Trial Radar
Sign in

NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

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

NCT04759131

CompletedPhase 3

Sponsor: Bioverativ, a Sanofi company

Conditions: Hemophilia A

Interventions: efanesoctocog alfa (BIVV001)

Countries: United States, Australia, Canada, France, Germany, Hungary, Ireland, Italy

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.

Eligibility overview

Sex: MALE

Age: — to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility 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 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.

Exclusion criteria:

* 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.
Locations (40)
  • Los Angeles, California, United States
  • Gainesville, Florida, United States
  • Atlanta, Georgia, United States
  • Chicago, Illinois, United States
  • Iowa City, Iowa, United States
  • New York, New York, United States
  • Greenville, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Columbus, Ohio, United States
  • Milwaukee, Wisconsin, United States
  • Westmead, New South Wales, Australia
  • South Brisbane, Queensland, Australia
  • Hamilton, Ontario, Canada
  • London, Ontario, Canada
  • Ottawa, Ontario, Canada
  • Toronto, Ontario, Canada
  • Bron, France
  • Le Kremlin-Bicêtre, France
  • Lille, France
  • Frankfurt am Main, Germany
  • München, Germany
  • Pécs, Hungary
  • Dublin, Ireland
  • Milan, Italy
  • Naples, Italy
  • Amsterdam, Netherlands
  • Utrecht, Netherlands
  • Esplugues de Llobregat, Catalunya [Cataluña], Spain
  • Madrid, Madrid, Comunidad de, Spain
  • Malmö, Sweden
  • + 10 more on CT.gov