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A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA-2)

2026年8月20日 更新者：Regeneron Pharmaceuticals

Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva

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)

研究概览

地位

尚未招聘

条件

干预/治疗

研究类型

介入性

注册 (估计的)

18

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子
  • 成人

接受健康志愿者

不

描述

Key Inclusion Criteria:

  1. 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
  2. Must have a confirmation of FOP diagnosis, as described in the protocol

  3. At the time of enrollment, participants must weight:

    1. Cohort 1 > 30 kg
    2. Cohort 2 > 30 kg
    3. Cohort 3 ≤ 30 kg

Key Exclusion Criteria:

  1. Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of screening
  2. Participant has significant concomitant illness or history of significant illness, as described in the protocol
  3. Previous history or diagnosis of cancer
  4. Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration
  5. History of severe respiratory compromise requiring oxygen, respiratory support
  6. Known history of cerebral vascular malformation
  7. Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise
  8. 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

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：非随机化
  • 介入模型：单组作业
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：Cohort 1: Adolescents
药品：garetosmab
Administered per the protocol
其他名称：
  • REGN2477
实验性的：Cohort 2: Children
药品：garetosmab
Administered per the protocol
其他名称：
  • REGN2477
实验性的：Cohort 3: Children and Adolescents
药品：garetosmab
Administered per the protocol
其他名称：
  • REGN2477

研究衡量的是什么？

主要结果指标

结果测量
大体时间
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
大体时间：Baseline to week 28
Baseline to week 28
Occurrence of TEAEs
大体时间：Baseline to week 56
Baseline to week 56
Severity of TEAEs
大体时间：Baseline to week 28
Baseline to week 28
Severity of TEAEs
大体时间：Baseline to week 56
Baseline to week 56
Concentrations of functional garetosmab in serum
大体时间：Through week 56
Through week 56

次要结果测量

结果测量
措施说明
大体时间
Total volume of new Heterotopic Ossification (HO) lesion
大体时间：At week 28 and week 56
At week 28 and week 56
Number of new HO lesions
大体时间：At week 28 and week 56
At week 28 and week 56
Occurrence of new HO lesions
大体时间：At week 28 and week 56
At week 28 and week 56
Number of clinician-assessed flare-ups
大体时间：Through week 28 and week 56
Through week 28 and week 56
Occurrence of clinician-assessed flare-ups
大体时间：Through week 28 and week 56
Through week 28 and week 56
Number of patient/caregiver-reported flare-ups
大体时间：Through Week 28 and week 56
Through Week 28 and week 56
Occurrence of patient/caregiver-reported flare-ups
大体时间：Through week 28 and week 56
Through week 28 and week 56
Change from baseline in Tanner puberty scale
大体时间：At week 28 and week 56
Tanner puberty scale or stages: Staging of sexual development is graded on a 5-point ordinal scale ranging from 1 (prepubertal) to 5 (adultlike) for female breast development, male external genitals, and pubic hair
At week 28 and week 56
Characteristics of menstrual cycles for female participants who reached menarche
大体时间：Over 28 weeks and 56 weeks
Over 28 weeks and 56 weeks
Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children
大体时间：Through week 56
Participants 5-19 years of age Z-score represents standardized measure of how far an individual deviated from study cohort average at baseline. A higher Z-score reflects better performance.
Through week 56
Concentrations of total activin A in serum
大体时间：Through week 56
Through week 56
Occurence of Anti-Drug Antibody (ADA) to garetosmab
大体时间：Through week 56
Through week 56
Magnitude of ADA to garetosmab
大体时间：Through week 56
Through week 56
Change from baseline in hearing function as assessed by audiometry
大体时间：At week 28 and week 56
At week 28 and week 56
Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores
大体时间：At week 28 and week 56
Age-appropriate PedsQL Generic Core Scales will be used to measure HRQoL in children and adolescents. Response options include 5-point Likert scale (or 3-point Likert scale for the young children self-report) for each item asking about experience within the past week. Global scores are transformed to a 0 to 100 scale with higher scores indicating better quality of life.
At week 28 and week 56
Acceptability and tolerability assessment via exit interview
大体时间：Up to week 30
Each interview will be conducted by trained external interviewers following a semi-structured interview guide of questions for the participants about their overall experience in the trial.
Up to week 30

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Regeneron Pharmaceuticals

调查人员

  • 研究主任：Clinical Trial Management、Regeneron Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2027年2月2日

初级完成 (估计的)

2028年12月10日

研究完成 (估计的)

2029年12月21日

研究注册日期

首次提交

2026年4月22日

首先提交符合 QC 标准的

2026年4月22日

首次发布 (实际的)

2026年4月30日

研究记录更新

最后更新发布 (实际的)

2026年8月24日

上次提交的符合 QC 标准的更新

2026年8月20日

最后验证

2026年8月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • R2477-FOP-2413
  • 2024-518415-19-01 (克蒂斯)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.

IPD 共享时间框架

When Regeneron has:

  • received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
  • made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry)
  • the legal authority to share the data, and
  • ensured the ability to protect participant privacy

IPD 共享访问标准

Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

IPD 共享支持信息类型

  • 研究方案
  • 树液
  • 国际碳纤维联合会
  • 分析代码
  • 企业社会责任

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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