ong-term safety and efficacy of BIVV001 in Previously Treated Patients with hemophilia A

Phase 1

• Conditions: Hemophilia A; MedDRA version: 20.0Level: LLTClassification code: 10060612Term: Hemophilia A Class: 10010331; Therapeutic area: Not possible to specify

• Registration Number: CTIS2023-508929-27-00
• Lead Sponsor: Bioverativ Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-11-27
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 272

Inclusion Criteria

For participants rolling over into Arm A • Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study., For participants rolling over into Arm A: • Male or Female, For participants new to BIVV001 (Arm B and C) • Participants who have severe hemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A., For participants new to BIVV001 (Arm B and C) • Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged <6 years., For participants new to BIVV001 (Arm B and C) • Platelet count =100 000 cells/µL at screening., For participants new to BIVV001 (Arm B and C) • A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count >200 cells/mm³ and viral load of <400 000 copies/mL, For participants new to BIVV001 (Arm B and C) • Male, For participants new to BIVV001 (Arm B and C) • Only for Arm B: Chinese participants, For participants new to BIVV001 (Arm B and C) • Only for Arm C: planned major surgery within 6 months after Day 1.

Exclusion Criteria

For participants rolling over into Arm A • Positive inhibitor result, defined as =0.6 Bethesda units (BU)/mL, For participants new to BIVV001 (Arm B and Arm C) • Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening., For participants new to BIVV001 (Arm B and Arm C) • Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus [HCV] or HIV)., For participants new to BIVV001 (Arm B and Arm C) • Emicizumab use within the 20 weeks prior to screening., For participants new to BIVV001 (Arm B and Arm C) • Major surgery within 8 weeks prior to screening., For participants rolling over into Arm A • Participation in another study., For participants new to BIVV001 (Arm B and Arm C) • Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis., For participants new to BIVV001 (Arm B and Arm C) • Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening., For participants new to BIVV001 (Arm B and Arm C) Other known coagulation disorder(s) in addition to hemophilia A., For participants new to BIVV001 (Arm B and Arm C) • History of hypersensitivity or anaphylaxis associated with any FVIII product., For participants new to BIVV001 (Arm B and Arm C) • History of a positive inhibitor (to FVIII) test defined as =0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant., For participants new to BIVV001 (Arm B and Arm C) • Positive inhibitor test (FVIII) result, defined as =0.6 BU/mL at screening., For participants new to BIVV001 (Arm B and Arm C) • Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A;Secondary Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment., To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes., To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes., To evaluate the effect of BIVV001 prophylaxis on joint health outcomes., To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes., To evaluate the safety and tolerability of BIVV001 treatment., To assess the pharmacokinetic (PK) of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B)., To evaluate the efficacy of BIVV001 for perioperative management.;Primary end point(s): Number of participants with the occurrence of inhibitor development (neutralizing antibodies detected against factor VIII [FVIII])