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

Vega Therapeutics

Status and phase: Enrolling; Phase 2; 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

The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.

Full description

This first-in-human study consists of 5 parts based on the subject population. 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 in healthy volunteers. Part 2 is an open-label, SAD of SC or IV VGA039 in up to 8 cohorts in subjects diagnosed with VWD. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD). Part 3 is an open-label, Phase 1b study of SC multiple doses (MD) of VGA039 in up to 4 cohorts. Part 4 is an open-label, Phase 2 study of SC single, surgical prophylaxis (SP) doses of VGA039 administered prior to a minor surgical procedure in subjects diagnosed with VWD in up to 2 cohorts. Part 5 is an open-label extension (OLE) study of SC MD of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial.

Enrollment: 116 estimated patients

Sex: All

Ages: 12 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 for Parts 1 and 2
• Subjects, 12 to 60 years of age, inclusive for Parts 3 and 5
• 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 Parts 2, 3, 4 and 5 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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116 participants in 5 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

Drug: VGA039

Other: Placebo

Part 2

Experimental group

Description:

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

Treatment:

Drug: VGA039

Drug: VGA039

Part 3

Experimental group

Description:

Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined

Treatment:

Drug: VGA039

Drug: VGA039

Part 4

Experimental group

Description:

Cohorts of VGA039 single dose for surgical prophylaxis

Treatment:

Drug: VGA039

Drug: VGA039

Part 5

Experimental group

Description:

Multiple doses of VGA039 in open label extension