NCT07115004招募中3 期
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)
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 36
- 主要终点
- Incidence of bleeding events [Efficacy]
研究概览
简要总结
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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)
研究组 & 干预措施
Multiple Doses of VGA039 after observational run-in
Experimental
Participants who meet eligibility criteria and complete a 24-week observational phase will receive VGA039 SC for approximately 49 weeks.
干预措施: VGA039 (Drug)
结局指标
主要结局
Incidence of bleeding events [Efficacy]
时间窗: Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation
次要结局
- Incidence of treated bleeding events [Efficacy](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(From informed consent until 49 weeks after SC study drug initiation)
- Plasma concentrations of SC doses of VGA039(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(From just prior to the start of study drug administration until 49 weeks after SC study drug initiation)
研究者
研究点 (36)
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相关资讯
Incyte to Acquire Vega Therapeutics for Up to $2 Billion, Adding Phase 3 Monoclonal Antibody for Von Willebrand Disease- Incyte has agreed to acquire Vega Therapeutics, a Star Therapeutics subsidiary, for $1.25 billion upfront with up to $750 million in sales milestone payments.
- The deal centers on VGA039, a first-in-class, Phase 3 monoclonal antibody targeting Protein S for von Willebrand disease, the most common inherited bleeding disorder.
- VGA039 has the potential to be the first subcutaneous prophylactic therapy with once-monthly dosing, replacing current intravenous infusions required two to three times weekly.
- The acquisition marks Incyte's first under CEO Bill Meury and aims to diversify the company's portfolio ahead of Jakafi's patent expiration in 2028.3 months agoStar Therapeutics Receives FDA Rare Pediatric Disease and Breakthrough Therapy Designations for VGA039 in Von Willebrand Disease- The FDA has granted both rare pediatric disease designation and Breakthrough Therapy designation to Star Therapeutics' VGA039, a once-monthly subcutaneous monoclonal antibody for von Willebrand disease prophylaxis.
- VGA039 targets Protein S with dual actions promoting platelet attachment and enhancing fibrin deposition, potentially serving as a universal hemostatic therapy for all types of von Willebrand disease.
- The designations are supported by interim Phase 1/2 data presented at ASH 2025 showing substantial bleed reductions across all VWD types, with a pivotal Phase 3 trial currently enrolling patients aged 12 and older.
- Von Willebrand disease affects more than 134,000 patients in the United States and is the most common inherited bleeding disorder, with current therapies requiring multiple weekly intravenous infusions.5 months agoStar Therapeutics Reports Promising Phase 1/2 Data for VGA039 in Von Willebrand Disease, Showing 73-100% Reduction in Bleeding Episodes- Star Therapeutics presented interim Phase 1/2 data for VGA039, a first-in-class monoclonal antibody targeting Protein S, demonstrating substantial bleeding reductions across all von Willebrand disease types at the ASH Annual Meeting.
- The once-monthly subcutaneous therapy achieved 73-87% bleeding reduction in treatment-naive patients and 75-100% reduction in patients switching from current IV prophylaxis treatments.
- VGA039 showed favorable safety and tolerability across 16 enrolled patients, with all 8 patients completing treatment transitioning to an open-label extension study.
- The company has initiated a pivotal Phase 3 trial (VIVID-6) for VGA039, which has received FDA Fast Track and orphan drug designations for treating von Willebrand disease.9 months agoStar Therapeutics Reports Positive Phase 1/2 Data for VGA039 in von Willebrand Disease at ASH 2025- Star Therapeutics will present interim data from its Phase 1/2 multidose study of VGA039, showing the novel Protein S-targeting monoclonal antibody is well-tolerated and substantially reduces bleeding rates in von Willebrand disease patients.
- VGA039 offers a convenient subcutaneous, once-monthly dosing regimen that could dramatically reduce treatment burden compared to current therapies requiring multiple weekly intravenous infusions.
- The therapy has received FDA Fast Track and orphan drug designations and has advanced to Phase 3 trials, positioning it as a potential universal hemostatic therapy for multiple bleeding disorders.10 months agoStar Therapeutics Secures $125 Million Series D to Advance VGA039 Phase 3 Trial for von Willebrand Disease- Star Therapeutics raised an oversubscribed $125 million Series D financing co-led by Sanofi Ventures and Viking Global Investors to advance its bleeding disorder pipeline.
- The funding supports VGA039, a first-in-class monoclonal antibody targeting Protein S, which has initiated a pivotal Phase 3 trial for von Willebrand disease treatment.
- VGA039 offers a once-monthly subcutaneous dosing regimen compared to current factor replacement therapies requiring two to three IV infusions per week.
- The therapy has received FDA Fast Track and orphan drug designations and could address all types of von Willebrand disease affecting over 50,000 U.S. patients.11 months ago