← Back to Search

Gene Therapy

Gene Therapy for Hemophilia A (GENEr8-3 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

Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history, at the time of signing the informed consent

Must have been on prophylactic hemophilia therapy for at least 12 months prior to study entry

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 52

Awards & highlights

GENEr8-3 Trial Summary

This trial will test a new gene therapy for hemophilia A, given with prophylactic corticosteroids.

Who is the study for?

This trial is for adult males with severe hemophilia A, who have been on prophylactic therapy for at least a year and have had significant exposure to FVIII treatments. They must not have any history of inhibitors to FVIII and agree to use contraception post-infusion. Those with antibodies against the treatment vector, active infections, immune disorders, or unrelated bleeding disorders cannot participate.Check my eligibility

What is being tested?

The study tests valoctocogene roxaparvovec's effectiveness and safety in treating severe hemophilia A when used alongside preventive steroids. It aims to see if this gene therapy can reduce bleeding episodes by providing a functional copy of the faulty gene causing hemophilia.See study design

What are the potential side effects?

Potential side effects may include reactions related to the body's immune response against the viral vector used in gene therapy or complications from steroid use such as increased infection risk, mood changes, elevated blood sugar levels, and possible organ inflammation.

GENEr8-3 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am a male over 18 with severe hemophilia A.

Select...

I have been on preventive treatment for hemophilia for at least a year.

Select...

I have been treated with FVIII or cryoprecipitate for at least 150 days.

GENEr8-3 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.

Secondary outcome measures

Change From Baseline in Annualized Utilization of Exogenous FVIII Replacement Therapy in EEP

Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 52

Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 52

+4 more

GENEr8-3 Trial Design

1Treatment groups

Experimental Treatment

Group I: valoctocogene roxaparvovecExperimental Treatment1 Intervention

Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor

156 Previous Clinical Trials

190,703 Total Patients Enrolled

11 Trials studying Hemophilia A

2,012 Patients Enrolled for Hemophilia A

Medical Monitor, MDStudy DirectorBioMarin Pharmaceutical

72 Previous Clinical Trials

18,054 Total Patients Enrolled

11 Trials studying Hemophilia A

344 Patients Enrolled for Hemophilia A

Media Library

Valoctocogene Roxaparvovec (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04323098 — Phase 3

Hemophilia A Research Study Groups: valoctocogene roxaparvovec

Hemophilia A Clinical Trial 2023: Valoctocogene Roxaparvovec Highlights & Side Effects. Trial Name: NCT04323098 — Phase 3

Valoctocogene Roxaparvovec (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04323098 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What does the research say about valoctocogene roxaparvovec thus far?

"valoctocogene roxaparvovec was first studied in 2015 at The Royal London Hospital. As of now, there are no completed clinical trials. However, there are 6 active trials, many of which are based in Columbus, Ohio."

Answered by AI

Could you please tell me about the risks associated with valoctocogene roxaparvovec?

"Valoctocogene roxaparvovec is considered safe, as it has passed multiple rounds of testing with positive results."

Answered by AI

How many people are included in this clinical research?

"This trial has completed recruitment for participants. The original posting was on November 10th, 2020 and the most recent update to the listing was on October 13th, 2022. For patients searching for other studies, 91 trials are actively recruiting patients with hemophilia A while 6 different valoctocogene roxaparvovec trials are also looking for volunteers."

Answered by AI

Is this a groundbreaking clinical trial?

"Valoctocogene roxaparvovec is being trialed in 6 separate studies that are taking place across 13 countries and 15 cities. The first study for valoctocogene roxaparvovec was held in 2015. The study, sponsored by BioMarin Pharmaceutical, involved 15 participants and completed its Phase 1 & 2 drug approval stage. Since 2015 years, 0 trials have been completed."

Answered by AI

Are investigators currently looking for more participants for this research project?

"This study is not currently recruiting patients. However, this may change in the future as the study was last updated on October 13th, 2020. If you are searching for other studies, 91 trials are actively seeking patients with hemophilia A and 6 studies involving valoctocogene roxaparvovec are also looking for participants."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

2020. "Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04323098.

All > Hemophilia A > Hemophilia