Study of VGA039 in Healthy Volunteers and Patients with Von Willebrand Disease (VIVID)
Purpose
The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.
Condition
- Von Willebrand Diseases
Eligibility
- Eligible Ages
- Between 12 Years and 60 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
(All Subjects) - Subjects, 18 to 60 years of age, inclusive for Parts 1 and 2 - Subjects, 12 to 60 years of age, inclusive for Parts 3 and 5 - 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
Exclusion Criteria
(Subjects in Parts 2, 3, 4 and 5 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 Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Part 1 |
Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined |
|
|
Experimental Part 2 |
Cohorts A-H IV or SC VGA039 dose to be determined |
|
|
Experimental Part 3 |
Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined |
|
|
Experimental Part 4 |
Cohorts of VGA039 single dose for surgical prophylaxis |
|
|
Experimental Part 5 |
Multiple doses of VGA039 in open label extension |
|
Recruiting Locations
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Vega Therapeutics, Inc
Detailed Description
This first-in-human study consists of 5 parts based on the subject population. 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 in healthy volunteers. Part 2 is an open-label, SAD of SC or IV VGA039 in up to 8 cohorts in subjects diagnosed with VWD. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD). Part 3 is an open-label, Phase 1b study of SC multiple doses (MD) of VGA039 in up to 4 cohorts. Part 4 is an open-label, Phase 2 study of SC single, surgical prophylaxis (SP) doses of VGA039 administered prior to a minor surgical procedure in subjects diagnosed with VWD in up to 2 cohorts. Part 5 is an open-label extension (OLE) study of SC MD of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial.