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

Von Willebrand Diseases Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05776069
• Other study ID #: VGA039-CP001
• Status: Recruiting
• Phase: Phase 1
• Start date: March 16, 2023
• Est. completion date: December 2024

Study information

• Verified date: December 2023
• Source: Vega Therapeutics, Inc
• Contact: Clinical Trials
• Phone: 650-466-8041
• Email: info[@]vega-therapeutics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 64
• Est. completion date: December 2024
• Est. primary completion date: November 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Von Willebrand Diseases

Intervention

Drug:
• VGA039
Single doses of VGA039

Other:
• Placebo
Single doses of Placebo

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Vega Therapeutics, Inc

Countries where clinical trial is conducted

United States,  Austria,  South Africa,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	Pharmacodynamics of single IV and SC doses of VGA039		From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Secondary	Incidence of Anti-drug antibodies to VGA039		From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively