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)
調査の概要
研究の種類
介入
入学 (推定)
18
段階
- フェーズ 3
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究連絡先
- 名前：Clinical Trials Administrator
- 電話番号：844-734-6643
- メール：clinicaltrials@regeneron.com
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
- 大人
健康ボランティアの受け入れ
いいえ
説明
Key Inclusion Criteria:
- 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
Key Exclusion Criteria:
- 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
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：非ランダム化
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Cohort 1: Adolescents
|
Administered per the protocol
他の名前：
|
|
実験的：Cohort 2: Children
|
Administered per the protocol
他の名前：
|
|
実験的：Cohort 3: Children and Adolescents
|
Administered per the protocol
他の名前：
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
時間枠 |
|---|---|
|
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
時間枠：Baseline to week 28
|
Baseline to week 28
|
|
Occurrence of TEAEs
時間枠：Baseline to week 56
|
Baseline to week 56
|
|
Severity of TEAEs
時間枠：Baseline to week 28
|
Baseline to week 28
|
|
Severity of TEAEs
時間枠：Baseline to week 56
|
Baseline to week 56
|
|
Concentrations of functional garetosmab in serum
時間枠：Through week 56
|
Through week 56
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Total volume of new Heterotopic Ossification (HO) lesion
時間枠：At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of new HO lesions
時間枠：At week 28 and week 56
|
At week 28 and week 56
|
|
|
Occurrence of new HO lesions
時間枠：At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of clinician-assessed flare-ups
時間枠：Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Occurrence of clinician-assessed flare-ups
時間枠：Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Number of patient/caregiver-reported flare-ups
時間枠：Through Week 28 and week 56
|
Through Week 28 and week 56
|
|
|
Occurrence of patient/caregiver-reported flare-ups
時間枠：Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Change from baseline in Tanner puberty scale
時間枠：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
時間枠：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
時間枠：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
時間枠：Through week 56
|
Through week 56
|
|
|
Occurence of Anti-Drug Antibody (ADA) to garetosmab
時間枠：Through week 56
|
Through week 56
|
|
|
Magnitude of ADA to garetosmab
時間枠：Through week 56
|
Through week 56
|
|
|
Change from baseline in hearing function as assessed by audiometry
時間枠：At week 28 and week 56
|
At week 28 and week 56
|
|
|
Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores
時間枠：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
時間枠：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
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
捜査官
- スタディディレクター：Clinical Trial Management、Regeneron Pharmaceuticals
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (推定)
2027年2月2日
一次修了 (推定)
2028年12月10日
研究の完了 (推定)
2029年12月21日
試験登録日
最初に提出
2026年4月22日
QC基準を満たした最初の提出物
2026年4月22日
最初の投稿 (実際)
2026年4月30日
学習記録の更新
投稿された最後の更新 (実際)
2026年8月24日
QC基準を満たした最後の更新が送信されました
2026年8月20日
最終確認日
2026年8月1日
詳しくは
本研究に関する用語
その他の研究ID番号
- R2477-FOP-2413
- 2024-518415-19-01 (Ctis)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
はい
IPD プランの説明
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
IPD 共有時間枠
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 共有アクセス基準
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 共有サポート情報タイプ
- STUDY_PROTOCOL
- SAP
- ICF
- ANALYTIC_CODE
- CSR
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
はい
米国FDA規制機器製品の研究
いいえ
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。