← Back to Search

Gene Therapy

Gene Therapy for Hemophilia A (BMN 270-301 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
No previous documented history of a detectable FVIII inhibitor, and results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least one week apart within the past 12 months
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

BMN 270-301 Trial Summary

This trial will test a new medication for hemophilia A and measure its effectiveness compared to current treatments.

Who is the study for?
This trial is for adult males with Hemophilia A who have very low levels of Factor VIII and have been on standard treatment for at least a year. They must not have had inhibitors to Factor VIII, significant liver issues, other bleeding disorders, or certain infections like HIV or hepatitis.Check my eligibility
What is being tested?
The study tests Valoctocogene Roxaparvovec (BMN 270), a gene therapy compared to standard Factor VIII prophylaxis. It measures the number of bleeds without FVIII treatment over two years and checks how much external FVIII is used after receiving BMN 270.See study design
What are the potential side effects?
Potential side effects are not detailed here but typically include immune reactions to the therapy, increased risk of blood clots due to changes in clotting factors, and possible liver-related issues.

BMN 270-301 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have never had a detectable FVIII inhibitor and recent tests confirm this.
Select...
I am a male over 18 with severe hemophilia A.
Select...
I have been treated with FVIII or cryoprecipitate for at least 150 days.
Select...
I have been on preventive FVIII therapy for over a year.

BMN 270-301 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years 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 Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP.
Secondary outcome measures
Change From Baseline in Annualized FVIII Utilization in EEP.
Change From Baseline in FVIII Activity at Week 104
Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 104
+4 more
Other outcome measures
Percentage of participants with treatment-related adverse events, as assessed by CTCAE v4.03 during years 2-5 following valoctocogene roxaparvovec infusion
Percentage of participants with treatment-related adverse events, as assessed by CTCAE v4.03 in the first 52 weeks following valoctocogene roxaparvovec infusion
Percentage of participants with treatment-related adverse events, as assessed by de novo development of FVIII inhibitors during years 2-5 following valoctocogene roxaparvovec infusion
+1 more

BMN 270-301 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

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
The most common treatments for Hemophilia A include recombinant factor VIII (rFVIII) products, gene therapy, and investigational approaches like bispecific antibodies and anti-TFPI antibodies. rFVIII products work by replacing the deficient factor VIII, essential for blood clotting, thereby reducing bleeding episodes. Gene therapy, such as BMN 270, aims to introduce a functional copy of the factor VIII gene into the patient's cells, potentially providing a long-term solution by enabling the body to produce its own factor VIII. Bispecific antibodies, like Mim8, and anti-TFPI antibodies, like concizumab, work by mimicking or enhancing the function of factor VIII, promoting clot formation. These treatments are crucial for Hemophilia A patients as they help manage bleeding risks, improve quality of life, and reduce the need for frequent infusions.

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
157 Previous Clinical Trials
189,867 Total Patients Enrolled
11 Trials studying Hemophilia A
1,076 Patients Enrolled for Hemophilia A
Medical Monitor, MDStudy DirectorBioMarin Pharmaceutical
72 Previous Clinical Trials
17,932 Total Patients Enrolled
11 Trials studying Hemophilia A
222 Patients Enrolled for Hemophilia A

Media Library

Valoctocogene Roxaparvovec (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03370913 — Phase 3
Hemophilia A Research Study Groups: valoctocogene roxaparvovec Open Label
Hemophilia A Clinical Trial 2023: Valoctocogene Roxaparvovec Highlights & Side Effects. Trial Name: NCT03370913 — Phase 3
Valoctocogene Roxaparvovec (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03370913 — Phase 3
~19 spots leftby Jun 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