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

Официальное название

A Multi-Modular Trial to Evaluate VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease and Other Bleeding Disorders (VIVID)

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат VGA039
  Single doses of VGA039
• Другое Placebo
  Single doses of Placebo
• Препарат VGA039
  Multiple doses of VGA039

Первичные конечные точки

• Incidence of Treatment-Emergent Adverse Events [Safety and tolerability] [Срок оценки: From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively]

Вторичные конечные точки (3)

• Plasma Concentrations of single IV and SC doses of VGA039 [Срок оценки: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively]
• Pharmacodynamics of single IV and SC doses of VGA039 [Срок оценки: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively]
• Incidence of Anti-drug antibodies to VGA039 [Срок оценки: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively]

Критерии участия

Key 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 Key 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Да

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Двойное слепое

Основная цель

Лечение

Центры проведения

США · 10 центров

• Orthopedic Institute for Children (UCLA) — Los Angeles
• UC Davis Medical Center — Sacramento
• University of Colorado School of Medicine — Aurora
• Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory — Atlanta
• Science 37, Inc. — Morrisville
• Hemophilia Center of Western PA — Pittsburgh
• Vanderbilt University Medical Center — Nashville
• University of Texas Southwestern — Dallas
• … и ещё 2 центра

Великобритания · 5 центров

• Queen Elizabeth Hospital Birmingham — Birmingham
• University Hospital Southampton NHS Foundation Trust — Southampton
• Royal Free Hospital — London
• Royal London Hospital, Clinical Haematology Research — Whitechapel
• Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital — London

Бразилия · 3 центра

• Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO — Rio de Janeiro
• Hemocentro Unicamp — Campinas
• Hospital das Clinicas - USP Endereco — São Paulo

Канада · 3 центра

• Hamilton Health Sciences Corporation — Hamilton
• Queens University — Kingston
• St. Michaels Hospital — Toronto

Австралия · 1 центр

• Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre — Herston

Австрия · 1 центр

• Medical University of Vienna — Vienna

Индия · 1 центр

• K J Somaiya Super Speciality Hospital & Research Centre — Sion

ЮАР · 1 центр

• Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg

Идентификаторы

NCT: NCT05776069 · VGA039-CP001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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