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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

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

Study contacts

• Study coordinator: Christian Schörgenhofer, Principal Investigator, MD, PHD
• Email: christian.schoergenhofer@meduniwien.ac.at
• Phone: +43 1 40400

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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  Recruiting at 9 sites Phase 2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07640893

• ALN-6400 for women with von Willebrand disease and heavy menstrual bleeding

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• Endothelial colony-forming cell behavior in people with von Willebrand disease and acquired von Willebrand syndrome

  This project will see how endothelial colony-forming cells (ECFCs) behave in people with VWD or AVWS compared with healthy volunteers.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.