(19429) A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-505827-29-00

Acronym

19429

Enrollment

Registered

2023-12-18

Start date

2018-10-30

Completion date

Unknown

Last updated

2025-07-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Brief summary

Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest

Detailed description

Expression pattern of FVIII activity., Proportion of patients in the respective dose step, that reached an expression of FVIII above 5% at 6 months and 12 months following the IV administration of BAY2599023

Interventions

DRUGBAY 2599023

Eligibility

Sex/Gender

Male

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest	—

Secondary

Measure	Time frame
Expression pattern of FVIII activity., Proportion of patients in the respective dose step, that reached an expression of FVIII above 5% at 6 months and 12 months following the IV administration of BAY2599023	—

Countries

Bulgaria, France, Germany, Netherlands

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026