Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-Demand Treatment
2026年9月2日 更新者：Hangzhou Gensciences Biopharmaceutical Co., Ltd.
A Single-arm, Open-label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, and Immunogenicity of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-demand Treatment in Patients With Severe Hemophilia A (Adults and Adolescents) .
The indication for this product is to control bleeding in patients with hemophilia A (congenital deficiency of factor VIII).
The primary objective:
Evaluation of the efficacy of recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW107) as an on-demand treatment in previously treated patients with severe hemophilia A.
Secondary objectives:
Evaluation of the safety and immunogenicity of FRSW107 as an on-demand therapy in previously treated patients with severe hemophilia A.
Evaluate the on-demand treatment's PK profile of FRSW107 in previously treated patients with severe hemophilia A based on population pharmacokinetic (PopPK) methods ; preliminarily investigate the exposure-response (E-R) relationship of FRSW107 on-demand treatment in these patients if data permit.
調査の概要
研究の種類
介入
入学 (推定)
60
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。
研究場所
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Fuyang、中国
- Fuyang Hospital, Affiliated to Anhui Medical University
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Fuzhou、中国
- Fujian Medical University Union Hospital
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Ganzhou、中国
- Ganzhou People's Hospital
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Guangzhou、中国
- Nanfang Hospital of Southern Medical University
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Hefei、中国
- Anhui Provincial Hospital
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Huai'an、中国
- Huai'an Second People's Hospital
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Jinan、中国
- Jinan Central Hospital
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Kunming、中国
- The Second Affiliated Hospital of Kunming Medical University
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Nanning、中国
- The First Affiliated Hospital of Guangxi Medical University
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Nantong、中国
- Affiliated Hospital of Nantong University
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Nanyang、中国
- The First Affiliated Hospital of Nanyang Medical College
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Qinghai、中国
- Qinghai Provincial People's Hospital
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Rizhao、中国
- Rizhao People's Hospital
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Shijiazhuang、中国
- The Second Hospital of Hebei Medical University
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Taiyuan、中国
- The Second Hospital of Shanxi Medical University
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Tangshan、中国
- North China University of Science and Technology Affiliated Hospital
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Wenzhou、中国
- Wenzhou People's Hospital
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Wuhan、中国
- Union Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology.
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Wuxi、中国
- Affiliated Hospital of Jiangnan University
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Xi'an、中国
- Xi'an Central Hospital
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Yangzhou、中国
- Subei People's Hospital of Jiangsu province
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Zhengzhou、中国
- Henan Cancer Hospital
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Zhengzhou、中国
- Zhengzhou People's Hospital
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Tianjin Municipality
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Tianjin、Tianjin Municipality、中国
- Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
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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
Inclusive Criteria:
- Males aged 12 or younger to 65 years old.
- Patients clinically diagnosed with severe hemophilia A, i.e., those confirmed through screening or previous medical records: FⅧ Activity < 1%.
- Previous records confirm receipt of any recombinant and/or blood-derived coagulation factor VIII products or cold precipitate products, with a treatment of ≥150 exposure days (EDs ≥150).
- The participants have fully understood and been informed of this study, signed the informed consent form, and voluntarily enrolled in the clinical trial. The trial participants and/or their guardians are capable of cooperating with the hemostatic treatment and have the ability to complete all study procedures.
Exclusion Criteria:
- Known or suspected allergy to the investigational drug or its excipients, including mouse or hamster proteins;
- Hypersensitivity or anaphylaxis after FⅧ or IgG2 injection in the past;
- FⅧ inhibitor positive (≥0.6 BU/mL) during the screening period, or have a history of FⅧ inhibitor positive in the past, or a family history of FⅧ inhibitor positive;
- Von Willebrand factor (vWF) antigen test results were lower than the lower limit of normal value;
- Severe anemia at the screening stage (hemoglobin < 60 g/L);
- Platelet count during screening period < 100×109 /L;
Abnormal liver function:
.Alanine aminotransferase (ALT), or aspartate aminotransferase (AST) >3 times upper limit of normal (ULN); or Serum total bilirubin (TBIL) >1.5x ULN;
Patients with abnormal renal function:
Creatinine clearance (Ccr) <50 ml/min (according to Cockcroft and Gault formula); or Serum creatinine (Cr) >1.5x ULN;
- People with active hepatitis C, that is, hepatitis C virus (HCV) antibody positive and HCV RNA positive; Or anti-treponema pallidum specific antibody (TPHA) positive; Or positive for antibodies against the human immunodeficiency virus (HIV);
- Patients with coagulation dysfunction other than hemophilia A;
- Have a medical condition that may increase the risk of bleeding;
- A history of drug or alcohol abuse;
- Have a known mental disorder that may affect trial compliance;
- Patients who have received transfusions of blood or blood components within 4 weeks prior to screening;
- Participants who had participated in other clinical trials within 1 month before screening;
- Use of any anticoagulant or antiplatelet drugs, off-label maximum dose of non-steroidal anti-inflammatory drugs (NSAID) within 7 days prior to screening; Or patients who need to be treated with anticoagulant or antiplatelet drugs or off-label maximum doses of SAID during clinical trials;
- Severe cardiovascular and cerebrovascular disease or major thromboembolic events, such as stroke, myocardial infarction, unstable angina, congestive heart failure (New York Heart Association [NYHA] grade ≥ III), and severe arrhythmias (including QTc interphase > 480 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic ≥ 160 mmHg or diastolic ≥100 mmHg), deep vein thrombosis, etc.
- Study patients who had used emesezumab within 6 months prior to first administration of the drug;
- Patients who had used monoclonal antibody therapy, Fc fusion protein products (except FRSW107 and FRSW117), PEG products (except FRSW117), or intravenous immunoglobulin infusion within 3 months before the first administration of the investigational drug;
- Study patients who underwent major surgery within 3 months prior to initial drug administration (major surgery is defined in 6.2.3 Perioperative management);
- Study patients who have used FⅧ preparation of any standard half-life (e.g., Bycoch, Coproch, Biinidin, Renjie, NoL, Antaine, etc.) within 3 days or 5 half-lives prior to first administration of the drug (taking the elderly); Patients who have used any other extended half-life preparation FⅧ within 4 days or 5 half-lives prior to first dosing (for the elderly);
- Study patients with fever, severe active bacterial or viral infection, and allergies within 2 weeks before the first administration of the drug;
- Systemic immunomodulators (such as glucocorticoids [> 10 mg/ day equivalent dose of prednisone], alpha-interferon, immunoglobulin, cyclophosphamide, cyclosporin, etc.) used within 14 days prior to the first administration of the study drug or planned during the study period were allowed to be inhaled, nasal spray, or topical corticosteroids;
- Those who had been vaccinated within 4 weeks prior to initial administration of the study drug; Or who plan to be vaccinated during PK blood collection (only for subjects in the PK subgroup);
- Plan to have a child or sperm donation during the entire trial period and within 3 months after the last dose, or do not want to use effective physical contraception (such as condoms, diaphragms, Iuds, etc.);
- Have other serious medical conditions that the researchers said could not benefit from them
- Subjects deemed unsuitable by other investigators.
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：On-demand Treatment.
On-demand treatment (recommended range: 20-50 IU/kg)
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Treatment for 6 months as needed.
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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 |
|---|---|---|
|
The haemostatic effective rate
時間枠：6 months
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The haemostatic effect of FRSW107 when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.
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6 months
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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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Number of infusions and IU/kg of FRSW107 needed for the treatment of bleeding episodes
時間枠：6 months
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6 months
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FVIII activity during on-demand: activity recovery rate
時間枠：Visit1,Day1
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FVIII activity recovery rate on Day1's haemostatic therapy.
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Visit1,Day1
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Hemophilia Joint Health Score (HJHS 2.1) during on-demand treatment
時間枠：6 month
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total score and each subscale score of HJHS 2.1, as well as their changes over time.
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6 month
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incidence of lack of drug efficacy
時間枠：6 months
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Lack of drug efficacy is defined as: during on-demand treatment, hemostatic efficacy is assessed as "ineffective" or "poor/no response" following two consecutive administrations within 72 hours for the same bleeding episode. incidence of lack of drug efficacy=number of lack of drug efficacy/number of evaluated treatment*100%. |
6 months
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Occurrence of AEs and SAEs
時間枠：6 month
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6 month
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Incidence rate of positive FVIII inhibitors
時間枠：6 month
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A positive FⅧ inhibitor is defined as a Bethesda inhibitor titer ≥ 0.6 BU/mL.
Incidence rate of positive FVIII inhibitors=number of positive FⅧ inhibitors participant / total number of trial participants *100%.
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6 month
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Incidence rate of positive ADA, Incidence rate of positive anti-CHO antibody
時間枠：6 month
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Incidence rate of positive ADA=number of positive ADA participant / total number of trial participants *100% Incidence rate of positive anti-CHO antibody=number of positive anti-CHO antibody participant / total number of trial participants *100%
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6 month
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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。
捜査官
- スタディチェア：Renchi Yang, PhD、Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
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14日
一次修了 (推定)
2027年2月1日
研究の完了 (推定)
2027年6月1日
試験登録日
最初に提出
2026年6月15日
QC基準を満たした最初の提出物
2026年6月17日
最初の投稿 (実際)
2026年6月23日
学習記録の更新
投稿された最後の更新 (実際)
2026年9月8日
QC基準を満たした最後の更新が送信されました
2026年9月2日
最終確認日
2026年6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
- SS-107-III05
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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