Trial Condition(s):

Hemophilia A

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".

Bayer Identifier:

19429

ClinicalTrials.gov Identifier:

NCT03588299

EudraCT Number:

2017-000806-39

Trial Purpose

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.

Key Participation Requirements

Sex

Age

18 YearsNo Limit

Inclusion Criteria

    - Males age 18 years or older

    - Confirmed diagnosis of hemophilia A as evidenced by their medical history with plasma FVIII activity levels < 1% of normal or at screening.

    - Have >150 exposure days (E...

Exclusion Criteria

    - History of allergic reaction to any FVIII product

    - Current evidence of measurable inhibitor against factor VIII, as assessed by the central laboratory and/or prior history of inhibitors to FVIII pr...

Trial Summary

Enrollment Goal
30
Trial Dates
November
2018
->
July
2025
Phase
1/2
Could I receive a placebo?
No
Products
BAY2599023 (DTX201)
Accepts Healthy Volunteers
No
Discuss with the trial
center for details

Where to Participate

Locations Status
C.S. Mott Children's Hospital
Ann Arbor United States, 48109
Recruiting
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia Bulgaria, 1756
Recruiting
Arkansas Children's Hospital
Little Rock United States, 72202-3500
Recruiting
Universitätsklinikum der Johann Wolfgang Goethe Universität
Frankfurt Germany, 60590
Not yet recruiting
Universitätsklinikum des Saarlandes
Homburg Germany, 66421
Not yet recruiting
Vivantes Klinikum im Friedrichshain
Berlin Germany, 10249
Not yet recruiting
Manchester Royal Infirmary
Manchester United Kingdom, M13 9WL
Recruiting
St Thomas' Hospital
London United Kingdom, SE1 7EH
Not yet recruiting
Academisch Medisch Centrum (AMC)
AMSTERDAM Netherlands, 1105 AZ
Recruiting
Erasmus Medisch Centrum
ROTTERDAM Netherlands, 3015 CE
Recruiting
Universitair Medisch Centrum Groningen
GRONINGEN Netherlands, 9713 GZ
Recruiting
University Medical Center Utrecht
UTRECHT Netherlands, 3584 CX
Recruiting
Hôpital Pontchaillou
RENNES CEDEX France, 35033
Not yet recruiting
Hôpital de la Timone - Marseille
MARSEILLE France, 13005
Not yet recruiting
Hopital Necker les enfants malades - Paris
PARIS France, 75015
Not yet recruiting
Hôpital Calmette - Lille Cedex
LILLE CEDEX France, 59037
Not yet recruiting
Hospital Regional de Málaga
Málaga Spain, 29010
Not yet recruiting
Hospital Central de Asturias
Oviedo Spain, 33011
Not yet recruiting
Hospital de la Santa Creu i de Sant Pau
Barcelona Spain, 08041
Not yet recruiting
Ciutat Sanitària i Universitaria de la Vall d'Hebron
Barcelona Spain, 08035
Not yet recruiting
Hospital Universitario "La Paz"
Madrid Spain, 28046
Not yet recruiting
Hospital Universitari i Politècnic La Fe
Valencia Spain, 46026
Not yet recruiting
University Hospitals Cleveland Medical Center
Cleveland United States, 44106
Not yet recruiting
University of Wisconsin - Madison
Madison United States, 53792
Recruiting
Loma Linda Children's Hospital
San Bernardino United States, 92408
Withdrawn
Trial Design
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
Trial Type : Interventional
Intervention Type : Drug
Trial Purpose : Treatment
Allocation : N/A
Blinding : N/A
Assignment : Single Group Assignment
Trial Arms : 1

Primary Outcome

  • Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interestTimeframe: Up to 52 weeks

Secondary Outcome

  • Expression pattern of FVIII activity.Timeframe: Up to 5 years
  • Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%Timeframe: At 6 months and 12 months following the IV administration of BAY2599023

Additional Information

Not Available