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Virus Therapy

Gene Therapy for Hemophilia A

Q: Are there currently vacancies for individuals to participate in this research?

<p>Evidently, clinicaltrials.gov data shows that this research is no longer actively seeking participants since its most recent update on November 9th 2022. However, there are currently 89 other trials with open recruitment policies now available.</p>

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest

Secondary outcome measures

Expression pattern of FVIII activity.

Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%

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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

BayerLead Sponsor

2,237 Previous Clinical Trials

25,326,967 Total Patients Enrolled

60 Trials studying Hemophilia A

5,628 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

Frequently Asked Questions

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

Are there currently vacancies for individuals to participate in this research?

"Evidently, clinicaltrials.gov data shows that this research is no longer actively seeking participants since its most recent update on November 9th 2022. However, there are currently 89 other trials with open recruitment policies now available."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".

2018. "Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03588299.

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