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NCT04431726 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
A Phase 3 study of Severe Hemophilia A, sponsored by Hoffmann-La Roche.
- Active
- Registry status
- Phase 3
- Development phase
- 55
- Enrollment target
- 20
- Study locations
NCT04431726: Active Phase 3 study of Severe Hemophilia A, sponsored by Hoffmann-La Roche.
NCT04431726 is a Phase 3 study of Severe Hemophilia A that is active but no longer recruiting, run by Hoffmann-La Roche. The registered enrollment target is 55 participants, roughly in line with the 57-participant average among 4 other Severe Hemophilia A trials with a reported enrollment target. The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04431726, a Phase 3 study of Severe Hemophilia A, is active but no longer recruiting, sponsored by Hoffmann-La Roche.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 55 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A (intrinsic factor VIII \[FVIII\] level \<1%) without FVIII inhibitors. The study is designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at 3 milligrams per kilogram of body weight (mg/kg) once every 2 weeks (Q2W) for 52 weeks. After 1 year of treatment, participants will continue to receive emicizumab (1.5 mg/kg once every week \[QW\], 3 mg/kg Q2W or 6 mg/kg once every 4 weeks \[Q4W\]) over a 7-year long-term follow-up period under this study frame.
Primary Outcome
The number of treated bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours fro
Conditions Studied
Interventions
- DRUG Emicizumab
Study Locations (20)
Other
- Medizinische Universität Wien - Vienna
- Cliniques Universitaires St-Luc - Brussels
- UZ Leuven Gasthuisberg - Leuven
- Groupe Hospitalier Necker Enfants Malades - Paris
- Universitätsklinikum Bonn - Bonn
- Hämophilie-Zentrum Rhein Main GmbH - Mörfelden-Walldorf
- Sheba Medical Center - National Hemophilia Center - Tel Litwinsky
Ontario
- Children's Hospital of Eastern Ontario - Ottawa
- The Hospital for Sick Children - Toronto
Arizona
- Phoenix Children's Hospital - Phoenix
California
- Children's Hospital Los Angeles - Los Angeles
Colorado
- University of Colorado Denver, Children's Hospital - Aurora
Louisiana
- Tulane University Health Sciences Center - New Orleans
Michigan
- University of Michigan - Ann Arbor
Washington
- Seattle Children's Hospital - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 55 participants |
| Start Date | 2021-02-04 |
| Est. Completion | 2030-05-18 |
| Phase | Phase 3 |
What the registry record for NCT04431726 still lists
NCT04431726 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 55 participants, a relatively small participant target, roughly in line with the 57-participant average among 4 other Severe Hemophilia A trials with a reported enrollment target.
The record links to 1 condition, with Severe Hemophilia A appearing as the primary indexed condition, and to 1 intervention - of which Emicizumab is the first listed.
NCT04431726 names 20 study sites across 13 states, led by Other, Ontario, Arizona.
Frequently Asked Questions
What is clinical trial NCT04431726 about?
NCT04431726 is a clinical study titled "A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors". This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A (intrinsic factor VIII \[FVIII\] level \<1%) without FVIII inhibitors. The st...
What is the current status of trial NCT04431726?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 55 participants. The study started on 2021-02-04. Estimated completion is 2030-05-18.
What conditions does trial NCT04431726 study?
This clinical trial studies the following conditions: Severe Hemophilia A.
What interventions are being tested in trial NCT04431726?
The interventions under investigation include: Emicizumab (DRUG).
Who is sponsoring clinical trial NCT04431726?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04431726 being conducted?
This trial has 20 study locations across Arizona, California, Colorado, Louisiana, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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