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Von Willebrand DiseasesPhase 2NCT04677803

BT200 in Hereditary Bleeding Disorders

A Phase 2 study testing BT200 in people with Von Willebrand Diseases and Hemophilia A, run by Medical University of Vienna at 1 site in Austria.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an increase in concentrations of both VWF Ag and Factor VIII (FVIII), but has negligible effect on the activity of either. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Von Willebrand Diseases, Hemophilia A
What is being tested?
BT200
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
1 siteAustria
Study dates
14 Dec 2020to 10 Sep 2021
Phase
Phase 2
Are there results?
None posted in the registry

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 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Inclusion Criteria:To be eligible for this study, patients must meet all of the following inclusion criteria:

  1. Hereditary bleeding disorder:
  • Congenital hemophilia A without inhibitors with a prophylactic treatment regime
  • Heterozygous carriers of hemophilia A with subnormal FVIII levels
  • VWD Type 1, "Vicenza" type
  • VWD Type 2b
  1. Male or female, age ≥18-70 years old at Screening
  2. If female, must be post-menopausal or surgically sterilized
  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: Patients meeting any of the following criteria will be excluded from the study:

  1. Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the patient or compromise the quality of the data derived from his/her participation in this study
  2. Medical History of spontaneous (not FVIII or FEIBA-associated) venous or arterial thromboembolic events
  3. History of significant drug allergy or anaphylactic reactions
  4. 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
  5. 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
  6. 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.

Austria1 site

  • Medical University of Vienna

    Vienna, A-1090

Other trials in Austria

About this study

Official title: A Phase 2a Multiple Dose Basket Study of the Safety, Tolerability, and Pharmacologic Activity of BT200 in Patients With Hereditary Bleeding Disorders

BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an increase in concentrations of both VWF Ag and Factor VIII (FVIII), but has negligible effect on the activity of either. At higher doses, BT200 blocks clearance of VWF and also inhibits its activity, but still does not inhibit FVIII activity. Therefore, low dose BT200 could potentially be used to correct deficiency of VWF and/or FVIII in patients with hereditary bleeding disorders. This study is designed as a "basket design" pilot study to determine the relevant dose and pharmacological activity of BT200 in such patients.

In this open basket study up to 25 patients with the following congenital blood-clotting disorders are to be included: Patients with hemophilia A, heterozygous carriers of hemophilia A with subnormal FVIII levels; patients with von Willebrand syndrome (VWD) type 1, "Vicenza type", and with VWD type 2b.

Read more

Participants will receive BT200 subcutaneously on day 0, day 4 and day 7 in the first week and then once a week for a total of five weeks - initially in a dose of 3 mg, then in week 3 individually after response in a dose of 3 to 9 mg.

Subsequently, blood samples are taken once a week for a further three weeks (wash-out phase).

Patients may be enrolled in an additional pharmacokinetics sub-study. For this purpose, approximately three blood samples are taken to estimate the half-life of substituted FVIII under the influence of BT200.

The primary objective of this study is to obtain clinical proof of mechanism for BT200 in one or more hereditary bleeding disorders.

Being tested
BT200
Medical subject headings
von Willebrand Diseases, Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BT200Drug

    BT200 is a PEGylated synthetic RNA oligonucleotide

Study groups in NCT04677803
Study groupRoleWhat they receive
TreatmentExperimentalSubcutaneous (SC) injection: BT200 dose 3 mg on Day 0, Day 4, and again on Day 7 BT200 dose titrated thereafter between 3 and 9 mg on Days 14, 21, and 28. It is anticipated that dose adjustments will be performed in 2 mg steps. The 9 mg dose will only be applied on day 28 in exceptional circumstances, if no relevant changes in pharmacodynamic and safety parameters will be observed on day 21.
Receives: Drug: BT200

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hemophilia A

    increase in FVIII activity

    Measured: Baseline through 4 weeks after dosing

  2. VWD Type 1

    increase in FVIII activity

    Measured: Baseline through 4 weeks after dosing

  3. VWD Type 2b

    increase in platelet count and/or FVIII activity

    Measured: Baseline through 4 weeks after dosing

Secondary outcomes (14)

  1. Pharmacokinetic (PK) Measured concentration of BT200 (and derived PK parameters)

    Measured concentration of BT200

    Measured: Baseline through 4 weeks after dosing

  2. Pharmacodynamics

    PFA-100

    Measured: Baseline through 4 weeks after dosing

  3. Pharmacodynamics

    Multiplate electrode platelet aggregometer (ristocetin induced)

    Measured: Baseline through 4 weeks after dosing

  4. Pharmacodynamics

    VWF antigen

    Measured: Baseline through 4 weeks after dosing

  5. Pharmacodynamics

    VWF:ristocetin co-factor assay

    Measured: Baseline through 4 weeks after dosing

  6. Pharmacodynamics

    VWF activity

    Measured: Baseline through 4 weeks after dosing

  7. Pharmacodynamics

    VWF collagen bindign assay

    Measured: Baseline through 4 weeks after dosing

  8. Pharmacodynamics

    ELISA for unbound VWF-A1 domain

    Measured: Baseline through 4 weeks after dosing

  9. Pharmacodynamics

    VWF propeptide

    Measured: Baseline through 4 weeks after dosing

  10. Pharmacodynamics

    Fibrin D-Dimer

    Measured: Baseline through 4 weeks after dosing

  11. Pharmacodynamics

    Prothrombin fragement (F1.2)

    Measured: Baseline through 4 weeks after dosing

  12. Pharmacodynamics

    Rotational thrombelastometry

    Measured: Baseline through 4 weeks after dosing

  13. Pharmacodynamics

    Clot strength assay

    Measured: Baseline through 4 weeks after dosing

  14. Pharmacodynamics

    Calibrated Thrombogram assay

    Measured: Baseline through 4 weeks after dosing

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Dec 2020
  2. Study start14 Dec 2020
  3. Primary completion10 Sep 2021
  4. Study completion10 Sep 2021

Resources

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
Not applicable
Intervention model
Single group
Model description
Open-label, individual dose titration-to-response, basket trial
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04677803
Sponsor's protocol ID
BT200-02
Status last verified
Nov 2021
First submitted
3 Dec 2020
Last updated
11 Nov 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Ay C, Kovacevic KD, Kraemmer D, Schoergenhofer C, Gelbenegger G, Firbas C, Quehenberger P, Jilma-Stohlawetz P, Gilbert JC, Zhu S, Beliveau M, Koenig F, Iorio A, Jilma B, Derhaschnig U, Pabinger I. The von Willebrand factor-binding aptamer rondaptivon pegol as a treatment for severe and nonsevere hemophilia A. Blood. 2023 Mar 9;141(10):1147-1158. doi: 10.1182/blood.2022016571.

    PubMed 36108308

  • Ay C, Pabinger I, Kovacevic KD, Gelbenegger G, Schorgenhofer C, Quehenberger P, Jilma-Stohlawetz P, Sunder-Plassman R, Gilbert JC, Zhu S, Jilma B, Derhaschnig U. The VWF binding aptamer rondoraptivon pegol increases platelet counts and VWF/FVIII in type 2B von Willebrand disease. Blood Adv. 2022 Sep 27;6(18):5467-5476. doi: 10.1182/bloodadvances.2022007805.

    PubMed 35772170

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BT200 in people with Von Willebrand Diseases and Hemophilia A, 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?

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: finished, as of the sponsor's last update on 11 Nov 2021. 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. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hemophilia A (time frame: Baseline through 4 weeks after dosing). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04677803, which also lists 2 linked publications.

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 11 Nov 2021.

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