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

NCT04759131 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 74

Last updated 2025-09-11

Study results available
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Summary

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.

Conditions

Interventions

DRUG

efanesoctocog alfa (BIVV001)

Pharmaceutical form: solution for injection Route of administration: IV

Sponsors & Collaborators

  • Bioverativ, a Sanofi company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
11 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-02-19
Primary Completion
2023-01-18
Completion
2023-01-18
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Hungary
  • Ireland
  • Italy
  • Netherlands
  • Spain
  • Sweden
  • Switzerland
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04759131 on ClinicalTrials.gov