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Virus Therapy

Gene Therapy for Hemophilia A

Phase 1 & 2
Waitlist Available
Research Sponsored by Bayer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Males age 18 years or older
Confirmed diagnosis of hemophilia A with plasma FVIII activity levels < 1% of normal or at screening
Must not have
Participant with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
Current evidence of measurable inhibitor against factor VIII, prior history of inhibitors to FVIII protein or clinical history suggestive of inhibitor
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new gene therapy to treat hemophilia A by delivering a healthy copy of the gene that's missing or defective in people with the disease. Researchers want to find the optimal dose of the therapy so that the body can produce enough clotting factor on its own.

Who is the study for?
This trial is for adult males with severe Hemophilia A, evidenced by very low clotting factor VIII levels and a history of frequent bleeding. Participants must have used clotting factor concentrates extensively and be willing to pause any prophylactic treatments during the study. They should not be obese, have no inhibitors against factor VIII or significant liver disease, no active hepatitis B or C, nor HIV with low CD4 counts.
What is being tested?
Researchers are testing BAY 2599023 (DTX201), which uses a modified virus to deliver a healthy gene for clotting factor VIII into liver cells. The goal is to enable patients' bodies to produce enough of this protein on their own, potentially improving blood clotting in those with Hemophilia A.
What are the potential side effects?
Potential side effects may include immune reactions to the viral vector or the new Factor VIII protein, allergic responses, liver inflammation due to the gene delivery process, and other typical risks associated with gene therapy such as mild fever or muscle aches.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a man aged 18 or older.
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My hemophilia A diagnosis shows FVIII activity levels below 1%.
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I have used Factor VIII concentrates for more than 150 days.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a bleeding disorder that is not hemophilia A.
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I have or had inhibitors against factor VIII.
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I am currently receiving treatment for hepatitis B or C.
Select...
I have a serious liver condition.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Expression pattern of FVIII activity.

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: BAY2599023 / (DTX201)Experimental Treatment1 Intervention
Adult patients with severe hemophilia A, who have been previously treated with FVIII products

Find a Location

Who is running the clinical trial?

BayerLead Sponsor
2,274 Previous Clinical Trials
25,532,370 Total Patients Enrolled
60 Trials studying Hemophilia A
5,624 Patients Enrolled for Hemophilia A
Ultragenix pharmaceuticalUNKNOWN

Media Library

BAY2599023 (DTX201) (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03588299 — Phase 1 & 2
Hemophilia A Research Study Groups: BAY2599023 / (DTX201)
Hemophilia A Clinical Trial 2023: BAY2599023 (DTX201) Highlights & Side Effects. Trial Name: NCT03588299 — Phase 1 & 2
BAY2599023 (DTX201) (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03588299 — Phase 1 & 2
~3 spots leftby Nov 2026
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