Valoctocogene roxaparvovec for Hemophilia A With Inhibitors

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Hemophilia A With Inhibitors+1 MoreValoctocogene roxaparvovec - Biological
Eligibility
18+
Male
What conditions do you have?
Select

Study 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.

Eligible Conditions
  • Hemophilia A With Inhibitors
  • Hemophilia A With Inhibitors to Factor VIII

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 8 Secondary · Reporting Duration: 60 months

60 months
A change in Factor VIII inhibitor titer (Part A) after administration of BMN 270.
A decrease 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 emicizumab or FVIII replacement treatment after administration of BMN 270.
Change in the annualized number of bleeding episodes requiring exogenous hemophilia therapy after administration of BMN 270.
Change in the annualized utilization (IU/kg) of emicizumab and exogenous FVIII replacement therapy for subjects receiving emicizumab or FVIII prophylaxis respectively after administration of BMN 270.
Change in the annualized utilization of hemophilia therapy after administration of BMN 270
Change of the median Factor VIII activity.
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.

Trial Safety

Safety Progress

1 of 3

Trial Design

1 Treatment Group

Valoctocogene roxaparvovec Open Label
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Valoctocogene roxaparvovec · No Placebo Group · Phase 1 & 2

Valoctocogene roxaparvovec Open Label
Biological
Experimental Group · 1 Intervention: Valoctocogene roxaparvovec · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 60 months

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
145 Previous Clinical Trials
13,451 Total Patients Enrolled
Medical Monitor, MDStudy DirectorBioMarin Pharmaceutical
70 Previous Clinical Trials
18,118 Total Patients Enrolled

Eligibility Criteria

Age 18+ · Male Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a history of bleeding, inhibitor or hemophilia therapy in the last 12 months.
You are willing to abstain from alcohol consumption for at least 52 weeks following BMN 270 infusion.