NL-OMON21367招募中不适用
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of PRO051 in Patients With Duchenne Muscular Dystrophy
Prosensa0 个研究点目标入组 18 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 18
研究概览
简要总结
/A
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •1. Boys aged between 5 and 16 years inclusive
- •2. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO051
排除标准
- •1. Aberrant RNA splicing and/or aberrant response to PRO051, detected by in vitro PRO051 assay during screening
- •2. Known presence of dystrophin in > 5% of fibres in a pre-study diagnostic muscle biopsy
研究者
相似试验
进行中（未招募）
不适用
A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophyMedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophyDuchenne Muscular DystrophyEUCTR2007-004819-54-NLProsensa Holding B.V.18
进行中（未招募）
1 期
A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne DiseaseDuchenne Muscular DystrophyMedDRA version: 17.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2007-004819-54-BEProsensa Therapeutics BV12
进行中（未招募）
1 期
A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne DiseaseDuchenne Muscular DystrophyEUCTR2007-004819-54-SEProsensa Therapeutics BV12
进行中（未招募）
不适用
A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyDuchenne Muscular DystrophyEUCTR2009-013762-63-NLProsensa Therapeutics B.V18
进行中（未招募）
1 期
A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyDuchenne Muscular DystrophyEUCTR2009-013762-63-BEProsensa Therapeutics B.V24