An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
2026年7月23日 更新者：BioMarin Pharmaceutical
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.
調査の概要
詳細な説明
This Phase 2, multi-center, open-label extension study is designed to assess the long-term safety, tolerability, and functional efficacy of weekly intravenous doses of BMN 351 administered to participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping in the Phase 1/2 study, 351-201. Up to 18 participants ages 4 through 23 at baseline will enroll in the trial after completing 351-201.
The first visit for this study is the same as the final visit of 351-201. To be eligible for this study, potential participants must satisfy the eligibility criteria described in the protocol.
研究の種類
介入
入学 (推定)
18
段階
- フェーズ2
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。
研究場所
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United Kingdom
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London、United Kingdom、イギリス、WC1N 3JH
- Great Ormond Street Hospital NHS Foundation Trust
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Italy
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Milan、Italy、イタリア
- Fondazione Serena ETS - Centro Clinico NeMO Milano
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Rome、Italy、イタリア
- UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Netherlands
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Leiden、Netherlands、オランダ、2333 ZA
- Leids Universitair Medisch Centrum
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Spain
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Seville、Spain、スペイン、41013
- Hospital Viamed Santa Angela De la Cruz
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Turkey
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Istanbul、Turkey、トルコ（Türkiye）
- Yeditepe University Kosuyolu Hospital
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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
- 高齢者
健康ボランティアの受け入れ
いいえ
説明
Inclusion Criteria:
- Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study
Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight
- Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries.
- Willing and able to adhere to the study visit schedule and other protocol requirements
Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
- Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure
- Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure
Exclusion Criteria:
- Have known coagulation disorder
Are taking any prohibited medications
- any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time
- anti-coagulants, anti-thrombotics, or anti-platelet agents
- immunosuppressants
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：なし
- 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 |
|---|---|
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実験的：BMN 351
Participants from 351-201 enrolling in 351-202 will initially receive BMN 351 at the dose level at which they completed 351-201.
Once all 351-201 participants have completed their Week 25 visit in 351-202, available safety and PD data will be analyzed and a single optimal dose level will be selected for 351-202.
All participants from 351-201 will transition to that dose level at their next applicable visit.
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Anti-sense Oligonucleotide BMN 351 will be administered intravenously
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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 |
メジャーの説明 |
時間枠 |
|---|---|---|
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To assess the long-term safety and tolerability of BMN 351 in participants with DMD
時間枠：Through study completion, at least 1 year
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The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
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Through study completion, at least 1 year
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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To evaluate the effect of BMN 351 on physical function
時間枠：Change from baseline and subsequent 24-week incremental visits
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Timed 4 stair climb (4SC) will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
時間枠：Change from baseline and subsequent 24-week incremental visits
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SV95C will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
時間枠：Change from baseline and subsequent 24-week incremental visits
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PUL 2.0 will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
時間枠：Change from baseline and subsequent 24-week incremental visits
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North Star Ambulatory Assessment (NSAA) will be assessed at the specific visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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To evaluate the trough plasma concentration of BMN 351
時間枠：Baseline, Week 13, Week 25, Week 37, Week 49
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Serial predose measurements of trough plasma PK
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Baseline, Week 13, Week 25, Week 37, Week 49
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To evaluate the immune response to BMN 351
時間枠：Change from baseline and subsequent 24-week incremental visits
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Anti-BMN 351 antibodies and Anti-dystrophin antibodies
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Change from baseline and subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
時間枠：Change from baseline to subsequent 24-week incremental visits
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Forced Expiratory Volume in 1 second (FEV1) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
時間枠：Change from baseline to subsequent 24-week incremental visits
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Forced Vital Capacity (FVC) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
時間枠：Change from baseline to subsequent 24-week incremental visits
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Peak Cough Flow (PCF) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
時間枠：Change from baseline to subsequent 24-week incremental visits
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Maximal Inspiratory Pressure (MIP) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
時間枠：Change from baseline to subsequent 24-week incremental visits
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Maximal Expiratory Pressure (MEP) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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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。
スポンサー
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 (実際)
2026年4月20日
一次修了 (推定)
2031年12月1日
研究の完了 (推定)
2031年12月1日
試験登録日
最初に提出
2026年4月17日
QC基準を満たした最初の提出物
2026年5月1日
最初の投稿 (実際)
2026年5月7日
学習記録の更新
投稿された最後の更新 (実際)
2026年7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7月23日
最終確認日
2026年7月1日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- 351-202
- 2024-511656-41-00 (Ctis)
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
いいえ
米国FDA規制機器製品の研究
いいえ
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