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Gene Therapy
Gene Therapy for Hemophilia A
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
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.
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.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 60 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~60 months
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 moreTrial 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).
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Who is running the clinical trial?
BioMarin PharmaceuticalLead Sponsor
156 Previous Clinical Trials
190,705 Total Patients Enrolled
11 Trials studying Hemophilia A
2,014 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
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been on hemophilia treatment in the past year.I have a bleeding disorder that is not hemophilia A.I am a male over 18 with severe hemophilia A.I am receiving or plan to receive immune therapy for FVIII during the study.I am willing to not drink alcohol for at least a year after my treatment.I do not have an active infection or a condition that weakens my immune system, except for HIV with undetectable viral load.I do not have severe kidney, liver problems, infections, or a history of liver cancer.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Research Study Groups:
This trial has the following groups:- Group 1: Valoctocogene roxaparvovec Open Label
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are people with the required health qualifications able to participate in this trial at this time?
"Yes, this is an active clinical trial that was first posted on December 10th, 2020. The listing was last edited on October 13th, 2022 according to the information available on clinicaltrials.gov"
Answered by AI
How many people are included in this research project?
"The information on clinicaltrials.gov does show that this study is looking for 20 more participants at 2 sites. The trial was first posted on December 10th, 2020 and the most recent update was October 13th, 2022."
Answered by AI
What sets this clinical trial apart from others like it?
"BioMarin Pharmaceuticals first studied Valoctocogene roxaparvovec in 2015 with a small trial of 15 patients. After the initial success, the drug received Phase 1 & 2 approval and is now being studied in 6 different trials taking place across 13 countries and 15 cities."
Answered by AI
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