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

Recruitment Complete

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.

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 (EDs) to FVIII concentrates (recombinant or plasma-derived). 
If on prophylaxis, are required to be willing to stop prophylactic treatment at specified time points throughout the study or
If on-demand: should have had > 4 bleeding events in the last 52 weeks
- Agree to use reliable barrier contraception.
Exclusion Criteria
- History of allergic reaction to any FVIII product.
- Clinically relevant findings in the physical examination considered critical by the treating physician, including obesity with BMI > 35 kg/m*2
- Current evidence of measurable inhibitor against factor VIII, prior history of inhibitors to FVIII protein or clinical history suggestive of inhibitor.
- Evidence of active hepatitis B or C.
- Currently on antiviral therapy for hepatitis B or C.
- Significant underlying liver disease.
- Serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm*3; HIV+ and stable participants with CD4 count >200/mm*3 and undetectable viral load are eligible to enroll.
- Detectable antibodies reactive with AAVhu37capsid.
- Participant with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B).
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks.
- Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY2599023 (DTX201), or a contraindication to prednisolone

Trial Summary

Enrollment Goal
11
Trial Dates
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Phase
1/2
Could I receive a placebo?
No
Products
BAY2599023 (DTX201)
Accepts Healthy Volunteers
No

Where to Participate

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Locations
Status
LocationsStatus
Locations

C.S. Mott Children's Hospital

Ann Arbor, United States, 48109

Status
Terminated
 
Locations

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

Sofia, Bulgaria, 1756

Status
Active, not recruiting
 
Locations

Arkansas Children's Hospital

Little Rock, United States, 72202-3500

Status
Terminated
 
Locations

Universitätsklinikum des Saarlandes

Homburg, Germany, 66421

Status
Active, not recruiting
 
Locations

Vivantes Klinikum im Friedrichshain

Berlin, Germany, 10249

Status
Completed
 
Locations

Manchester Royal Infirmary

Manchester, United Kingdom, M13 9WL

Status
Active, not recruiting
 
Locations

Academisch Medisch Centrum (AMC)

AMSTERDAM, Netherlands, 1105 AZ

Status
Active, not recruiting
 
Locations

Erasmus Medisch Centrum

ROTTERDAM, Netherlands, 3015 CE

Status
Active, not recruiting
 
Locations

Universitair Medisch Centrum Groningen

GRONINGEN, Netherlands, 9713 GZ

Status
Completed
 
Locations

University Medical Center Utrecht

UTRECHT, Netherlands, 3584 CX

Status
Completed
 
Locations

Hôpital Pontchaillou

RENNES CEDEX, France, 35033

Status
Completed
 
Locations

Hopital Necker les enfants malades - Paris

PARIS, France, 75015

Status
Active, not recruiting
 
Locations

University of Wisconsin - Madison

Madison, United States, 53792

Status
Active, not recruiting
 

Trial Design