Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease

Vega Therapeutics

Status and phase: Enrolling; Phase 1

Conditions: Von Willebrand Diseases

Treatments: Other: Placebo; Drug: VGA039

Study type: Interventional

Funder types: Industry

Identifiers

NCT05776069

VGA039-CP001

Details and patient eligibility

About

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.

Full description

This first in human study consists of 2 parts based on the subject population: Part 1 and Part 2. Part 1 is a randomized, double-blind, placebo-controlled, single ascending dose (SAD) evaluation of IV or SC VGA039 or placebo in up to 8 cohorts. Part 2 is an open-label, SAD of SC and IV VGA039 in up to 8 cohorts. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD).

Enrollment: 64 estimated patients

Sex: All

Ages: 18 to 60 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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64 participants in 2 patient groups, including a placebo group

Part 1

Placebo Comparator group

Description:

Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined

Treatment:

Drug: VGA039

Other: Placebo

Part 2

Experimental group

Description:

Cohorts A-H IV or SC VGA039 dose to be determined

Treatment:

Drug: VGA039