At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 1 enrolled
Drug / intervention
Valoctocogene Roxaparvovecbiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions
In Brief
A Phase 3 clinical trial evaluating Valoctocogene Roxaparvovec for Hemophilia A. Completed, enrolled 1 participant across 1 site.
Detailed Summary
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsHemophilia A
CountriesUnited States
Collaborators--
Timeline
Phase 3CompletedFinished
201820192020202120222023202420252026
First PostedJan 2018
Enrollment StartMar 2018
Primary CompletionMay 2019
Study CompletionJun 2023
TodayJul 2026
First PostedJan 8, 2018
Enrollment StartMar 14, 2018
Primary CompletionMay 22, 2019
Study CompletionJun 5, 2023
TodayJul 2, 2026
Enrollment to primary: 1.2 yearsPosted 8.5 years ago
Interventions
Valoctocogene Roxaparvovecbiological
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A