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One-Time gene therapy could free hemophilia patients from frequent infusions

NCT ID NCT03588299

Ongoing Disease control Sponsor: Bayer Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 07, 2026 · Updated 4 times

Summary

This study tests a one-time gene therapy called BAY2599023 for adults with severe hemophilia A. The therapy uses a harmless virus to deliver a working copy of the factor VIII gene, so the body can produce its own clotting factor. The trial involves 11 participants and aims to find the safest and most effective dose. If it works, it may reduce or eliminate the need for regular clotting factor infusions.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
BAY2599023 (DTX201) – a gene therapy using a modified virus to deliver a working copy of the factor VIII gene
What this could lead to
If successful, this could allow people with severe hemophilia A to produce their own clotting factor, reducing or eliminating the need for regular infusions.
What could go wrong
This is an early, small trial (11 people) focused on safety and dosing. Gene therapy may not work for everyone, and long-term effects are unknown. There is a risk of immune reactions or the therapy not producing enough clotting factor.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hemophilia A severe hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA A

Contacts and locations

Locations

  • APHP-Hopital Necker Enfants malades

    Paris, Île-de-France Region, 75015, France

  • Academisch Medisch Centrum (AMC)

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Arkansas Children's Hospital - Hematology / Oncology

    Little Rock, Arkansas, 72202, United States

  • C.S. Mott Children's Hospital - Hematology / Oncology

    Ann Arbor, Michigan, 48109, United States

  • CHU Rennes - Hopital Pontchaillou

    Rennes, Brittany Region, 35033, France

  • Erasmus Medisch Centrum

    Rotterdam, South Holland, 3015 CE, Netherlands

  • Manchester Royal Infirmary

    Manchester, Greater Manchester, M13 9WL, United Kingdom

  • SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD

    Sofia, Sofia City Province, 1756, Bulgaria

  • UW Health Carbone Cancer Center

    Madison, Wisconsin, 53792, United States

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • University Medical Center Utrecht

    Utrecht, 3584 CX, Netherlands

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland, 66421, Germany

  • Vivantes Klinikum im Friedrichshain

    Berlin, 10249, Germany

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