← Back to Search

Gene Therapy

Valoctocogene Roxaparvovec for Hemophilia A (GENEr8-2 Trial)

Phase 3
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up weeks 5 though week 52
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial will test whether BMN 270 is effective at raising FVIII activity and reducing the need for exogenous replacement therapy and the number of bleeding episodes.

Eligible Conditions
  • Hemophilia A

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 5 though week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 5 though week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change of the Median Factor VIII (FVIII) Activity
Secondary study objectives
Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy

Side effects data

From 2023 Phase 3 trial • 1 Patients • NCT03392974
100%
Myalgia
100%
Alanine aminotransferase increased
100%
Pyrexia
100%
Nasopharyngitis
100%
Hepatocellular injury
100%
Oropharyngeal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Total
BMN 270 4E13 vg/kg

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Valoctocogene Roxaparvovec Open LabelExperimental Treatment1 Intervention
Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Valoctocogene roxaparvovec
Not yet FDA approved

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
159 Previous Clinical Trials
115,049 Total Patients Enrolled
11 Trials studying Hemophilia A
1,130 Patients Enrolled for Hemophilia A
Medical Director, MDStudy DirectorBioMarin Pharmaceutical
80 Previous Clinical Trials
16,491 Total Patients Enrolled
3 Trials studying Hemophilia A
190 Patients Enrolled for Hemophilia A
~0 spots leftby Dec 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top