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

Completed · Phase 3

Conditions studied: Hemophilia A

In brief

Primary Objective: \- To evaluate the safety of BIVV001 in previously treated pediatric participants 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.

Key facts

Study ID

NCT04759131

Run by

Bioverativ, a Sanofi company

People needed

74

Starts

2021-02-19

Expected to finish

2023-01-18

Last updated by the study team

2025-09-11

Who can join

Age: any, up to 11. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Participant must be younger than 12 years of age, at the time of signing the informed consent.
• Severe hemophilia A defined as <1 international units per deciliter (IU/dL) (<1 percent [%]) endogenous Factor VIII (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 exposure days (EDs) for participants aged 6 to <12 years and above 50 EDs for participants aged <6 years.
• Weight above or equal to 10 kg.

You may not qualify if…

• History of hypersensitivity or anaphylaxis associated with any FVIII product.
• History of a positive inhibitor (to FVIII) test defined as greater than or equal to (>=) 0.6 Bethesda units (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 would not exclude the participant.
• Positive inhibitor test result, defined as >=0.6 BU/mL at Screening.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Children's Hospital Los Angeles-Site Number:8400006 — Los Angeles, California, United States
• University of Florida-Site Number:8400009 — Gainesville, Florida, United States
• Children's Healthcare of Atlanta-Site Number:8400019 — Atlanta, Georgia, United States
• Rush University Medical Center-Site Number:8400001 — Chicago, Illinois, United States
• University of Iowa Hospitals and Clinics-Site Number:8400002 — Iowa City, Iowa, United States
• NY Presbyterian - Weill Cornell Medical Center-Site Number:8400020 — New York, New York, United States
• East Carolina University/Brody Medical Sciences Building-Site Number:8400015 — Greenville, North Carolina, United States
• Cincinnati Children's Hospital Medical Center-Site Number:8400008 — Cincinnati, Ohio, United States
• Childrens Hospital Of Columbus-Site Number:8400012 — Columbus, Ohio, United States
• Children's Hospital Of Wisconsin-Site Number:8400005 — Milwaukee, Wisconsin, United States
• Investigational Site Number :0360001 — Westmead, New South Wales, Australia
• Investigational Site Number :0360002 — South Brisbane, Queensland, Australia
• Investigational Site Number :1240004 — Hamilton, Ontario, Canada
• Investigational Site Number :1240003 — London, Ontario, Canada
• Investigational Site Number :1240002 — Ottawa, Ontario, Canada
• Investigational Site Number :1240001 — Toronto, Ontario, Canada
• Investigational Site Number :2500004 — Bron, France
• Investigational Site Number :2500001 — Le Kremlin-Bicêtre, France
• Investigational Site Number :2500003 — Lille, France
• Investigational Site Number :2760001 — Frankfurt am Main, Germany
• Investigational Site Number :2760002 — München, Germany
• Investigational Site Number :3480005 — Pécs, Hungary
• Investigational Site Number :3720001 — Dublin, Ireland
• Investigational Site Number :3800001 — Milan, Italy
• Investigational Site Number :3800002 — Naples, Italy

Full record on ClinicalTrials.gov

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