Study of Velaglucerase Beta ERT in Children With Gaucher Disease
A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
調査の概要
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：Charlene Song
- 電話番号：+86 18817946880
- メール：xiaoling.song@canbridgepharma.com
研究連絡先のバックアップ
- 名前：Amelia Zhou
- 電話番号：+86 18016327369
- メール：feng.zhou@canrbdigepharma.com
研究場所
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Shanghai Municipality
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Shanghai、Shanghai Municipality、中国
- Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone
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コンタクト：
- Amelia Zhou
- 電話番号：+86 18016327369
- メール：feng.zhou@canrbdigepharma.com
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-
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:
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Subject has a documented diagnosis of GD1 or GD3 according to the diagnostic criteria of the Expert Consensus on the Diagnosis and Treatment of Gaucher Disease in Chinese Children 2021;
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Written informed consent provided by the subject's parent/legally authorized representative;
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Subject is less than 12 years of age;
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Subject has GD-related splenomegaly, defined as at least 2 to 3 cm below the left costal margin by palpation, and one or more of the following:
- GD-related anemia, with a decrease of ≥1 g/dL in hemoglobin concentration below the lower limit of normal for age of the central laboratory; or
- GD-related hepatomegaly by palpation; or
- GD-related thrombocytopenia, with a platelet count < 100 × 10^9/L;
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Subject has not received ERT or SRT for at least 3 months prior to screening; Female subjects of childbearing age have a negative serum pregnancy test during screening.
Exclusion Criteria:
- Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening;
- Subjects have received erythropoietin, whole blood transfusion or transfusion of red blood cells, or long-term (continuous treatment for more than 3 months) systemic corticosteroids 3 months prior to screening; or received a platelet transfusion within 1 month prior to screening;
- Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and/or vitamin B12 deficiency or infection/immune-mediated reasons);
- Subject has had a prior hepatectomy and/or splenectomy (including partial liver and/or splenectomy) or plans to have a hepatectomy and/or splenectomy (including partial liver and/or splenectomy) during the study;
- Subject has received organ transplantation, including hematopoietic stem cell transplantation;
- Subject has had a history of CTCAE Grade 3 or above infusion-related reaction or hypersensitivity reaction to imiglucerase or other ERTs (approved or experimental).
- Injection with a live vaccine within 30 days of the first study dose.
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：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 |
|---|---|
|
実験的：velaglucerase beta 60 U/kg
This is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks. |
All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months). |
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 |
|---|---|---|
|
Primary Endpoints
時間枠：From Baseline to Week 39
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Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase |
From Baseline to Week 39
|
|
Mean percent change in MN spleen volume
時間枠：From Baseline to Week 39
|
Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume / normal spleen volume |
From Baseline to Week 39
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Mean change in hemoglobin concentration (g/dL)
時間枠：From baseline to Week 39
|
From baseline to Week 39
|
|
|
Mean percent change in MN liver volume
時間枠：From Baseline to Week 39
|
Normal liver volume = 2.5% body weight. Multiples of normal (MN) liver volume= actual MRI testing volume / normal liver volume |
From Baseline to Week 39
|
|
Mean percent change in platelet count ( 10^9/L)
時間枠：From Baseline to Week 39
|
From Baseline to Week 39
|
|
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Mean change in height Z-score
時間枠：From Baseline to Week 39
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From Baseline to Week 39
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|
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Mean change in weight Z-score
時間枠：From Baseline to Week 39
|
From Baseline to Week 39
|
協力者と研究者
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
- CAN103-GD-301
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?
IPD プランの説明
only IPD used in the results publication
IPD 共有時間枠
Beginning 3 months and ending 3 years after the publication of results
IPD 共有アクセス基準
The criteria that must be met the types of analyses that qualify for data sharing.
A proposal that describes planned analyses must be submitted.
IPD 共有サポート情報タイプ
- STUDY_PROTOCOL
- SAP
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
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。