A Master Protocol to Evaluate the Safety, Tolerability, and Efficacy of Islet Cell Replacement Therapies in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia: A Phase 1/2/3 Study of VX-880 and a Phase 1/2 Study of VX-017
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 57
- 试验地点
- 52
- 主要终点
- Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
研究概览
简要总结
This study will evaluate the safety, tolerability and efficacy of VX-880 infusion and VX-017 infusion in participants with type 1 diabetes (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical history of T1D with >=5 years of duration of insulin dependence
- •At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment
- •Stable diabetic treatment
- •Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening and willingness to use CGM for the duration of the study
排除标准
- •Prior islet cell transplant, organ transplant, or cell therapy
- •Other protocol-defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
VX-880
干预措施: VX-880 (Biological)
VX-017
干预措施: VX-017 (Biological)
结局指标
主要结局
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
时间窗: From VX-880 infusion to end of study (up to 5 years)
Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)
时间窗: 1 year after achieving insulin independence
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
时间窗: From VX-880 infusion to end of study (at least 5 years)
Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)
时间窗: 1 year after achieving insulin independence (following VX-880 infusion)
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
时间窗: From VX-017 infusion to end of study (at least 5 years)
次要结局
- Parts B and C: Proportion of Participants who are Insulin Independent(At 1 year after VX-880 infusion)
- Parts B and C: Proportion of Participants Free from SHEs with a Glycosylated Hemoglobin (HbA1c) less than (<) 7.0%(At 1 year after VX-880 infusion)
- Parts B and C: Change From Baseline in Glycosylated Hemoglobin (HbA1c)(At 1 year after VX-880 infusion)
- Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year(From VX-880 infusion to end of study (up to 5 years))
- Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)(From Signing of informed consent to end of study (up to 5 years))
- Parts B and C: Proportion of Participants Free from SHEs with a Glycated Hemoglobin (HbA1c) less than (<) 7.0%(SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion)
- Parts B and C: Change From Baseline in Glycated Hemoglobin (HbA1c)(At 1 year after VX-880 infusion)
- Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year(From VX-880 infusion to end of study (at least 5 years))
- Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)(From VX-880 infusion up to end of study (at least 5 years))
- Part D: Change from baseline in peak C-peptide during mixed-meal tolerance test (MMTT)(At Day 90 after VX-017 infusion)