Phase 1a study of VGA039 in healthy volunteers and patients with von Willebrand disease

  • Research type

    Research Study

  • Full title

    A Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of VGA039 Following Intravenous or Subcutaneous Administration of Single Ascending Doses in Healthy Adults and Adult Patients with von Willebrand Disease



  • Contact name

    Michelle Carpenter

  • Contact email

  • Sponsor organisation

    Vega Therapeutics, Inc.

  • Eudract number


  • Identifier


  • Research summary

    This study is divided into 2 parts. Part 1 will test the study drug on healthy volunteers for the first time. Part 2 will test the study drug on participants with Von Willebrand Disease for the first time. The UK will take part in Part 2 only.

    The main purpose of this clinical trial is to research the safety and tolerability of the study drug, VGA039. The study will be conducted on Von Willebrand Disease (VWD) patients (called participants) (Part 2, single ascending dose). VGA039 is being given to VWD participants for the first time, however the drug has already been tested in animals, human samples (assessment in test tubes), and healthy volunteer people.

    This study will look at the amount and distribution in the body (“pharmacokinetics”) and test the effect and tolerability (“pharmacodynamics”) of the study drug in VWD participants. The immunogenicity of the substance will also be investigated, i.e., the possibility of an allergic reaction and/or immunisation against the study drug after the administration of one dose. Blood samples will be collected and then tested.

    This drug is called a human monoclonal immunoglobulin (also called an antibody). Monoclonal antibodies are proteins that can specifically recognise and bind antigens (certain structures) in the body. The study drug is an antibody directed against a specific protein in the blood called Protein S that plays a key role in regulating bleeding tendency. The study drug could be effective in decreasing key functions of Protein S, which could lead to a reduction in bleeding for individuals with VWD.

    This study is being conducted in multiple countries. It is anticipated that approximately 24 people will participate in this part 2 of the study. Of these 24 people, approximately 12 people will receive an infusion of the study drug into the vein (intravenous; IV), and approximately 12 people will receive an injection of the study drug under the skin (subcutaneous; SC).

  • REC name

    London - Brent Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    27 Oct 2023

  • REC opinion

    Further Information Favourable Opinion