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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 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- DRUG BAY2599023 (DTX201)
Study Locations (13)
Other
- Vivantes Klinikum im Friedrichshain — Berlin
- Universitair Medisch Centrum Groningen — Groningen
- University Medical Center Utrecht — Utrecht
Arkansas
- Arkansas Children's Hospital - Hematology / Oncology — Little Rock
Michigan
- C.S. Mott Children's Hospital - Hematology / Oncology — Ann Arbor
Wisconsin
- UW Health Carbone Cancer Center — Madison
Sofia City Province
- SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD — Sofia
Brittany Region
- CHU Rennes - Hopital Pontchaillou — Rennes
Île-de-France Region
- APHP-Hopital Necker Enfants malades — Paris
Saarland
- Universitätsklinikum des Saarlandes — Homburg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2018-11-07 |
| Est. Completion | 2026-11-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03588299
The ClinicalTrials.gov registry entry for NCT03588299 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 11 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Bayer, which has 143 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Hemophilia A appearing as the primary indexed condition, and to 1 intervention — of which BAY2599023 (DTX201) is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03588299 reports 13 study locations spanning 11 distinct geographic areas — top geographies include Other, Arkansas, Michigan. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03588299 about?
NCT03588299 is a clinical study titled "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"". 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 b...
What is the current status of trial NCT03588299?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 11 participants. The study started on 2018-11-07. Estimated completion is 2026-11-03.
What conditions does trial NCT03588299 study?
This clinical trial studies the following conditions: Hemophilia A. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03588299?
The interventions under investigation include: BAY2599023 (DTX201) (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03588299?
This trial is sponsored by Bayer, which has 143 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03588299 being conducted?
This trial has 13 study locations across Arkansas, Michigan, Wisconsin, Sofia City Province, Brittany Region. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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