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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Von Willebrand Diseases
• Intervention / Treatment: Drug: VGA039; Other: Placebo

Detailed 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).

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Clinical Trials; Phone Number: 650-466-8041; Email: info@vega-therapeutics.com

Study Locations

• Austria
  • Vienna, Austria
    • Recruiting
    • Medical University of Vienna
• South Africa
  • Johannesburg, South Africa
    • Recruiting
    • Charlotte Maxeke Johannesburg Academic Hospital
• United Kingdom
  • London, United Kingdom
    • Recruiting
    • Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital
• United States
  • California
    • Los Angeles, California, United States, 90007
      • Recruiting
      • Orthopedic Institute for Children (UCLA)
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado School of Medicine
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Part 1; Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined	Drug: VGA039; Single doses of VGA039; Other: Placebo; Single doses of Placebo
Experimental: Part 2; Cohorts A-H IV or SC VGA039 dose to be determined	Drug: VGA039; Single doses of VGA039

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]	Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs).	From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively

Secondary Outcome Measures

Outcome Measure	Time Frame
Plasma Concentrations of single IV and SC doses of VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Pharmacodynamics of single IV and SC doses of VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Incidence of Anti-drug antibodies to VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively

Collaborators and Investigators

• Sponsor: Vega Therapeutics, Inc

Study record dates

Study Major Dates

• Study Start (Actual): March 16, 2023
• Primary Completion (Estimated): November 1, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: March 8, 2023
• First Submitted That Met QC Criteria: March 8, 2023
• First Posted (Actual): March 20, 2023

Study Record Updates

• Last Update Posted (Actual): December 21, 2023
• Last Update Submitted That Met QC Criteria: December 19, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Von Willebrand Disorder
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: VGA039-CP001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No