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

Phase 1

Recruiting

• Conditions: Von Willebrand Diseases
• Interventions: Drug: VGA039; Other: Placebo

• Registration Number: NCT05776069
• Lead Sponsor: Vega Therapeutics, Inc

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.

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).

Basic Information
|
Recruitment & Eligibility
|
Study & Design
|
Trial Locations
|
Related News

Study Timeline

• Study First Submitted: 2023-03-08
• Study First Submitted QC: 2023-03-08
• Study Start: 2023-03-16
• Study First Posted: 2023-03-20
• Status Verified: 2023-12
• Last Update Submitted: 2024-07-16
• Last Update Posted: 2024-07-17
• Primary Completion: 2024-11
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 64

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Part 1	VGA039	Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined
Part 1	Placebo	Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined
Part 2	VGA039	Cohorts A-H IV or SC VGA039 dose to be determined

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]	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

Name	Time	Method
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

Trial Locations

Locations (19)

Orthopedic Institute for Children (UCLA); 🇺🇸 Los Angeles, California, United States

Medical University of Vienna; 🇦🇹 Vienna, Austria

K J Somaiya Super Speciality Hospital & Research Centre; 🇮🇳 Sion, Mumbai, India

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Versiti Comprehensive Center for Bleeding Disorders; 🇺🇸 Milwaukee, Wisconsin, United States

University of Texas Southwestern; 🇺🇸 Dallas, Texas, United States

Hemocentro Unicamp; 🇧🇷 Campinas, Sao Paulo, Brazil

Hospital das Clinicas - USP Endereco; 🇧🇷 São Paulo, Brazil

Hamilton Health Sciences Corporation; 🇨🇦 Hamilton, Ontario, Canada

Queens University; 🇨🇦 Kingston, Ontario, Canada

Royal London Hospital, Clinical Haematology Research; 🇬🇧 Whitechapel, London, United Kingdom

St. Michaels Hospital; 🇨🇦 Toronto, Ontario, Canada

Royal Free Hospital; 🇬🇧 London, United Kingdom

Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital; 🇬🇧 London, United Kingdom

University of Colorado School of Medicine; 🇺🇸 Aurora, Colorado, United States

Hemophilia Center of Western PA; 🇺🇸 Pittsburgh, Pennsylvania, United States

Charlotte Maxeke Johannesburg Academic Hospital; 🇿🇦 Johannesburg, South Africa

Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO; 🇧🇷 Rio De Janeiro, Brazil

UC Davis Medical Center; 🇺🇸 Sacramento, California, United States