BT200 (Rondaptivon Pegol) for people with type 2B von Willebrand disease and low platelets

Efficacy and Safety of BT200 (Rondoraptivon Pegol) in Patients With Type 2B Von Willebrand Disease

This trial will test whether BT200 (Rondaptivon Pegol) can raise von Willebrand factor, platelet count, and factor VIII and reduce bleeding in adults with type 2B VWD who have low platelets.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Medical University of Vienna (Academic / other)
• Locations: 1 site (Vienna, State of Vienna)
• Trial ID: NCT07273721 on ClinicalTrials.gov

What this trial studies

This randomized, placebo-controlled crossover Phase 2 trial at the Medical University of Vienna will enroll 4–6 adults with type 2B von Willebrand disease and thrombocytopenia. After an initial observation phase participants will receive either a 12 mg subcutaneous dose of BT200 or placebo followed by about 64 days of self-administered 6 mg subcutaneous doses, then cross over to the other arm for a second ~64-day period. The study will monitor safety and measure changes in von Willebrand factor levels, platelet counts, and factor VIII activity as well as bleeding events. Findings will help determine whether BT200 reduces bleeding tendency in this specific patient group.

Who should consider this trial

Good fit: Adults (≥18) with confirmed type 2B VWD, thrombocytopenia and a recent bleeding history who can consent, attend visits at the Vienna site, and self-administer subcutaneous injections.

Not a fit: Patients without thrombocytopenia or with other types of VWD, those with significant comorbidities or drug allergies excluded by the protocol, or those unable to comply with visits or self-injection are unlikely to benefit from this trial.

Why it matters

Potential benefit: If effective, BT200 could increase VWF, platelet counts, and factor VIII and thereby reduce bleeding risk in people with type 2B VWD and thrombocytopenia.

How similar studies have performed: Early studies of BT200 have shown it can inhibit VWF breakdown and raise VWF and platelet levels, but randomized crossover data specifically in type 2B VWD are limited.

Eligibility criteria

Inclusion Criteria:

1. ≥18 years old
2. Type 2B VWD with thrombocytopenia and a recent bleeding history (e.g. recurrent haematomas)
3. Able to comprehend and to give informed consent
4. 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 Criteria:

1. Clinically significant medical history or ongoing chronic illness that would jeopardise the safety of the patient or compromise the quality of the data derived from his/her participation in this study
2. History of significant drug allergy or anaphylactic reactions
3. 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
4. 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
5. 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

Where this trial is running

Vienna, State of Vienna

• Medical University of Vienna, Department of Clinical Pharmacology — Vienna, State of Vienna, Austria (Recruiting)

Study contacts

• Study coordinator: Christian Schörgenhofer, Principal Investigator, MD, PHD

Conditions Von Willebrand Disease, Type 2; von Willebrand's disease; thrombocytopenia; bleeding; placebo