Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
6
Countries
1
Sites
1
Von Willebrand's Disease Type 2B
To examine the effects of BT200 on platelet count, VWF indices, FVIII and bleeding in type 2B VWD
Key facts
- Sponsor
- Medical University Of Vienna
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 21 Jul 2023 → ongoing
- Decision date (initial)
- 2024-12-09
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-518294-34-01
- EudraCT number
- 2023-000044-34
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Prophylaxis, Efficacy, Safety, Pharmacodynamic, Pharmacokinetic
To examine the effects of BT200 on platelet count, VWF indices, FVIII and bleeding in type 2B VWD
Secondary objectives 2
- The secondary objective of this study is to assess the pharmacokinetic (PK)/pharmacodynamic (PD) relationship for BT200 in patients with type 2B VWD.
- To further evaluate the safety and tolerability of BT200 in patients with type 2B VWD.
Conditions and MedDRA coding
Von Willebrand's Disease Type 2B
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10047715 | Von Willebrand's disease | 100000004850 |
Regulatory references
- Plan to share IPD
- Yes
- IPD plan description
- The corresponding author/prinicipal investigator are willing to share IPD upon request (anonymized).
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-518294-34-00 | Efficacy and Safety of BT200 (rondaptivon pegol) in Patients with Type 2B von Willebrand disease | Medical University Of Vienna |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- 1. Type 2B VWD with thrombocytopenia and a recent bleeding history
- repeated thrombocytopenia in medical history
- ≥18 years of age
- 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 protocolrelated procedures
Exclusion criteria 5
- 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
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Co-Primary endpoints: Platelet counts, number of monthly bleedings events
Secondary endpoints 2
- Pharmacokinetics: • BT200 concentrations (and derived PK parameters) • Half-life of the substituted Factor VIII product used with and without BT200 (pop-PK-sub-study)
- Pharmacodynamics: • VWF antigen (VWF Ag) • VWF:ristocetin co-factor assay (VWF:RCo) • VWF activity (VWF:GpIbM assay) • VWF collagen binding assay (VWF:CBA) • Enzyme-linked immunosorbent assay (ELISA) for unbound VWF A1 domain (REAADS®) • Whole blood ristocetin induced platelet aggregation (Multiplate®) • Collagen Adenosine Diphosphate closure times measured by the platelet function analyser (PFA-100®)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11657131 · Product
- Active substance
- Rondaptivon Pegol
- Substance synonyms
- Rondoraptivon pegol, Poly(oxy-1,2-ethanediyl), alfa-hydro-omega-methoxy-, 5'-ester with RNA (mG-mC-mC-mA-mG-mG-mG-mA-mC-mC-mU-mA-mA-mG-mG-mC-mA-mC-mA-mU-mG-mG-mU-mC-mC-mC-mU-mG-mG-mC-(3'->3')-dT, 5'-[6-amidohexyl hydrogen phosphate], BT200
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 12 mg/Kg milligram(s)/kilogram
- Max total dose
- 36 mg/Kg milligram(s)/kilogram
- Max treatment duration
- 4 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- MEDICAL UNIVERSITY OF VIENNA
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- Route of administration
- SUBCUTANEOUS USE
- Max daily dose
- 0.8 ml millilitre(s)
- Max total dose
- 2.4 ml millilitre(s)
- Max treatment duration
- 4 Week(s)
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Medical University Of Vienna
- Sponsor organisation
- Medical University Of Vienna
- Address
- Spitalgasse 23, Alsergrund Alsergrund
- City
- Vienna
- Postcode
- 1090
- Country
- Austria
Scientific contact point
- Organisation
- Medical University Of Vienna
- Contact name
- Department of Clinical Pharmacology
Public contact point
- Organisation
- Medical University Of Vienna
- Contact name
- Department of Clinical Pharmacology
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ongoing, recruiting | 6 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Austria | 2023-07-21 | 2025-07-14 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 8 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_protocol_2024-518294-34-00 | 1.1 |
| Protocol (for publication) | D1_protocol_2024-518294-34-00_redacted | 1.1 |
| Recruitment arrangements (for publication) | Placeholder document | 1 |
| Recruitment arrangements (for publication) | Placeholder document_redacted | 1 |
| Subject information and informed consent form (for publication) | L1_sisandicf_adults | 1.2 |
| Subject information and informed consent form (for publication) | L1_sisandicf_adults_redacted | 1.2 |
| Summary of Product Characteristics (SmPC) (for publication) | Placeholder document | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | Placeholder document_redacted | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-17 | Austria | Acceptable 2024-12-06
|
2024-12-09 |