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Gene Therapy

Gene Therapy for Hemophilia A (GENEr8-INH Trial)

Phase 1 & 2
Recruiting
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 60 months
Awards & highlights

GENEr8-INH Trial Summary

This trial will study a new gene therapy for hemophilia A, which is a bleeding disorder. The trial will have two parts, A and B, studying patients with and without a history of inhibitors to factor VIII, respectively.

Who is the study for?
This trial is for adult males with severe Hemophilia A who have had inhibitors to Factor VIII. They must not drink alcohol for a year post-treatment, use effective contraception, and cannot have other bleeding disorders or significant organ dysfunction. Part A includes those currently with inhibitors; Part B is for those with a past history of inhibitors.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Valoctocogene Roxaparvovec in two groups: one with active Factor VIII inhibitors (Part A) and another that previously had these inhibitors but now tolerates FVIII (Part B).See study design
What are the potential side effects?
Potential side effects may include immune reactions to the treatment, liver issues due to viral vector used in gene therapy, injection site reactions, headaches, fatigue, muscle pain and possibly new inhibitor development.

GENEr8-INH Trial Eligibility Criteria

Inclusion Criteria

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

GENEr8-INH Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
Secondary outcome measures
A change in Factor VIII inhibitor titer (Part A) after administration of BMN 270.
Absence of recurrence of Factor VIII inhibitors (Part B) after administration of BMN 270.
Change in the annualized number of bleeding episodes requiring exogenous hemophilia therapy after administration of BMN 270.
+2 more

GENEr8-INH Trial Design

1Treatment groups
Experimental Treatment
Group I: Valoctocogene roxaparvovec Open LabelExperimental Treatment1 Intervention
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg in Active Inhibitor Population (Part A) and Prior Inhibitor Population (Part B).

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Hemophilia A primarily aim to replace or bypass the deficient factor VIII (FVIII) to prevent or control bleeding episodes. Recombinant factor VIII products directly replace the missing FVIII, allowing the blood to clot properly. Bypassing agents like activated prothrombin complex concentrate (aPCC) and recombinant factor VIIa (rFVIIa) work by activating the clotting cascade downstream of FVIII, effectively bypassing the need for FVIII. Gene therapy, such as valoctocogene roxaparvovec, introduces a functional FVIII gene into the patient's cells, enabling the body to produce its own FVIII. This approach has the potential to provide a long-term solution by addressing the underlying genetic cause of Hemophilia A. These treatments are crucial for managing bleeding risks and improving the quality of life for patients with Hemophilia A.

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
157 Previous Clinical Trials
189,991 Total Patients Enrolled
11 Trials studying Hemophilia A
1,200 Patients Enrolled for Hemophilia A
Medical Monitor, MDStudy DirectorBioMarin Pharmaceutical
72 Previous Clinical Trials
18,056 Total Patients Enrolled
11 Trials studying Hemophilia A
346 Patients Enrolled for Hemophilia A

Media Library

Valoctocogene Roxaparvovec (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04684940 — Phase 1 & 2
Valoctocogene Roxaparvovec (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04684940 — Phase 1 & 2
Hemophilia A Research Study Groups: Valoctocogene roxaparvovec Open Label
Hemophilia A Clinical Trial 2023: Valoctocogene Roxaparvovec Highlights & Side Effects. Trial Name: NCT04684940 — Phase 1 & 2
~12 spots leftby Feb 2029
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