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Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT04684940 · View on ClinicalTrials.gov ↗
Study Summary
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
Conditions Studied
Interventions
- BIOLOGICAL Valoctocogene roxaparvovec
Study Locations (9)
Other
- Hemocentro Da UNICAMP — Campinas
- Arthur De Siqueira Cavalcanti Hematology State Institute — Rio de Janeiro
- Chaim Sheba Medical Center — Ramat Gan
- Kyung Hee University Hospital at Gangdong — Seoul
- Kaohsiung Medical University - Chung-Ho Memorial Hospital — Kaohsiung City
- Taichung Veterans General Hospital — Taichung
- National Taiwan University Hospital — Taipei
- Ege University School of Medicine — Izmir
California
- Children's Hospital Los Angeles — Los Angeles
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2020-12-10 |
| Est. Completion | 2029-04 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04684940
The ClinicalTrials.gov registry entry for NCT04684940 describes a study currently listed as active not recruiting. It is categorized as Phase 1, 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 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 31 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Hemophilia A With Inhibitor appearing as the primary indexed condition, and to 1 intervention — of which Valoctocogene roxaparvovec 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: NCT04684940 reports 9 study locations spanning 2 distinct geographic areas — top geographies include Other, California. 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 NCT04684940 about?
NCT04684940 is a clinical study titled "Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors". This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhib...
What is the current status of trial NCT04684940?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2020-12-10. Estimated completion is 2029-04.
What conditions does trial NCT04684940 study?
This clinical trial studies the following conditions: Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04684940?
The interventions under investigation include: Valoctocogene roxaparvovec (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04684940?
This trial is sponsored by BioMarin Pharmaceutical, which has 31 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04684940 being conducted?
This trial has 9 study locations across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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