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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This randomized clinical trial with a cross-over design is being conducted at the Department of Clinical Pharmacology at the Medical University of Vienna, and a total of 4-6 patients with type 2B von Willebrand disease (VWD) will participate. The main purpose of this clinical trial is to investigate the efficacy and safety of BT200, a new drug for thrombocytopenic patients with type 2B von Willebrand disease (VWD). Based on previous studies, we expect that this drug will inhibit the breakdown of von Willebrand factor (VWF) in small doses, leading to an increase in von Willebrand factor (VWF), platelet count, and factor VIII. This should also lead to a reduced tendency to bleed. This study will begin with an observation phase and will then proceed in two periods of approximately 64 days each: Placebo or BT200 will be administered subcutaneously at a dose of 12 mg on the first day of the study. After that, patients will self-administer the drug at a dose of 6 mg (0.4 mL) or placebo once a week for another 4 weeks starting the following week (a total of 4 times over a period of 4 weeks). During this time, they will be asked to come to our clinic for a follow-up visit. After a washout phase lasting several weeks, during which patients do not receive the study drug/placebo but are asked to record any bleeding events, the second period begins on day 64: BT200 or placebo is administered again, depending on what the patients received in the first period. Patients therefore receive the study drug for 4 weeks and placebo for 4 weeks; which is administered when is randomized; a follow-up examination also takes place during this period. At the end of the second period, there is another washout phase lasting several weeks. On day 127, the final examination takes place at the clinic, after which patients have the opportunity to participate in an extension study (to be amended).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• ≥18 years old

• Type 2B VWD with thrombocytopenia and a recent bleeding history (e.g. recurrent haematomas)

• 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

Locations
Other Locations
Austria
Medical University of Vienna, Department of Clinical Pharmacology
RECRUITING
Vienna
Contact Information
Primary
Christian Schörgenhofer, Principal Investigator, MD, PHD
christian.schoergenhofer@meduniwien.ac.at
+43 1 40400
Backup
Bernd Jilma, Subinvestigator, MD
bernd.jilma@meduniwien.ac.at
+43 1 40400
Time Frame
Start Date: 2025-08-14
Estimated Completion Date: 2026-07-31
Participants
Target number of participants: 6
Treatments
Placebo_comparator: Placebo
Patients randomized to receive placebo first. They will receive the verum BT200 after an adequate washout period
Experimental: BT200
Patients randomized to receive BT200 (verum) first and placebo in the second phase after an adequate washout period.
Sponsors
Leads: Medical University of Vienna

This content was sourced from clinicaltrials.gov