Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (XTEND-Kids)

Bioverativ

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Drug: efanesoctocog alfa (BIVV001)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04759131

017464 (Other Identifier)

U1111-1244-0558 (Other Identifier)

2020-000769-18 (EudraCT Number)

EFC16295

Details and patient eligibility

About

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.

Full description

Study duration per participants was approximately 60 weeks (maximum 8 weeks for screening and 52 weeks of treatment).

All participants completing or remaining at the end of study were offered participation in the planned extension trial.

Enrollment: 74 patients

Sex: Male

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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74 participants in 2 patient groups

BIVV001: Participants aged <6 Years

Experimental group

Description:

Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks.

Treatment:

Drug: efanesoctocog alfa (BIVV001)

BIVV001: Participants aged 6 to <12 Years

Experimental group

Description:

Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks.

Treatment:

Drug: efanesoctocog alfa (BIVV001)