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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05776069
Recruitment Status : Recruiting
First Posted : March 20, 2023
Last Update Posted : April 7, 2023
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Von Willebrand Diseases Drug: VGA039 Other: Placebo 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 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).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : March 16, 2023
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


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 A-H IV or SC VGA039 dose to be determined
Drug: VGA039
Single doses of VGA039




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and tolerability] [ Time Frame: 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 :
  1. Plasma Concentrations of single IV and SC doses of VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]
  2. Pharmacodynamics of single IV and SC doses of VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]
  3. Incidence of Anti-drug antibodies to VGA039 [ Time Frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05776069


Contacts
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Contact: Clinical Trials 650-466-8041 info@vega-therapeutics.com

Locations
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Austria
Medical University of Vienna Recruiting
Vienna, Austria
Sponsors and Collaborators
Vega Therapeutics, Inc
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Responsible Party: Vega Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT05776069    
Other Study ID Numbers: VGA039-CP001
First Posted: March 20, 2023    Key Record Dates
Last Update Posted: April 7, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Vega Therapeutics, Inc:
Von Willebrand Disorder
Additional relevant MeSH terms:
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Von Willebrand Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Blood Platelet Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn