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Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Treatment.

2 settembre 2026 aggiornato da: Hangzhou Gensciences Biopharmaceutical Co., Ltd.

A Single-Arm, Open-Label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, Immunogenicity and Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Therapy in Patients With Severe Hemophilia A (Adults and Adolescents)

The indication for this product is to control and prophylaxis in patients with Hemophilia A (congenital Factor VIII deficiency):

The Primary Objective： To evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated patients with severe Hemophilia A.

Secondary Objectives： To evaluate the health-related quality of life, pharmacokinetic (PK) profiles, safety and immunogenicity of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated subjects with severe Hemophilia A.

Panoramica dello studio

Stato

Attivo, non reclutante

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

60

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Cina
      • Fuyang, Cina
        • Fuyang Hospital, Affiliated to Anhui Medical University
      • Fuzhou, Cina
        • Fujian Medical University Union Hospital
      • Guangzhou, Cina
        • Nanfang Hospital of Southern Medical University
      • Hefei, Cina
        • Anhui Provincial Hospital
      • Jinan, Cina
        • Jinan Central Hospital
      • Kunming, Cina
        • The Second Affiliated Hospital of Kunming Medical University
      • Nanning, Cina
        • The First Affiliated Hospital of Guangxi Medical University
      • Nantong, Cina
        • Affiliated Hospital of Nantong University
      • Nanyang, Cina
        • The First Affiliated Hospital of Nanyang Medical College
      • Qinghai, Cina
        • Qinghai Provincial People's Hospital
      • Taiyuan, Cina
        • The Second Hospital of Shanxi Medical University
      • Tangshan, Cina
        • North China University of Science and Technology Affiliated Hospital
      • Wenzhou, Cina
        • Wenzhou People's Hospital
      • Wuhan, Cina
        • Union Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology.
      • Wuxi, Cina
        • Affiliated Hospital of Jiangnan University
      • Xi'an, Cina
        • Xi'an Central Hospital
      • Zhengzhou, Cina
        • Henan Cancer Hospital
      • Zhengzhou, Cina
        • Zhengzhou People's Hospital
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, Cina
        • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

Inclusion Criteria:

1.12≤ age ≤65 year-old men; 2.Subjects with clinically confirmed severe hemophilia A, i.e. at screening (central laboratory testing) or previous medical records confirm: FⅧ activity < 1%; 3.Previous documented treatment with any recombinant and/or blood-derived coagulation factor Ⅷ products or cryoprecipitation products and dosed ≥150 exposure days (EDs≥150) ; 4.Normal prothrombin time (PT) or International Normalized Ratio (INR)<1.3; 5.Bleeding events were recorded in detail for at least 6 months prior to screening; 6.Fully understand and know about this study and sign informed consent to participate in the clinical study voluntarily, subject and/or their guardian can cooperate with them for bleeding treatment at home, and have the ability to complete all study procedures

Exclusion Criteria:

  1. Known or suspected allergy to the investigational drug or its excipients, including mouse or hamster proteins;
  2. Hypersensitivity or anaphylaxis after FⅧ or IgG2 injection in the past;
  3. 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;
  4. Von Willebrand factor (vWF) antigen test results were lower than the lower limit of normal value;
  5. Severe anemia at the screening stage (hemoglobin < 60 g/L);
  6. Platelet count during screening period < 100×109 /L;
  7. 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;
  8. Subjects with abnormal renal function: Creatinine clearance (Ccr) <50 ml/min (according to Cockcroft and Gault formula); or Serum creatinine (Cr) >1.5x ULN;
  9. Subjects 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);
  10. Subjects with coagulation dysfunction other than hemophilia A;
  11. Have a medical condition that may increase the risk of bleeding;
  12. A history of drug or alcohol abuse;
  13. Have a known mental disorder that may affect trial compliance;
  14. Subjects who have received transfusions of blood or blood components within 4 weeks prior to screening;
  15. Participants who had participated in other Interventional clinical trials within 1 month before screening;
  16. 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 subjects who need to be treated with anticoagulant or antiplatelet drugs or off-label maximum doses of SAID during clinical trials;
  17. 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.
  18. Subjects who have received emicizumab within 6 months prior to the first administration of study drug, or have previously received fitusiran (siRNA, brand name: CEPHEIN®) or gene therapy;
  19. Subjects who have received monoclonal antibody therapy, Fc fusion protein products, or intravenous immunoglobulin within 3 months prior to the first administration of study drug;
  20. Subjects who have undergone major surgery within 3 months prior to the first administration of study drug, or those who plan to receive surgery during the study period;
  21. Subjects who have received any standard half-life FⅧ preparations (e.g., Advate, Kovaltry, Octate, Recombinate, NovoEight, Anjiyin, etc.) within 3 days or 5 half-lives (whichever is longer) prior to the first administration of study drug; patients who have received any other extended half-life FⅧ preparations (e.g., Noxyte) within 4 days or 5 half-lives (whichever is longer) prior to the first administration of study drug;
  22. Study patients with fever, severe active bacterial or viral infection, and allergies within 2 weeks before the first administration of the drug;
  23. 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;
  24. 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);
  25. 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.);
  26. Have other serious medical conditions that the researchers said could not benefit from them
  27. Subjects deemed unsuitable by other investigators.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: N / A
  • Modello interventistico: Assegnazione di gruppo singolo
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: prophylactic treatment

Subjects in PK Subgroup receive a single and multiple dose of 50 IU/kg FRSW107 at Visit 1 and Visit 5, respectively. PK samples will be collected up to 72 hours after the start of administration.After completion of PK blood sampling for the first dose and prior to availability of the corresponding PK data, subjects may continue prophylactic treatment with FRSW107 at a dose of 50 IU/kg every 3 days until their PK data are obtained.Once the first-dose PK data of a subject are available, individualized prophylactic treatment with FRSW107 will be implemented based on the PK results. On the premise of maintaining a trough FVIII activity level of ≥1%, the investigator will determine the appropriate individualized prophylactic regimen for the subject. The recommended prophylactic dosing interval is Q3D, with an optional dose range of 25-50 IU/kg.

For subjects in the non-PK subgroup, the investigator will select the initial prophylactic dose within the recommended range of 25-50 IU/kg.
Droga: FRSW107

For subjects in the PK subgroup: they will receive a dose of 50 IU/kg at the first dose visit 1 to obtain preliminary pharmacokinetic (PK) data. After assessment by the investigator, individualized prophylactic treatment (25~50 IU/kg, Q3D) will be administered to maintain the trough concentration of FVIII activity at ≥1%.

For subjects not in the PK subgroup: they will receive prophylactic treatment at a dose of 25~50 IU/kg once every three days.

If a subject experiences a breakthrough bleeding episode requiring treatment, the investigator shall determine the appropriate dosage (recommended dose range: 20~50 IU/kg) and administration frequency.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
ABR
Lasso di tempo: 6 months
Annual rate of bleeding (ABR) during preventive treatment = Number of bleeding episode during the efficacy evaluation period/(number of treatment days /365.25)
6 months

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Safety Evaluation
Lasso di tempo: 6 months
Incidence of positive FⅧ inhibitor (key secondary endpoint)
6 months
Immunogenicity Evaluation
Lasso di tempo: 6 months
Incidence of positive anti-FRSW107 antibodies and anti-CHO antibodies; for subjects with positive anti-FRSW107 antibodies, additional testing for anti-rhFVIII antibodies shall be performed to assess their positive incidence.
6 months
Peak activity (Cmax)
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).

Applicable to PK Subgroup:

  1. Single administration: Peak activity (Cmax).
  2. Multiple administrations: Cmax.
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
Effective rate of hemostatic treatment
Lasso di tempo: 6 months
During the prophylactic treatment period, breakthrough hemostatic therapy was evaluated based on a four-level scoring scale (excellent, good, moderate, ineffective), with scores of "excellent" or "good" indicating efficacy.
6 months
Annualized rate of spontaneous bleeds and annualized rate of traumatic bleeds.
Lasso di tempo: 6 months
Annualized rate of spontaneous bleeds and annualized rate of traumatic bleeds.
6 months
Annualized Joint Bleed Rate (AJBR)
Lasso di tempo: 6 months

Annualized Joint Bleed Rate (AJBR), including overall AJBR, annualized rate of spontaneous joint bleeds and annualized rate of traumatic joint bleeds.

AJBR = Number of joint bleeds during efficacy evaluation period / (Number of treatment days / 365.25).
6 months
Number of target joints.
Lasso di tempo: 6 months
Number of target joints. Target joint definition: A joint with ≥3 spontaneous bleeds within any consecutive 6 months is defined as a target joint; a joint will no longer be classified as a target joint if it experiences ≤2 bleeds within any consecutive 12 months.
6 months
Dosing parameters of prophylactic treatment
Lasso di tempo: 6 months
Dosing parameters of prophylactic treatment: total cumulative dose during study, annual total dose, mean dose per prophylactic administration; administration frequency: total number of injections, mean annual injection frequency; dosing interval: mean interval between prophylactic administrations throughout the prophylaxis period.
6 months
Factor VIII incremental recovery and trough levels during prophylactic treatment.
Lasso di tempo: 6 months
Factor VIII incremental recovery and trough levels during prophylactic treatment.
6 months
Time interval between each bleeding episode and the prior prophylactic dose during prophylaxis.
Lasso di tempo: 6 months
Time interval between each bleeding episode and the prior prophylactic dose during prophylaxis.
6 months
Dosing parameters for rescue hemostatic treatment of breakthrough bleeds during prophylaxis
Lasso di tempo: 6 months
Dosing parameters for rescue hemostatic treatment of breakthrough bleeds during prophylaxis: mean dose, total cumulative dose and total number of administrations.
6 months
Hemophilia Joint Health Score version 2.1 (HJHS 2.1)
Lasso di tempo: 6 months
Hemophilia Joint Health Score version 2.1 (HJHS 2.1): total HJHS 2.1 score, individual domain subscores, and their respective changes from baseline.
6 months
EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L) and EuroQol Visual Analogue Scale (EQ VAS) .
Lasso di tempo: 6 months
EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L) and EuroQol Visual Analogue Scale (EQ VAS), together with their changes from baseline.The total healthy utility index score ranges of EQ-5D-5L from a minimum value of 0 points to a maximum value of 1 points,higher scores mean a better outcome.The total score ranges of EuroQol Visual Analogue Scale (EQ VAS) from a minimum value of 0 points to a maximum value of 100 points,higher scores mean a better outcome.
6 months
Incidence of insufficient therapeutic response
Lasso di tempo: 6 months
Incidence of insufficient therapeutic response.
6 months
time to peak (Tmax)
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).

Applicable to PK Subgroup:

  1. Single administration: time to peak (Tmax).
  2. Multiple administrations: Tmax.
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
area under the concentration-time curve from time zero to the last quantifiable time point (AUC₀-ₗₐₛₜ)
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).

Applicable to PK Subgroup:

  1. Single administration: area under the concentration-time curve from time zero to the last quantifiable time point (AUC₀-ₗₐₛₜ).
  2. Multiple administrations: AUC₀-ₗₐₛₜ.
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
elimination half-life (t₁/₂)
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
elimination half-life (t₁/₂)
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
incremental recovery
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
incremental recovery (calculated based on FⅧ Cmax measured after the end of infusion, unit: [IU/dL]/[IU/kg])
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
The time for FⅧ activity to decline to 15%, 5%, 3% and 1% .
Lasso di tempo: At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).
The time for FⅧ activity to decline to 15%, 5%, 3% and 1% respectively after study drug infusion.
At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Hangzhou Gensciences Biopharmaceutical Co., Ltd.

Investigatori

  • Cattedra di studio: Renchi Yang, PhD, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

12 giugno 2026

Completamento primario (Stimato)

9 marzo 2027

Completamento dello studio (Stimato)

21 giugno 2027

Date di iscrizione allo studio

Primo inviato

16 giugno 2026

Primo inviato che soddisfa i criteri di controllo qualità

29 giugno 2026

Primo Inserito (Effettivo)

6 luglio 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

4 settembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

2 settembre 2026

Ultimo verificato

1 giugno 2026

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • SS-107-III04

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

INDECISO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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