NCT07559513尚未招募3 期
Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 18
- 主要终点
- Concentrations of functional garetosmab in serum
研究概览
简要总结
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
The aim of the study is to see how safe, tolerable, and effective the study drug is.
The study is looking at several other research questions, including:
- What side effects may happen from taking the study drug
- How much study drug is in the blood at different times
- Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For USA participants, age criteria are 4 to < 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to < 18 years old
- •Must have a confirmation of FOP diagnosis, as described in the protocol
- •At the time of enrollment, participants must weight:
- •Cohort 1 > 30 kg
- •Cohort 2 > 30 kg
- •Cohort 3 ≤ 30 kg
排除标准
- •Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of screening
- •Participant has significant concomitant illness or history of significant illness, as described in the protocol
- •Previous history or diagnosis of cancer
- •Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration
- •History of severe respiratory compromise requiring oxygen, respiratory support
- •Known history of cerebral vascular malformation
- •Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise
- •Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol
- •NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply
研究组 & 干预措施
Cohort 1: Adolescents
Experimental
干预措施: garetosmab (Drug)
Cohort 2: Children
Experimental
干预措施: garetosmab (Drug)
Cohort 3: Children and Adolescents
Experimental
干预措施: garetosmab (Drug)
结局指标
主要结局
Concentrations of functional garetosmab in serum
时间窗: Through week 56
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
时间窗: Baseline to week 28
Occurrence of TEAEs
时间窗: Baseline to week 56
Severity of TEAEs
时间窗: Baseline to week 28
Severity of TEAEs
时间窗: Baseline to week 56
次要结局
- Occurence of Anti-Drug Antibody (ADA) to garetosmab(Through week 56)
- Total volume of new Heterotopic Ossification (HO) lesion(At week 28 and week 56)
- Number of new HO lesions(At week 28 and week 56)
- Occurrence of new HO lesions(At week 28 and week 56)
- Number of clinician-assessed flare-ups(Through week 28 and week 56)
- Occurrence of clinician-assessed flare-ups(Through week 28 and week 56)
- Number of patient/caregiver-reported flare-ups(Through Week 28 and week 56)
- Occurrence of patient/caregiver-reported flare-ups(Through week 28 and week 56)
- Change from baseline in Tanner puberty scale(At week 28 and week 56)
- Characteristics of menstrual cycles for female participants who reached menarche(Over 28 weeks and 56 weeks)
- Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children(Through week 56)
- Concentrations of total activin A in serum(Through week 56)
- Magnitude of ADA to garetosmab(Through week 56)
- Change from baseline in hearing function as assessed by audiometry(At week 28 and week 56)
- Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores(At week 28 and week 56)
- Acceptability and tolerability assessment via exit interview(Up to week 30)
研究者
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