Clinical trial • Phase III • Haematology|Rare Disease
VGA039 for Von Willebrand disease
Phase III trial of VGA039 for Von Willebrand disease. open-label, none/not specified-controlled. 38 participants.
Overview
- Trial Therapeutic Area
- Haematology|Rare Disease
- Trial Disease
- Von Willebrand disease
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 13-08-2025
- First CTIS Authorization Date
- 08-12-2025
Trial design
open-label, none/not specified-controlled Phase III trial in Germany, Austria, Ireland and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 38
Eligibility
Recruits 38 No vulnerable populations selected; participants are adults (18-75 years) and will provide informed consent. Assent is not applicable..
- Pregnancy Exclusion
- Subjects who are pregnant or breastfeeding
- Vulnerable Population
- No vulnerable populations selected; participants are adults (18-75 years) and will provide informed consent. Assent is not applicable.
Inclusion criteria
- {"criterion_text":"- Male or female subjects, 18-75 years of age, inclusive\n- No clinically significant abnormalities in 12-lead ECG determined at screening.\n- No clinically significant abnormalities in vital signs (blood pressure, pulse rate, respiration rate, oral temperature) determined at screening.\n- Documented diagnosis of and laboratory results consistent with VWD of any type.\n- Subjects must have a historical ABR (excluding menstrual bleeds and bleeds under the skin) ≥ 12 over the 6 months prior to screening.\n- Subjects must be judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes.\n- Subjects must have a hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 × 10^9/L (or ≥ 50 × 10^9/L if diagnosed with Type 2B VWD) at screening."}
Exclusion criteria
- {"criterion_text":"- Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening.\n- Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study.\n- Detection of FV Leiden or Prothrombin G20210A mutation, or laboratory results consistent with protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at screening\n- Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation\n- Previous or current treatment for or history of coronary artery diseases, atrial fibrillation, mechanical heart valves or other intravascular foreign materials, venous or arterial thrombosis (Common Terminology Criteria for Adverse Events [CTCAE] Grade >1), or ischemic disease (except for catheter associated thrombosis). Subjects with a positive family history of venous or arterial thromboembolism should also be excluded\n- Severe liver disease (alanine aminotransferase [ALT] and aspartate transaminase [AST] levels >5 times of ULN) or severe kidney disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2)\n- FVIII activity > lower limit of normal (LLN) during Screening. The LLN shall be determined using the assay-specific reference ranges.\n- Subjects who are pregnant or breastfeeding"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Annualized bleeding rate (ABR)","definition_or_measurement_approach":""}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 38
- Recruitment Window Months
- 37
- Consent Approach
- Informed consent obtained from adult participants (study inclusion age 18-75). Country-specific subject information and informed consent forms are provided (documents present for Germany, Austria, Ireland, Italy, Finland and English versions). There are specific ICF variants for main participant, pregnancy follow-up, and pregnant partner. Assent is not applicable because only adults are eligible.
Methods
- Country-specific recruitment arrangements documents (K1_Recruitment arrangements) available for Austria, Germany, Ireland, Italy, Finland (document titles listed in CTIS document list).
- Digital/mobile recruitment in Ireland using 'Mural Link' participant mobile web and app (documents include Mural Link Participant Mobile Web and App Screenshots, Participant Reference Guide, Privacy Policy, Notifications and Terms).
Geography
- Total Number Of Sites
- 7
- Total Number Of Participants
- 16
Germany
- Earliest CTIS Part Ii Submission Date
- 11-11-2025
- Latest Decision Or Authorization Date
- 26-01-2026
- Processing Time Days
- 76
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Vivantes Netzwerk fuer Gesundheit GmbH
- Department Name
- #30:Haemostaseology
- Contact Person Name
- Robert Klamroth
- Contact Person Email
- Robert.klamroth@vivantes.de
- Site Name
- Goethe University Frankfurt
- Department Name
- #31: Haemostaseology
- Contact Person Name
- Wolfgang Miesbach
- Contact Person Email
- miesbach@em.uni-frankfurt.de
Austria
- Earliest CTIS Part Ii Submission Date
- 11-11-2025
- Latest Decision Or Authorization Date
- 08-12-2025
- Processing Time Days
- 27
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Medicine I, Division of Haematology and Haemostaseology
- Contact Person Name
- Cihan Ay
- Contact Person Email
- cihan.ay@meduniwien.ac.at
Ireland
- Earliest CTIS Part Ii Submission Date
- 11-11-2025
- Latest Decision Or Authorization Date
- 26-01-2026
- Processing Time Days
- 76
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- St James's Hospital
- Department Name
- Haematology
- Contact Person Name
- Niamh O'Connell
- Contact Person Email
- nocconnellct@stjames.ie
Italy
- Earliest CTIS Part Ii Submission Date
- 18-11-2025
- Latest Decision Or Authorization Date
- 26-01-2026
- Processing Time Days
- 69
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- SC di Medicina Emostasi e Trombosi - Centro Emofilia e Trombosi Angelo Bianchi
- Contact Person Name
- Flora Peyvandi
- Contact Person Email
- flora.peyvandi@unimi.it
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- Dipartimento Cardiotoracovascolare- SOD Malattie emorragiche e della coagulazione
- Contact Person Name
- Giancarlo Castaman
- Contact Person Email
- castaman@aou-careggi.toscana.it
Finland
- Earliest CTIS Part Ii Submission Date
- 30-03-2026
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 9
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- HUS-yhtymae
- Department Name
- Hematology
- Contact Person Name
- Timea Szanto
- Contact Person Email
- timea.szanto@hus.fi
Sponsor
Primary sponsor
- Full Name
- Vega Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Eresearchtechnology Inc.
- Responsibilities
- sponsorDuties code: 7
- Name
- Medidata Solutions Inc.
- Responsibilities
- sponsorDuties code: 7
Third parties
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties code: 7","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties code: 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Mural Health Technologies Inc.","duties_or_roles":"sponsorDuties code: 15; value: Patient reimbursement","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Atreo Inc.","duties_or_roles":"sponsorDuties code: 3","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- VGA039
- Active Substance
- VGA039
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- SUBCUTANEOUS INJECTION
- Route
- SUBCUTANEOUS INJECTION
- Authorisation Status
- prodAuthStatus: 1
- Orphan Designation
- Yes
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