CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 15 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.

Search/NCT02576795
NCT02576795Phase 2Completed
CompareCT.gov

A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A

BioMarin Pharmaceutical·interventional·Posted Oct 15, 2015·Updated Apr 10, 2025

In Brief

A Phase 2 clinical trial evaluating valoctocogene roxaparvovec for Severe Haemophilia A. Completed, enrolled 15 participants across 5 sites.

Detailed Summary

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy vector in participants with severe haemophilia A.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSevere Haemophilia A
CountriesUnited Kingdom
Collaborators--

Timeline

Phase 2CompletedFinished
20162017201820192020202120222023202420252026
First PostedOct 15, 2015
Enrollment StartSep 28, 2015
Primary CompletionFeb 14, 2024
TodayJul 2, 2026
Enrollment to primary: 8.4 yearsPosted 10.7 years ago

Interventions

valoctocogene roxaparvovecbiological

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Severe Hemophilia A