Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease
Study Details
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 single SC dose administration in subjects with Von Willebrand disease.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
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 VGA039 in up to 4 cohorts. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD).
Study Design
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 9-12 SC VGA039 dose to be determined |
Drug: VGA039
Single doses of VGA039
|
Outcome Measures
Primary Outcome Measures
- 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
- 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]
Eligibility Criteria
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
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Medical University of Vienna | Vienna | Austria |
Sponsors and Collaborators
- Vega Therapeutics, Inc
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- VGA039-CP001