Phase 3 study of VGA039 prophylaxis in adolescent and adult patients with von Willebrand Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vega Therapeutics Inc.

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

19 Mar 2026 → ongoing

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2025-522056-10-01

ClinicalTrials.gov

NCT07115004

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Others, Efficacy, Pharmacodynamic

To assess the efficacy of subcutaneous (SC) VGA039 prophylaxis on bleeding events in subjects with von Willebrand Disease (VWD) who previously did not receive von Willebrand Factor (VWF)-containing concentrates on a prophylactic basis

Conditions and MedDRA coding

von Willebrand Disease

Regulatory references

Scientific advice from competent authorities

Paul-Ehrlich-Institut, Austrian Agency For Health And Food Safety

Plan to share IPD

No

IPD plan description

IPD will not be shared due to legal, ethical, or regulatory restrictions related to the study population or data use agreements.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Male or female subjects, 18-75 years of age, inclusive
2. No clinically significant abnormalities in 12-lead ECG determined at screening.
3. No clinically significant abnormalities in vital signs (blood pressure, pulse rate, respiration rate, oral temperature) determined at screening.
4. Documented diagnosis of and laboratory results consistent with VWD of any type.
5. Subjects must have a historical ABR (excluding menstrual bleeds and bleeds under the skin) ≥ 12 over the 6 months prior to screening.
6. Subjects must be judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes.
7. 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 8

1. 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.
2. Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study.
3. 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
4. Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation
5. 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
6. 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)
7. FVIII activity > lower limit of normal (LLN) during Screening. The LLN shall be determined using the assay-specific reference ranges.
8. Subjects who are pregnant or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Annualized bleeding rate (ABR)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vega Therapeutics Inc.

Sponsor organisation

Vega Therapeutics Inc.

Address

201 Haskins Way Floor 5th

City

South San Francisco

Postcode

94080-6215

Country

United States

Scientific contact point

Organisation

Vega Therapeutics Inc.

Contact name

Clinical Trials

Public contact point

Organisation

Vega Therapeutics Inc.

Contact name

Clinical Trials

Third parties 5

Locations

5 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 96 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications