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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
NCT04431726 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 55 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Hoffmann-La Roche, which has 758 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04431726 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Other, Ontario, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04431726?
The interventions under investigation include: Emicizumab (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04431726?
This trial is sponsored by Hoffmann-La Roche, which has 758 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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