Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04431726
The ClinicalTrials.gov registry entry for NCT04431726 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 55 participants, a figure that helps gauge the scale of data the investigators plan to collect, roughly in line with the 57-participant average among 4 other Severe Hemophilia A trials with a reported enrollment target. The listed sponsor is Hoffmann-La Roche, which has 909 total studies on file at ClinicalTrials.gov.
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 reports 20 study locations spanning 13 distinct geographic areas - top geographies include 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.
Learn More About Clinical Trials
Similar trials for Severe 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.
-
Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
COMPLETED · Phase 3
-
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
RECRUITING · Phase 1
-
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
RECRUITING · Phase 4
-
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
ACTIVE NOT RECRUITING · Phase 4
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.