Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

• Status: Recruiting
• Trial end date: 2023-02-01
• Target enrollment: 0
• Participant gender: Male

Summary

Primary Objective:

• To evaluate the safety of BIVV001 in previously treated pediatric subjects with hemophilia A

Secondary Objectives:

• 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 efficacy of BIVV001 for perioperative management

• To evaluate the safety and tolerability of BIVV001 treatment

• To assess the pharmacokinetics (PK) of BIVV001

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioverativ, a Sanofi company; Sanofi

Criteria

Inclusion criteria :

• Participant must be younger than 12 years of age, at the time of signing the informed consent

• Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII 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.

• Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs for patients aged 6-11 years and above 50 EDs for patients aged <6 years

• Weight above or equal to 10 kg.

Exclusion criteria:

• History of hypersensitivity or anaphylaxis associated with any FVIII product.

• 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.

• Positive inhibitor test result, defined as ≥0.6 BU/mL at Screening.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.