Summary

Eligibility
for people ages 18-60 (full criteria)
Healthy Volunteers
healthy people welcome
Location
at UCLA
Dates
study started
completion around

Description

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.

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

Details

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).

Keywords

Von Willebrand Diseases, Von Willebrand Disorder, VGA039

Eligibility

You can join if…

Open to people ages 18-60

(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

You CAN'T join if...

(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.

Locations

  • Orthopedic Institute for Children (UCLA) accepting new patients
    Los Angeles California 90007 United States
  • University of Colorado School of Medicine accepting new patients
    Aurora Colorado 80045 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Vega Therapeutics, Inc
ID
NCT05776069
Phase
Phase 1 Von Willebrand Disease Research Study
Study Type
Interventional
Participants
Expecting 64 study participants
Last Updated