Von Willebrand Disease (VWD), Type 2Phase 2BT200-VWD2BNCT07273721
Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease
A Phase 2 study testing BT200 in people with Von Willebrand Disease (VWD), Type 2, run by Medical University of Vienna at 1 site in Austria.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Willebrand Disease (VWD), Type 2
- What is being tested?
- BT200
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteAustria
- Study dates
- 14 Aug 2025to 31 Jul 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥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
Who cannot join
- 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
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Christian Schörgenhofer, Principal Investigator, MD, PHD contact
Austria1 site, 1 recruiting
- Recruiting
Medical University of Vienna, Department of Clinical Pharmacology
Vienna, State of Vienna, 1090
Bernd Jilma, MD, +43 1 40400 ext. 29810, klin-pharmakologie@meduniwien.ac.at
Cihan Ay, MD
About this study
Official title: Efficacy and Safety of BT200 (Rondoraptivon Pegol) in Patients With Type 2B Von Willebrand Disease
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.
Read more
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).
- Conditions
- Von Willebrand Disease (VWD), Type 2
- Being tested
- BT200, Placebo
- Keywords
- von Willebrand's disease, thrombocytopenia, bleeding, placebo
- Medical subject headings
- von Willebrand Diseases, Thrombocytopenia, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BT200Drug
Aptamer directed against the A1 domain of von Willebrand factor
PlaceboDrug
Placebo for BT200
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Patients randomized to receive placebo first. They will receive the verum BT200 after an adequate washout period Receives: Drug: Placebo |
| BT200 | Experimental | Patients randomized to receive BT200 (verum) first and placebo in the second phase after an adequate washout period. Receives: Drug: BT200 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome measure Platelet Counts
Platelet Counts
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Co-Primary Endpoint Clinically evident bleeding
number of clinically evident bleedings
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Secondary outcomes (10)
von Willebrand factor antigen
Concentration of von Willebrand factor antigen quantified by Enzyme-linked immunoassay
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
von Willebrand factor activity
von Willebrand factor activity quantified by Gp1bM assay
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
VWF activity collagen binding
Activity of VWF quantified with a collagen binding assay
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
VWF:ristocetin co-factor activity
Activity of VWF quantified with a ristocetin co-factor assay
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Enzyme-linked immunosorbent assay (ELISA) for unbound VWF-A1 domain (REAADS® )
Concentration of unbound VWF-A1 domain quantified by Enzyme-linked immunosorbent assay (ELISA) (REAADS® )
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Platelet function under high shear rates
Platelet Function Analyzer
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
BT200 plasma concentrations
plasma concentrations of BT200
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Serious, drug-related AEs
Serious, drug-related AEs
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Premature terminations due to drug-related AEs
Number of participants who premature terminate treatment due to drug-related AEs
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Adverse events indicative of BT200 toxicity
Patterns of serious or non-serious, drug-related AEs, and/or clinically relevant laboratory abnormalities, vital signs, or physical findings suggestive of one or more specific target organs for toxicity of BT200
Measured: During the five-week Treatment Phase compared with the five-week Control Phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2025
- Study start14 Aug 2025
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Jul 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD), Type 2Studies of the same condition, open and under way.
- More trials run by Medical University of ViennaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- randomized, controlled, double blind, crossover study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of Vienna
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Christian Schoergenhofer, Medical University of Vienna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07273721
- Sponsor's protocol ID
- BT200-VWD2B Version 1.1
- Other IDs
- 2024-518294-34-01
- Acronym
- BT200-VWD2B
- Status last verified
- Nov 2025
- First submitted
- 29 Sep 2025
- Last updated
- 9 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Upon reasonable request to the PI.
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of BT200 (Rondoraptivon Pegol) in Patients With Type 2B Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT07273721
- Acronym / sponsor ID
- BT200-VWD2B / BT200-VWD2B Version 1.1
- Conditions
- Von Willebrand Disease (VWD), Type 2
- Keywords
- von Willebrand's disease, thrombocytopenia, bleeding, placebo
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Medical University of ViennaUniversity, hospital or other
- Study start
- 14 Aug 2025 (actual)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Jul 2026 (estimated)
- First posted
- 9 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- randomized, controlled, double blind, crossover study
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPatients randomized to receive placebo first. They will receive the verum BT200 after an adequate washout period |
| BT200 | Experimental | Drug: BT200Patients randomized to receive BT200 (verum) first and placebo in the second phase after an adequate washout period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- ≥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
Key exclusion criteria (5 listed)
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BT200Aptamer directed against the A1 domain of von Willebrand factor | Drug | BT200 |
| PlaceboPlacebo for BT200 | Drug | Placebo |
Primary outcome
Primary Outcome measure Platelet Counts
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Platelet Counts
Co-Primary Endpoint Clinically evident bleeding
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
number of clinically evident bleedings
10 secondary outcomes
von Willebrand factor antigen
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
von Willebrand factor activity
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
VWF activity collagen binding
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
VWF:ristocetin co-factor activity
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Enzyme-linked immunosorbent assay (ELISA) for unbound VWF-A1 domain (REAADS® )
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Platelet function under high shear rates
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
BT200 plasma concentrations
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Serious, drug-related AEs
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Premature terminations due to drug-related AEs
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Adverse events indicative of BT200 toxicity
Time frame: During the five-week Treatment Phase compared with the five-week Control Phase
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2025.
- Individual participant dataSharing planned: Yes. Upon reasonable request to the PI.
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RecruitingObservational study
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Christian Schörgenhofer, Principal Investigator, MD, PHD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BT200 in people with Von Willebrand Disease (VWD), Type 2, run by Medical University of Vienna at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christian Schörgenhofer, Principal Investigator, MD, PHD (+43 1 40400 ext. 29810, christian.schoergenhofer@meduniwien.ac.at), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Outcome measure Platelet Counts (time frame: During the five-week Treatment Phase compared with the five-week Control Phase). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Dec 2025.