An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 130
- 试验地点
- 23
- 主要终点
- Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
研究概览
简要总结
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.
详细描述
The study includes a 104-week open-label treatment period with edasalonexent. Patients who completed CAT-1004-201 or CAT-1004-301 and eligible siblings of these boys will be enrolled in this trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- •Completion of either CAT-1004-201 or CAT-1004-301
排除标准
- •In the Investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures
- •For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301:
- •Inclusion Criteria:
- •Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
- •A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301
- •Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
- •Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
- •Exclusion Criteria:
- •Use of oral corticosteroids at screening; use of inhaled, intranasal, and topical corticosteroids is permitted
- •Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. Exception: Patients who are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible
- •Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus
- •Use of human growth hormone within 3 months prior to Day 1
- •Other prior or ongoing significant medical conditions
研究组 & 干预措施
Dose 1
Edasalonexent 100mg/kg/day. Capsules taken by mouth three times per day.
干预措施: Edasalonexent (Drug)
结局指标
主要结局
Safety and tolerability of long-term treatment with edasalonexent measured by number of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
时间窗: 104 Weeks
次要结局
- Durability of effects of edasalonexent on physical function as measured by the 10-meter walk/run test(104 Weeks)
- Durability of effects of edasalonexent on physical function as measured by the time to stand from supine(104 Weeks)
- Durability of effects of edasalonexent on physical function as measured by the North Star Ambulatory Assessment (NSAA)(104 Weeks)
- Durability of effects of edasalonexent on physical function as measured by the 4-stair climb(104 Weeks)