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ClinicalTrials.gov public records Last synced Nov 14, 2026, 10:17 PM EST

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Showing 1–4 of 4 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Valoctocogene Roxaparvovec Clear all
Completed Phase 3 Interventional Results available

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
15
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2025 · Synced Nov 14, 2026, 10:17 PM EST
Completed Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Sacramento, California • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Nov 14, 2026, 10:17 PM EST
Completed Phase 1Phase 2 Interventional

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

NCT04684940
View directory record Official record
Conditions
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Interventions
Valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Nov 14, 2026, 10:17 PM EST
Completed Phase 3 Interventional Results available

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

NCT03392974
View directory record Official record
Conditions
Hemophilia A
Interventions
Valoctocogene Roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Nov 14, 2026, 10:17 PM EST