Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)

Официальное название

A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients With Von Willebrand Disease (VIVID-6)

Обзор

This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)

Подробное описание

This Phase 3 multicenter, open-label, single-sequence cross-over study will investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. The study consists of an Observational Period of at least 24 weeks followed by an Active Treatment Period of approximately 49 weeks of VGA039 treatment. Bleeding data and details of treatments used will be collected from each patient during both study periods. The number, duration, location, and types of bleeds experienced, as well as treatments for bleeds, will be recorded in patient diaries. Adverse events will also be monitored and recorded throughout both study periods.

Вмешательства

• Препарат VGA039
  VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.

Первичные конечные точки

• Incidence of bleeding events [Efficacy] [Срок оценки: Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation]

Вторичные конечные точки (4)

• Incidence of treated bleeding events [Efficacy] [Срок оценки: From 7 days from Informed consent until 49 weeks after SC study drug initiation]
• Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including hypersensitivity, anaphylaxis, or anaphylactoid reactions [Срок оценки: From informed consent until 49 weeks after SC study drug initiation]
• Plasma concentrations of SC doses of VGA039 [Срок оценки: From just prior to the start of study drug administration until 49 weeks after SC study drug initiation]
• Incidence of Anti-drug antibodies to VGA039 [Срок оценки: From just prior to the start of study drug administration until 49 weeks after SC study drug initiation]

Критерии участия

Критерии включения

• 12 to 75 years of age, inclusive
• No clinically significant laboratory, ECG, or vital signs results
• Documented diagnosis consistent with VWD of any type
• Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year
• Patients with VWD who are judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes
• Hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 x 109/L at Screening

Критерии исключения

• Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening
• Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study
• Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented
• 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
• Baseline FVIII activity > lower limit of normal (LLN)

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 11 центров

• Arkansas Children's Hospital — Little Rock
• Luskin Orthopaedic Institute For Children — Los Angeles
• UC Davis Medical Center — Sacramento
• University of California San Francisco — San Francisco
• Emory University Hospital — Atlanta
• Children's Healthcare of Atlanta — Atlanta
• University of Minnesota — Minneapolis
• Science 37, Inc. (Virtual Clinical MetaSite) — Morrisville
• … и ещё 3 центра

Грузия · 1 центр

• K Eristavi National Center of Experimental and Clinical Surgery (ქირურგიის ეროვნული ცენტრი — Tbilisi

Германия · 1 центр

• Frankfurt University Hospital (Universitätsmedizin Frankfurt) — Frankfurt

ЮАР · 1 центр

• Charlotte Maxeke Johannesburg Academic Hospital School of Pathology Clinical Haematologist — Johannesburg

Идентификаторы

NCT: NCT07115004 · VGA039-CP002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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