Clinical trial

A Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of VGA039 Following IV or SC Administration of Single Ascending Doses in Healthy Adults and Subcutaneous Adult Patients With Von Willebrand Disease

Name

VGA039-CP001

Description

This is a multi-center, Phase 1a study to assess the safety, tolerability, PK, and PD of VGA039 following single IV or SC dose administration in healthy subjects and Von Willebrand disease patients.

Trial arms

Trial start

2023-03-16

Estimated PCD

2024-11-01

Trial end

2024-12-01

Status

Recruiting

Phase

Early phase I

Treatment

VGA039

Single doses of VGA039

Arms:

Part 1, Part 2

Placebo

Single doses of Placebo

Arms:

Part 1

Size

Primary endpoint

Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]

From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively

Eligibility criteria

Key Inclusion Criteria (All Subjects) * Subjects, 18 to 60 years of age, inclusive. * No clinically significant laboratory, ECG, or vital signs results. Additional Key Inclusion Criteria (for Subjects in Part 1 Only) • Body mass index of 18-32 kg/m2 Additional Key Inclusion Criteria (for Subjects in Part 2 Only) * Subjects with VWD who are symptomatic, defined as having a history of bleeding or bruising. * Hemoglobin level ≥ 8 g/dL and platelet count ≥ 150 × 109/L at Screening. Exclusion Key Criteria (All Subjects) * Use of hormonal contraceptives within 56 days prior to administration of the study drug. * Subjects with detection of FV Leiden or Prothrombin G20210A mutation, protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at Screening. * Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation. * History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism. * Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction. Additional Key Exclusion Criterion (Subjects in Part 1 Only) • Baseline FVIII activity \> 150 IU/dL. Additional Key Exclusion Criteria (Subjects in Part 2 Only) * Baseline FVIII activity \> 50 IU/dL. * Any acute, clinically significant bleeding event requiring surgical or procedural intervention within 7 days prior to receiving study drug.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 64, 'type': 'ESTIMATED'}}

Updated at

2023-12-21

1 organization

1 product

1 indication

Organization

Vega Therapeutics

Product

VGA039

Indication

Von Willebrand Disease