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

• ClinicalTrials.gov Identifier: NCT05776069
• Recruitment Status: Recruiting
• First Posted: March 20, 2023
• Last Update Posted: December 21, 2023
• Sponsor: Vega Therapeutics, Inc
• Information provided by (Responsible Party): Vega Therapeutics, Inc

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Von Willebrand Diseases	Drug: VGA039; Other: Placebo	Phase 1

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 64 participants
• Allocation: Randomized
• Intervention Model: Sequential Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: March 16, 2023
• Estimated Primary Completion Date: November 2024
• Estimated Study Completion Date: December 2024

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

1. Plasma Concentrations of single IV and SC doses of VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]
2. Pharmacodynamics of single IV and SC doses of VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]
3. Incidence of Anti-drug antibodies to VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]

Eligibility Criteria

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

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.

Contacts and Locations

Contacts

Contact: Clinical Trials; 650-466-8041; info@vega-therapeutics.com

Locations

United States, California

Orthopedic Institute for Children (UCLA); Recruiting

Los Angeles, California, United States, 90007

United States, Colorado

University of Colorado School of Medicine; Recruiting

Aurora, Colorado, United States, 80045

United States, Tennessee

Vanderbilt University Medical Center; Recruiting

Nashville, Tennessee, United States, 37232

Austria

Medical University of Vienna; Recruiting

Vienna, Austria

South Africa

Charlotte Maxeke Johannesburg Academic Hospital; Recruiting

Johannesburg, South Africa

United Kingdom

Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital; Recruiting

London, United Kingdom

Sponsors and Collaborators

Vega Therapeutics, Inc

More Information

• Responsible Party: Vega Therapeutics, Inc
• ClinicalTrials.gov Identifier: NCT05776069
• Other Study ID Numbers: VGA039-CP001
• First Posted: March 20, 2023
• Last Update Posted: December 21, 2023
• Last Verified: December 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Vega Therapeutics, Inc:

Von Willebrand Disorder

Additional relevant MeSH terms:

Von Willebrand Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn