Study Completed

Hemophilia A

Bayer Identifier:

19429

ClinicalTrials.gov Identifier:

NCT03588299

EudraCT Number:

2017-000806-39

EU CT Number:

2023-505827-29-00

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"

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 Participants Requirements

Sex

Male

Age

18 - N/A (No 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 (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

Trial Dates

November 2018 - September 2026

Phase

Phase I/II

Could I Receive a placebo

Products

Peboctocogene camaparvovec, BAY 2599023

Accepts Healthy Volunteer

Where to participate

Status	Institution	Location
Terminated	Vivantes Klinikum im Friedrichshain	Berlin, 10249, Germany
Completed	Universitätsklinikum des Saarlandes	Homburg, 66421, Germany
Completed	Manchester Royal Infirmary \| Haematology	Manchester, M13 9WL, United Kingdom
Withdrawn	St Thomas' Hospital \| Haemostasis and Thrombosis Centre	London, SE1 7EH, United Kingdom
Completed	C.S. Mott Children's Hospital - Hematology / Oncology	Ann Arbor, 48109, United States
Terminated	Arkansas Children's Hospital - Hematology / Oncology	Little Rock, 72202, United States
Completed	UW Health Carbone Cancer Center	Madison, 53792, United States
Withdrawn	UH Seidman Cancer Center	Cleveland, 44106, United States
Withdrawn	Loma Linda University Children's Hospital - Hematology / Oncology	Loma Linda, 92354, United States
Terminated	CHU Rennes - Hopital Pontchaillou	Rennes, 35033, France
Completed	APHP-Hopital Necker Enfants malades	Paris, 75015, France
Completed	Academisch Medisch Centrum (AMC)	Amsterdam, 1105 AZ, Netherlands
Completed	Erasmus Medisch Centrum	Rotterdam, 3015 CE, Netherlands
Terminated	Universitair Medisch Centrum Groningen	Groningen, 9713 GZ, Netherlands
Terminated	University Medical Center Utrecht	Utrecht, 3584 CX, Netherlands
Completed	SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD	Sofia, 1756, Bulgaria

Primary Outcome

Number of patients with adverse events (AEs), treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and AEs/SAEs of special interest
Time Frame:
Up to 5 years.

Secondary Outcome

Expression pattern of FVIII activity.
Determined using both a one-stage assay and chromogenic assay.
Time Frame:
Up to 5 years
Proportion of patients in the respective dose step, that reached an expression of FVIII above 5%
Time Frame:
At 6 months and 12 months following the IV administration of BAY2599023

Trial design

Phase 1/2 Open-Label Safety and Dose-Finding Study of 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

Open Label

Assignment

Single Group

Trial Arms

Additional Information

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