Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia (cAtCH)
A Phase 3, Randomized, Double-blind, Active Controlled, Multi-center Trial Investigating the Efficacy and Safety of Dual Therapy With Once-weekly Navepegritide and Once-weekly Lonapegsomatropin Compared to Navepegritide Monotherapy and Lonapegsomatropin Monotherapy in Children and Adolescents With Achondroplasia
This pivotal trial is being conducted to evaluate the efficacy, safety and tolerability of dual therapy with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 17 years.
調査の概要
状態
条件
研究の種類
入学 (推定)
段階
- フェーズ 3
連絡先と場所
研究連絡先
- 名前：Ascendis Registry Inquiries
- 電話番号：+45 61242484
- メール：asnd_registryinquiries@ascendispharma.com
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of ACH with historical genetic confirmation
- 3. At least 1 height measurement available from medical records
Exclusion Criteria:
- 1. Treatment with growth hormone within 1 year prior to randomization
- 2. Prior treatment with certain medication(s) which potentially could have affected growth (prior treatment with vosoritide is not exclusive)
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than achondroplasia that could affect growth
- 6. Known history or presence of injury of the growth plates that affects growth potential of long bones
- 7. Known history or presence of malignant disease
研究計画
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：ランダム化
- 介入モデル：並列代入
- マスキング：4倍
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Once weekly navepegritide SC and lonapegsomatropin SC
|
Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks |
|
実験的：Once weekly navepegritide SC and placebo for lonapegsomatropin SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of navepegritide and placebo for lonapegsomatropin followed by 52 weeks of navepegritide and lonapegsomatropin. |
|
実験的：Once weekly lonapegsomatropin SC and placebo for navepegritide SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of lonapegsomatropin and placebo for navepegritide followed by 52 weeks of navepegritide and lonapegsomatropin. |
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 |
|---|---|---|
|
Annualized Growth Velocity (AGV)
時間枠：52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone |
52 weeks
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Treatment-emergent Adverse Events (AE)
時間枠：Throughout trial duration 104 weeks
|
Number of events |
Throughout trial duration 104 weeks
|
|
Annualized Growth Velocity (AGV)
時間枠：52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to lonapegsomatroptin alone |
52 weeks
|
|
Annualized Growth Velocity (AGV) change from baseline
時間枠：52 weeks
|
To evaluate the change from baseline in AGV, cm per year |
52 weeks
|
|
ACH-specific height Z-score
時間枠：52 weeks
|
To evaluate the change from baseline in ACH-specific height Z-score |
52 weeks
|
|
CDC-based height Z-score
時間枠：52 weeks
|
To evaluate the change from baseline in CDC-based height Z-score |
52 weeks
|
|
Bone safety
時間枠：Throughout trial duration 104 weeks
|
Qualitative assessment with radiology |
Throughout trial duration 104 weeks
|
|
Arm span Z-score
時間枠：52 weeks
|
Change from baseline in arm span Z-score. |
52 weeks
|
|
Upper to lower body segment ratio
時間枠：52 weeks
|
Change from baseline in upper to lower body segment ratio. |
52 weeks
|
|
Arm span
時間枠：52 weeks
|
Change from baseline in arm span, cm. |
52 weeks
|
|
Interpedicular distance (IDP)
時間枠：52 weeks
|
Change from baseline in IDP (L1-L4 average), mm |
52 weeks
|
協力者と研究者
スポンサー
捜査官
- スタディディレクター：Medical Director, MD、Ascendis Pharma A/S
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
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
- ASND0053
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
米国FDA規制機器製品の研究
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