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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

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I have never had a detectable FVIII inhibitor and recent tests confirm this.

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I am a male over 18 with severe hemophilia A.

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

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I have been on preventive FVIII therapy for over a year.

BMN 270-301 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

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

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor

156 Previous Clinical Trials

190,581 Total Patients Enrolled

11 Trials studying Hemophilia A

1,890 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

Frequently Asked Questions

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

Is this clinical trial innovative or novel in its design?

"BioMarin Pharmaceutical first sponsored a clinical trial for valoctocogene roxaparvovec in 2015, which involved 15 participants. Since the study's success, this gene therapy has undergone Phase 1 & 2 drug approval and there are now 6 active trials taking place all over the world."

Answered by AI

What other scientific tests have been done using valoctocogene roxaparvovec?

"At present, there are six clinical trials involving valoctocogene roxaparvovec. Three of those live studies are currently in Phase 3. The numerous trials for valoctocogene roxaparvovec tend to be based in Melbourne and Missouri; however, there are 75 total locations running these sorts of tests."

Answered by AI

Could you tell me how many different sites are being used to oversee this research?

"Currently, there are 14 sites enrolling patients for this study. If you choose to participate in this research, please try to select a location near you from the list of Chapel Hill, Columbus, Chicago or the other 14 locations to minimize travel."

Answered by AI