ISRCTN11652897进行中（未招募）2 期
A Phase II/III, multicenter, double-blind, randomized study to determine the efficacy and safety of DNL310 vs idursulfase in pediatric participants with neuronopathic or non-neuronopathic mucopolysaccharidosis type II
Denali Therapeutics (United States)0 个研究点目标入组 54 人开始时间: 2022年6月30日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 54
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Participants aged =2 to <6 years (Cohort A) or =6 to <17 years (Cohort B)
- •2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
- •3. Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening
排除标准
- •1. Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
- •2. Previously received an IDS gene therapy or stem cell therapy
- •3. Received any CNS-targeted MPS ERT within 6 months prior to screening
- •4. Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
- •5. Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study
研究者
相似试验
进行中（未招募）
不适用
A Phase IIb/III, multi-centre, double-blind, randomised, placebo-controlled, dose ranging study of tamsulosin hydrochloride (low, medium and high dose) as treatment in children with neuropathic bladder for three monthseurogenic BladderMedDRA version: 8.1Level: LLTClassification code 10029279Term: Neurogenic bladderEUCTR2006-003048-52-DEBoehringer Ingelheim Pharma GmbH & Co. KG120
进行中（未招募）
不适用
A Phase IIb/III, multi-centre, double-blind, randomised, placebo-controlled, dose ranging study of tamsulosin hydrochloride (low, medium and high dose) as treatment in children with neuropathic bladder for three monthseurogenic BladderMedDRA version: 8.1Level: LLTClassification code 10029279Term: Neurogenic bladderEUCTR2006-003048-52-BESCS Boehringer Ingelheim Comm.V120
进行中（未招募）
不适用
A Phase IIb/III, multi-centre, double-blind, randomised, placebo-controlled, dose ranging study of Tamsulosin hydrochloride (low, medium and high dose) as treatment in children with neuropathic bladder for three months - NDneurogenic bladderEUCTR2006-003048-52-ITBOEHRINGER ING.250
进行中（未招募）
1 期
Study to Evaluate the Efficacy and Safety of MK-5475 in Adults with Pulmonary Arterial HypertensioMedDRA version: 20.0Level: LLTClassification code 10065150Term: Associated with pulmonary arterial hypertensionSystem Organ Class: 100000004855Pulmonary arterial hypertensionEUCTR2020-001108-40-ITMERCK SHARP & DOHME CORP. UNA SUSSIDIARIA DI MERCK & CO. INC.450
进行中（未招募）
1 期
Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Oral Ozanimod in Pediatric Participants with Moderately to Severely Active Crohn’s Disease with an Inadequate Response to Conventional TherapyEUCTR2021-005019-30-PLCelgene International II Sarl120