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NCT04759131 · ClinicalTrials.gov registry record · Phase 3
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
A Phase 3 study of Hemophilia A, sponsored by Bioverativ, a Sanofi company.
- Completed
- Registry status
- Phase 3
- Development phase
- 74
- Enrollment target
- 20
- Study locations
NCT04759131: Completed Phase 3 study of Hemophilia A, sponsored by Bioverativ, a Sanofi company.
NCT04759131 is a Phase 3 study of Hemophilia A that has completed, run by Bioverativ, a Sanofi company. The registered enrollment target is 74 participants, below the 90-participant average among 42 other Hemophilia A trials with a reported enrollment target (18% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04759131, a Phase 3 study of Hemophilia A, has completed, sponsored by Bioverativ, a Sanofi company.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 74 participants
- Enrollment target
- 20
- Study locations
Study 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.
Primary Outcome
Inhibitor development was defined as an inhibitor result of greater than or equal to (\>=0.6) Bethesda units (BU/mL) that was confirmed by a second test result from a separate sample, drawn 2 to 4 weeks following the date when the original sample was drawn. Both tests must have been performed by the central laboratory using the Nijmegen modified Bethesda assay.
Conditions Studied
Interventions
- DRUG efanesoctocog alfa (BIVV001)
Study Locations (20)
Ontario
- Investigational Site Number :1240004 - Hamilton
- Investigational Site Number :1240003 - London
- Investigational Site Number :1240002 - Ottawa
- Investigational Site Number :1240001 - Toronto
Other
- Investigational Site Number :2500004 - Bron
- Investigational Site Number :2500001 - Le Kremlin-Bicêtre
- Investigational Site Number :2500003 - Lille
- Investigational Site Number :2760001 - Frankfurt am Main
Ohio
- Cincinnati Children's Hospital Medical Center-Site Number:8400008 - Cincinnati
- Childrens Hospital Of Columbus-Site Number:8400012 - Columbus
California
- Children's Hospital Los Angeles-Site Number:8400006 - Los Angeles
Florida
- University of Florida-Site Number:8400009 - Gainesville
Georgia
- Children's Healthcare of Atlanta-Site Number:8400019 - Atlanta
Illinois
- Rush University Medical Center-Site Number:8400001 - Chicago
Iowa
- University of Iowa Hospitals and Clinics-Site Number:8400002 - Iowa City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 74 participants |
| Start Date | 2021-02-19 |
| Est. Completion | 2023-01-18 |
| Phase | Phase 3 |
What the finished NCT04759131 record still lists
NCT04759131 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 74 participants, a relatively small participant target, below the 90-participant average among 42 other Hemophilia A trials with a reported enrollment target (18% lower).
The record links to 1 condition, with Hemophilia A appearing as the primary indexed condition, and to 1 intervention - of which efanesoctocog alfa (BIVV001) is the first listed.
NCT04759131 names 20 study sites across 13 states, led by Ontario, Other, Ohio.
Frequently Asked Questions
What is clinical trial NCT04759131 about?
NCT04759131 is a clinical study titled "Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A". 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 evaluat...
What is the current status of trial NCT04759131?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 74 participants. The study started on 2021-02-19. Estimated completion is 2023-01-18.
What conditions does trial NCT04759131 study?
This clinical trial studies the following conditions: Hemophilia A.
What interventions are being tested in trial NCT04759131?
The interventions under investigation include: efanesoctocog alfa (BIVV001) (DRUG).
Who is sponsoring clinical trial NCT04759131?
This trial is sponsored by Bioverativ, a Sanofi company, which has 14 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04759131 being conducted?
This trial has 20 study locations across California, Florida, Georgia, Illinois, Iowa. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hemophilia A
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04759131's enrollment target sits among peer trials
74 19th of 42 higher than 23 of 42 other Hemophilia A trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Hemophilia A trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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