Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Incidence of AEs in all participants
研究概览
简要总结
This is a Phase 1/2a, monocentric, open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51 in patients with Duchenne muscular dystrophy
详细描述
Avance1 is a Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of SQY51 in 12 patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy, This study will include i) 13-week Phase 1 Multiple Dose Escalation Phase, and a ii) 32-week Phase 2a.
Twelve (12) patients ≥ 6 years, both ambulant and non-ambulant, will be sequentially enrolled in phase 1 and will receive escalating doses of SQY51 once every two weeks. In phase 2a, patients will be allocated in three cohorts in a non-randomized manner.
On the 25th March 2024, SQY Therapeutics received the authorization from the European Medicines Agency (EMA) to initiate the Phase 2a clinical trial. All the patients involved in the Phase 1 will progress to the Phase 2a.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Phase 1
Participants will receive single escalating doses of 2, 4, 6, 10, 16 and 25 mg/kg by intravenous infusion of SQY51 every 2 weeks.
干预措施: Phase 1, SQY51 (Drug)
Phase 2a - Treatment arm (Dose 1)
Non randomized participants will receive by IV dose 1 of SQY51 in 4 blocks of 4-weeks.
干预措施: Phase 2a, SQY51 (cohort 1) (Drug)
Phase 2a - Treatment arm (Dose 2)
Non randomized participants will receive by IV dose 2 of SQY51 in 4 blocks of 4-weeks.
干预措施: Phase 2a, SQY51 (cohort 2) (Drug)
Phase 2a - Treatment arm (Dose 3)
Non randomized participants will receive by IV dose 3 of SQY51 in 4 blocks of 4-weeks.
干预措施: Phase 2a, SQY51 (cohort 3) (Drug)
结局指标
主要结局
Incidence of AEs in all participants
时间窗: From baseline up to week 49
次要结局
- Change from baseline in time to rise from floor, time to complete 1-min, 6-min and 10-min walk in ambulant patients as well as MFM and PUL scores in both ambulant and non-ambulant patients(From baseline up to week 49)
- Changes from baseline in skeletal muscle dystrophin expression(From baseline up to week 49)
- Pharmacokinetic plasma concentration of SQY51 (µg/ml)(From baseline up to week 49)