Clinical trial • Phase II • Haematology

Rondaptivon pegol for Von Willebrand disease type 2B

Phase II trial of Rondaptivon pegol for Von Willebrand disease type 2B.

Overview

Trial Therapeutic Area
Haematology
Trial Disease
Von Willebrand disease type 2B
Trial Stage
Phase II
Drug Modality
Oligonucleotide | Other

Key dates

Initial CTIS Submission Date
17-10-2024
First CTIS Authorization Date
09-12-2024

Trial design

0.9% sodium chloride solution (placebo), subcutaneous; max daily dose 0.8 ml; max total dose 2.4 ml-controlled Phase II trial across 1 site in Austria.

Comparator
0.9% sodium chloride solution (placebo), subcutaneous; max daily dose 0.8 ml; max total dose 2.4 ml
Target Sample Size
6

Eligibility

Recruits 6 Vulnerable populations not selected (isVulnerablePopulationSelected: false). Inclusion requires participants to be able to comprehend and give informed consent. A subject information and informed consent form for adults is provided (L1_sisandicf_adults). No assent procedures or special vulnerable-population consent arrangements are described..

Vulnerable Population
Vulnerable populations not selected (isVulnerablePopulationSelected: false). Inclusion requires participants to be able to comprehend and give informed consent. A subject information and informed consent form for adults is provided (L1_sisandicf_adults). No assent procedures or special vulnerable-population consent arrangements are described.

Inclusion criteria

  • {"criterion_text":"- 1. Type 2B VWD with thrombocytopenia and a recent bleeding history"}
  • {"criterion_text":"- repeated thrombocytopenia in medical history"}
  • {"criterion_text":"- ≥18 years of age"}
  • {"criterion_text":"- Able to comprehend and to give informed consent"}
  • {"criterion_text":"- 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

  • {"criterion_text":"- 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"}
  • {"criterion_text":"- History of significant drug allergy or anaphylactic reactions"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- 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 endpoints

  • {"endpoint_text":"- Co-Primary endpoints: Platelet counts, number of monthly bleedings events","definition_or_measurement_approach":"Not specified in the available documents."}

Secondary endpoints

  • {"endpoint_text":"- Pharmacokinetics: • BT200 concentrations (and derived PK parameters) • Half-life of the substituted Factor VIII product used with and without BT200 (pop-PK-sub-study)","definition_or_measurement_approach":"As listed: measurement of BT200 concentrations and derived PK parameters; half-life assessed in a population PK sub-study."}
  • {"endpoint_text":"- 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®)","definition_or_measurement_approach":"Measured using the listed assays (VWF Ag, VWF:RCo, VWF:GpIbM, VWF:CBA, ELISA for unbound VWF A1 domain (REAADS®), Multiplate® for ristocetin-induced aggregation, and PFA-100® closure time)."}

Recruitment

Planned Sample Size
6
Recruitment Window Months
24
Consent Approach
Participants must be able to comprehend and provide informed consent themselves (inclusion criterion). A subject information and informed consent form for adults is provided (L1_sisandicf_adults). No assent procedures or language-specific consent details are specified in the available documents.

Geography

Total Number Of Sites
1
Total Number Of Participants
6

Austria

Earliest CTIS Part Ii Submission Date
28-10-2024
Latest Decision Or Authorization Date
09-12-2024
Processing Time Days
42
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Medical University Of Vienna
Department Name
Department of Clinical Pharmacology
Principal Investigator Name
Christian Schörgenhofer
Principal Investigator Email
christian.schoergenhofer@meduniwien.ac.at
Contact Person Name
Christian Schörgenhofer
Number Of Participants
6

Sponsor

Primary sponsor

Full Name
Medical University Of Vienna
Organisation Type
Educational Institution
Country Of Registered Address
Austria

Investigational products

Investigational Product Name
Rondaptivon pegol
Active Substance
Rondaptivon pegol
Modality
Oligonucleotide
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Maximum Dose
max daily 12 mg/Kg; max total 36 mg/Kg
Investigational Product Name
0.9% sodium chloride solution
Active Substance
Sodium chloride solution
Modality
Other
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Maximum Dose
max daily 0.8 ml; max total 2.4 ml

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