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" (NCT03588299)
A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A
🩺 Plain-English Summary (8th-Grade Level)
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.
Who can join: Review the eligibility criteria below.
Location: Arkansas Children's Hospital - Hematology / Oncology — Little Rock, Arkansas
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (13 Sites)
- Arkansas Children's Hospital - Hematology / Oncology — Little Rock, Arkansas
- C.S. Mott Children's Hospital - Hematology / Oncology — Ann Arbor, Michigan
- UW Health Carbone Cancer Center — Madison, Wisconsin
- SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD — Sofia, Sofia City Province
- CHU Rennes - Hopital Pontchaillou — Rennes, Brittany Region
- APHP-Hopital Necker Enfants malades — Paris, Île-de-France Region
- Universitätsklinikum des Saarlandes — Homburg, Saarland
- Vivantes Klinikum im Friedrichshain — Berlin,
- Academisch Medisch Centrum (AMC) — Amsterdam, North Holland
- Erasmus Medisch Centrum — Rotterdam, South Holland
- Universitair Medisch Centrum Groningen — Groningen,
- University Medical Center Utrecht — Utrecht,
- Manchester Royal Infirmary — Manchester, Greater Manchester
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hemophilia A
- Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A — Los Angeles, California
- Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A — Milwaukee, Wisconsin
- A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A — Gainesville, Florida
- A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A — Gilbert, Arizona
- Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults — Mountain View, California
Explore by condition: All Hemophilia A clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.