10 Participants Needed

Gene Therapy for Hemophilia A

(GENEr8-INH Trial)

Recruiting at 16 trial locations
TS
Overseen ByTrial Specialist
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a gene therapy called valoctocogene roxaparvovec in patients with severe haemophilia A who have developed antibodies against standard treatments. The therapy aims to provide a working version of the gene needed for proper blood clotting.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently undergoing immune tolerance induction therapy or prophylaxis with FVIII, you cannot participate in Part A of the study.

Is Valoctocogene Roxaparvovec safe for humans?

Valoctocogene Roxaparvovec, a gene therapy for severe hemophilia A, has been studied for safety in clinical trials. It has received conditional approval in the EU, indicating that it has been deemed safe enough for use in adults with specific conditions, although ongoing monitoring and further studies are likely required.12345

How does the treatment Valoctocogene Roxaparvovec for Hemophilia A differ from other treatments?

Valoctocogene Roxaparvovec is unique because it is a gene therapy designed to provide a long-term solution by delivering a functional copy of the gene responsible for producing factor VIII, a protein essential for blood clotting, potentially reducing or eliminating the need for regular factor VIII infusions.678910

What data supports the effectiveness of the treatment Valoctocogene Roxaparvovec for Hemophilia A?

Valoctocogene Roxaparvovec has shown sustained clinical benefits in people with severe Hemophilia A, with significant increases in factor VIII levels and a reduction in bleeding episodes, as demonstrated in phase III clinical trials. It has been approved by the European Commission and FDA, indicating its effectiveness and safety for treating Hemophilia A.111121314

Who Is on the Research Team?

MM

Medical Monitor, MD

Principal Investigator

BioMarin Pharmaceutical

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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.
I have been on hemophilia treatment in the past year.
Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.
See 2 more

Exclusion Criteria

Detectable pre-existing antibodies to the AAV5 capsid.
I have a bleeding disorder that is not hemophilia A.
I am receiving or plan to receive immune therapy for FVIII during the study.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

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

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

60 months

What Are the Treatments Tested in This Trial?

Interventions

  • Valoctocogene Roxaparvovec
Trial Overview 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).
How Is the Trial Designed?
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).

Find a Clinic Near You

Who Is Running the Clinical Trial?

BioMarin Pharmaceutical

Lead Sponsor

Trials
162
Recruited
115,000+
Alexander Hardy profile image

Alexander Hardy

BioMarin Pharmaceutical

Chief Executive Officer since 2023

MBA from INSEAD

Greg Friberg profile image

Greg Friberg

BioMarin Pharmaceutical

Chief Medical Officer

MD from New York Medical College

Published Research Related to This Trial

Recent advancements in hemophilia treatment include monoclonal antibodies that mimic factor VIII activity, providing effective therapy for all hemophilia A phenotypes, including those with inhibitors, and improving patient quality of life with safer and longer-lasting effects.
Gene therapy, particularly using CRISPR/Cas technology, shows promise for potentially curing hemophilia by permanently editing genes, with ongoing developments aimed at minimizing risks and enhancing effectiveness.
Current Therapies in Hemophilia: From Plasma-Derived Factor Modalities to CRISPR/Cas Alternatives.Jair Lara-Navarro, I., Rebeca Jaloma-Cruz, A.[2022]
Gene therapies like Roctavian® and Hemgenix® have been approved for treating severe hemophilia A and B, respectively, showing significant increases in clotting factor levels and reductions in bleeding episodes based on phase III clinical trial data.
While most patients experienced short-term liver inflammation that was manageable with immune suppression, there remains variability in treatment response, and further research is needed to determine the long-term stability of factor expression and the potential for a permanent cure.
Hemophilia Gene Therapy: The End of the Beginning?De Wolf, D., Singh, K., Chuah, MK., et al.[2023]
Gene therapy for hemophilia, specifically with products like valoctocogene roxaparvovec for hemophilia A and etranacogene dezaparvovec for hemophilia B, is nearing approval and represents a significant advancement in treatment, allowing for a one-time infusion that leads to the body's own production of missing clotting factors.
Clinical observations indicate that gene therapy can provide sustained benefits for over 5 years after a single treatment, with manageable side effects such as temporary increases in liver enzymes, suggesting a promising safety profile for these therapies.
Gene therapy for hemophilia.Nathwani, AC.[2023]

Citations

Hemophilia gene therapy: first, do no harm. [2023]
Current Therapies in Hemophilia: From Plasma-Derived Factor Modalities to CRISPR/Cas Alternatives. [2022]
Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A. [2022]
Hemophilia Gene Therapy: The End of the Beginning? [2023]
Gene therapy for hemophilia. [2023]
Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B. [2023]
Valoctocogene Roxaparvovec: First Approval. [2022]
Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis. [2023]
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A. [2023]
10.United Statespubmed.ncbi.nlm.nih.gov
Intravenous administration of retroviral replicating vector, Toca 511, demonstrates therapeutic efficacy in orthotopic immune-competent mouse glioma model. [2022]
Clinical applications of attenuated MVA poxvirus strain. [2013]
Modified vaccinia virus Ankara as a vector for suicide gene therapy. [2020]
13.United Statespubmed.ncbi.nlm.nih.gov
Long-term outcome of phase I/II clinical trial of Ad-OC-TK/VAL gene therapy for hormone-refractory metastatic prostate cancer. [2018]
14.United Statespubmed.ncbi.nlm.nih.gov
Vaccination of colorectal cancer patients with modified vaccinia Ankara delivering the tumor antigen 5T4 (TroVax) induces immune responses which correlate with disease control: a phase I/II trial. [2022]
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