Safety, efficacy and PK of BIVV001 in pediatric patients with hemophilia A
- Conditions
- Hemophilia AMedDRA version: 20.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2020-000769-18-Outside-EU/EEA
- Lead Sponsor
- Bioverativ Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- Not specified
- 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- 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.
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 safety of BIVV001 in previously treated pediatric subjects with hemophilia A;Secondary Objective: - To evaluate the efficacy of BIVV001 as a prophylaxis treatment<br>- To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes<br>- To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes<br>- To evaluate the effect of BIVV001 prophylaxis on joint health outcomes<br>- To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes<br>- To evaluate the efficacy of BIVV001 for perioperative management<br>- To evaluate the safety and tolerability of BIVV001 treatment<br>- To assess the pharmacokinetics (PK) of BIVV001 <br>;Primary end point(s): Occurrence of inhibitor development ;Timepoint(s) of evaluation of this end point: baseline to 52 weeks
- Secondary Outcome Measures
Name Time Method