MedTrace Logo

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

NCT03520712 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-08-22

Study results available
· View outcomes & findings →

Summary

This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.

Conditions

Interventions

BIOLOGICAL

Valoctocogene Roxaparvovec

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

Sponsors & Collaborators

Principal Investigators

  • Medical Director, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-24
Primary Completion
2024-08-07
Completion
2024-08-07
FDA Drug
Yes

Countries

  • South Africa
  • South Korea
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03520712 on ClinicalTrials.gov