An Open-Label, Phase 3 Long-Term Extension (LTE) Study To Assess The Safety, Tolerability, And Efficacy Of Treatment With Pegtibatinase In Participants With Classical Homocystinuria (HCU) Due To Cystathionine Beta Synthase Deficiency (ENSEMBLE)
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 100
- 试验地点
- 54
- 主要终点
- Treatment-emergent AEs
研究概览
简要总结
The goal of this long-term extension (LTE) study is to evaluate the safety and efficacy of pegtibatinase in patients with classical homocystinuria (HCU). Patients who are active in the Phase 1/2 COMPOSE study or those who complete the 24 weeks of blinded treatment in the Phase 3 HARMONY are eligible to participate.
Participants will be in this clinical study for a minimum of 108 weeks including:
- a treatment period of up to 104 weeks
- a 4-week safety follow-up period
详细描述
Overall Design:
This is a global, multicenter, single-arm, open-label study. Participants who meet all eligibility criteria may transition from other pegtibatinase studies and enroll into this LTE study for long-term safety, efficacy, and clinical assessments of pegtibatinase treatment.
All participants will follow a protocol of self-administration of pegtibatinase unless exempted. During the study, pegtibatinase pharmacokinetic assessments will be conducted. An optional protein tolerance modification (PTM) sub-study will also be conducted for eligible participants.
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
- 5 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study
排除标准
- •Participant permanently discontinued from study intervention treatment in a previous pegtibatinase study due to serious AE (SAE) or intolerance
研究组 & 干预措施
pegtibatinase
干预措施: Pegtibatinase (Drug)
结局指标
主要结局
Treatment-emergent AEs
时间窗: Week 1 - Week 108
Incidence of treatment-emergent AEs
Hypermethioninemia
时间窗: Week 1 - Week 108
Incidence of hypermethioninemia
Hypomethioninemia
时间窗: Week 1 - Week 108
Incidence of hypomethioninemia
Dietary Protein Rescue
时间窗: Week 1 - Week 108
Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia
次要结局
- Total Homocysteine (tHcy) Levels(Week 1 - Week 108)