MedTrace Logo

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

NCT04684940 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-07-14

No results posted yet for this 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

  • Hemophilia A With Inhibitor
  • Hemophilia A With Anti Factor VIII

Interventions

BIOLOGICAL

Valoctocogene roxaparvovec

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

Sponsors & Collaborators

Principal Investigators

  • Medical Monitor, 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
2020-12-10
Primary Completion
2029-04-30
Completion
2029-04-30
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Israel
  • South Korea
  • Taiwan
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

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 NCT04684940 on ClinicalTrials.gov