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CompletedNCT04677803Updated Nov 11, 2021

BT200 in Hereditary Bleeding Disorders

A Phase 2 interventional study of BT200 in Von Willebrand Diseases and Hemophilia A, sponsored by Medical University of Vienna. Completed at 1 site in Austria. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2021-11-11.

Sponsored by Medical University of Vienna · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
26
Allocation
Not applicable
Ages
18 Years to 70 Years
Sex
All
01

Study 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. 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.

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.

02

Conditions studied

  • Von Willebrand Diseases
  • Hemophilia A

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03

In context

Hemophilia A

885 studies on the registry are indexed under Hemophilia A; 139 are open to participants now.

This study's enrollment of 26 is close to the median of 27 across 523 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Medical University of Vienna is the lead sponsor of 1,105 studies on the registry; 182 are open to participants now.

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Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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
  2. Male or female, age ≥18-70 years old at Screening
  3. If female, must be post-menopausal or surgically sterilized
  4. Able to comprehend and to give informed consent
  5. 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 -
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
26 participants (actual)

Study arms

  • Experimental
    Treatment

    Subcutaneous (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.

    Drug: BT200

Interventions

  • DrugBT200

    BT200 is a PEGylated synthetic RNA oligonucleotide

06

What researchers measure

Primary outcomes

  1. Hemophilia A

    increase in FVIII activity

    Time frame: Baseline through 4 weeks after dosing

  2. VWD Type 1

    increase in FVIII activity

    Time frame: Baseline through 4 weeks after dosing

  3. VWD Type 2b

    increase in platelet count and/or FVIII activity

    Time frame: Baseline through 4 weeks after dosing

Secondary outcomes

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

    Measured concentration of BT200

    Time frame: Baseline through 4 weeks after dosing

  2. Pharmacodynamics

    PFA-100

    Time frame: Baseline through 4 weeks after dosing

  3. Pharmacodynamics

    Multiplate electrode platelet aggregometer (ristocetin induced)

    Time frame: Baseline through 4 weeks after dosing

  4. Pharmacodynamics

    VWF antigen

    Time frame: Baseline through 4 weeks after dosing

  5. Pharmacodynamics

    VWF:ristocetin co-factor assay

    Time frame: Baseline through 4 weeks after dosing

  6. Pharmacodynamics

    VWF activity

    Time frame: Baseline through 4 weeks after dosing

  7. Pharmacodynamics

    VWF collagen bindign assay

    Time frame: Baseline through 4 weeks after dosing

  8. Pharmacodynamics

    ELISA for unbound VWF-A1 domain

    Time frame: Baseline through 4 weeks after dosing

  9. Pharmacodynamics

    VWF propeptide

    Time frame: Baseline through 4 weeks after dosing

  10. Pharmacodynamics

    Fibrin D-Dimer

    Time frame: Baseline through 4 weeks after dosing

  11. Pharmacodynamics

    Prothrombin fragement (F1.2)

    Time frame: Baseline through 4 weeks after dosing

  12. Pharmacodynamics

    Rotational thrombelastometry

    Time frame: Baseline through 4 weeks after dosing

  13. Pharmacodynamics

    Clot strength assay

    Time frame: Baseline through 4 weeks after dosing

  14. Pharmacodynamics

    Calibrated Thrombogram assay

    Time frame: Baseline through 4 weeks after dosing

Other outcomes

  1. Overall safety and tolerability of BT200

    Serious, drug-related adverse events (AEs)

    Time frame: Baseline through 4 weeks after dosing

  2. Overall safety and tolerability of BT200

    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

    Time frame: Baseline through 4 weeks after dosing

  3. Overall safety and tolerability of BT200

    Clinically evident bleeding assessed using the International International Society on Thrombosis and Haemostasis (ISTH) Bleeding Score

    Time frame: Baseline through 4 weeks after dosing

07

Study locations

1 site
  • Medical University of Vienna
    Vienna, A-1090, Austria
08

References and documents

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 ↗

Individual participant data

Plan to share: No

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 11, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04677803
Lead sponsor
Medical University of Vienna
Responsible party
Ulla Derhaschnig, MD (Associate Professor, Medical University of Vienna) — Principal investigator
First posted
Dec 21, 2020
Start date
Dec 14, 2020
Primary completion
Sep 10, 2021
Completion
Sep 10, 2021
Last update
Nov 11, 2021

Study contacts

Ulla Derhaschnig, MD
principal investigator · MU Vienna, Dept. of Clinical Pharmacology

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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