT02736188Ultragenyx Pharmaceutical Inc143