Status:
COMPLETED
BT200 in Hereditary Bleeding Disorders
Lead Sponsor:
Medical University of Vienna
Conditions:
Von Willebrand Diseases
Hemophilia A
Eligibility:
All Genders
18-70 years
Phase:
PHASE2
Brief Summary
BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an incre...
Eligibility Criteria
Inclusion
- To be eligible for this study, patients must meet all of the following inclusion criteria:
- Hereditary bleeding disorder:
- Congenital hemophilia A without inhibitors with a prophylactic treatment regime
- Heterozygous carriers of hemophilia A with subnormal FVIII levels
- VWD Type 1, "Vicenza" type
- VWD Type 2b
- Male or female, age ≥18-70 years old at Screening
- If female, must be post-menopausal or surgically sterilized
- Able to comprehend and to give informed consent
- Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocol-related procedures -
Exclusion
- Patients meeting any of the following criteria will be excluded from the study:
- Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the patient or compromise the quality of the data derived from his/her participation in this study
- Medical History of spontaneous (not FVIII or FEIBA-associated) venous or arterial thromboembolic events
- History of significant drug allergy or anaphylactic reactions
- Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the patient to be able to comply fully with study procedures
- Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the patient's welfare or the integrity of the study's results
- Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start -
Key Trial Info
Start Date :
December 14 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 10 2021
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT04677803
Start Date
December 14 2020
End Date
September 10 2021
Last Update
November 11 2021
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Medical University of Vienna
Vienna, Austria, A-1090