A Phase 1/2/3 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Part A - Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings
研究概览
简要总结
Evaluate the efficacy of VX-880 infusion in subjects who have T1D with impaired hypoglycemic awareness (IAH) and severe hypoglycemia
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomized
- 主要目的
- Phase 1/2 Study
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Clinical history and laboratory evidence of T1D
- •At least 2 episodes of severe hypoglycemia (confirmed by independent adjudication for subjects in Parts B and C) in the 12 months prior to signing of informed consent at Screening.
- •Reduced awareness of hypoglycemia at Screening
- •Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening. In regions where CGM is not standard of care for T1D, subjects are exempted from the requirement for use of CGM before Screening.
- •Compatible blood group (A or AB)
排除标准
- •Prior islet cell transplant, organ transplant, or cell therapy
- •Advanced complications associated with diabetes including untreated proliferative retinopathy, skin ulcers, or amputations attributable to diabetes.
- •Subjects who have any 1 of the following criteria: o Insulin requirements: >0.8 U/(kg*day), >55 U/day, or <10 U/day; o HbA1c: <6.0% or >9.5%
- •Clinically significant active infection or chronic infection such as hepatitis B, hepatitis C, human immunodeficiency virus (HIV), and/or tuberculosis (TB); or invasive aspergillus, histoplasmosis, or coccidioidomycosis infection within 1 year prior to signing of informed consent at Screening.
- •Negative screen for Epstein-Barr virus (EBV) by immunoglobulin G (IgG) determination
结局指标
主要结局
Part A - Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings
Part A - Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings
Part B and C - The following endpoints apply to the analysis of all infused subjects who intended to receive 0.8 × 10E9 total SC-islet cells with 1 infusion: •Proportion of subjects who are insulin independent at Day 365 after VX-880 infusion
Part B and C - The following endpoints apply to the analysis of all infused subjects who intended to receive 0.8 × 10E9 total SC-islet cells with 1 infusion: •Proportion of subjects who are insulin independent at Day 365 after VX-880 infusion
次要结局
- Part B and C: Secondary Endpoints - Proportion of subjects free of SHEs from Day 90 through Day 365 (inclusive) and HbA1c <7.0% at Day 365 after VX 880 infusion
- Part B and C: Secondary Endpoints - Change from baseline in HbA1c at Day 365 after VX-880 infusion
- Part B and C: Secondary Endpoints- Proportion of subjects who achieve insulin independence and are insulin independent 12 months later, with absence of SHEs
- Part B and C: Secondary Endpoints- Proportion of subjects who maintain insulin independence for at least 1 year
- Part B and C: afety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), clinical laboratory values, vital signs, standard 12-lead ECGs, and imaging findings
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.