Actively Recruiting
Efficacy and Safety of BT200 (Rondoraptivon Pegol) in Patients With Type 2B Von Willebrand Disease
Led by Medical University of Vienna · Updated on 2025-12-09
6
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of BT200 (Rondaptivon Pegol), a new drug for patients with type 2B von Willebrand disease (VWD) who have low platelet counts and a history of bleeding. This phase 2 randomized clinical trial with a cross-over design involves 4 to 6 patients and is conducted at the Medical University of Vienna. The drug is expected to reduce the breakdown of von Willebrand factor (VWF), potentially increasing VWF, platelet count, and factor VIII to reduce bleeding tendency. The study starts with an observation phase, followed by two treatment periods of about 64 days each. On day one, patients receive either BT200 or placebo by injection under the skin. Then, they self-administer 6 mg of BT200 or placebo once weekly for four weeks. After a washout period without treatment, patients switch to the other treatment for another four weeks with weekly injections. Follow-up visits occur during treatment periods, and a final clinic visit happens after the second washout phase. Participants may join an extension study after completion. During the study, researchers monitor platelet counts and clinically evident bleeding as primary outcomes, comparing treatment versus control phases. They also measure various von Willebrand factor activities, platelet function under high shear rates, BT200 levels in plasma, and record adverse events related to the drug. Participants complete all study procedures, including follow-ups and safety assessments, over the full study duration ending with the option of continuing in an extension phase.
CONDITIONS
Brief Title
Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 18 years or older
- Diagnosed with type 2B von Willebrand disease with low platelet counts and recent bleeding history (e.g., recurrent hematomas)
- Able to understand and give informed consent
- Able to cooperate with the study team and complete all study procedures
You will not qualify if you...
- Significant medical history or ongoing illness that could risk safety or affect study data quality
- History of serious drug allergy or anaphylactic reactions
- Substance abuse, mental illness, or other reasons affecting study compliance
- Use of medications within 2 weeks before study start that may affect safety or study results
- Participation in another clinical trial with investigational drugs within 30 days or 5 elimination half-lives prior to treatment start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Five weeks per treatment phase
Participants receive BT200 or placebo in a crossover design, with an adequate washout period between treatments.
Visits during each five-week treatment phase as scheduled by the study team
Trial Site Locations
Total: 1 location
1
Medical University of Vienna, Department of Clinical Pharmacology
Vienna, State of Vienna, Austria, 1090
Actively Recruiting
Research Team
C
Christian Schörgenhofer, Principal Investigator, MD, PHD
B
Bernd Jilma, Subinvestigator, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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