A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
概览
- 阶段
- 1/2 期
- 干预措施
- 未指定
- 疾病 / 适应症
- 未指定
- 发起方
- Vertex Pharmaceuticals Inc., Vertex Pharmaceuticals Inc.
- 入组人数
- 4
- 试验地点
- 4
- 主要终点
- Part A: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues.
- 状态
- 进行中（未招募）
- 最后更新
- 去年
概览
简要总结
- Evaluate the safety and tolerability of VX-264 in subjects with T1D (Part A and Part B).
- Evaluate VX-264 function in subjects with T1D (Part B and Part C).
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.
入排标准
入选标准
- •Subjects (male and female) between the ages of 18 and 65 years (inclusive) on the date of first informed consent.
- •HbA1c ≥6.0% and ≤9.5%.
- •Clinical history and laboratory evidence of T1D based on the American Diabetes Association/European Association for the Study of Diabetes algorithm 13 for investigation of suspected T1D including: o insulin dependence for ≥5 years at time of Screening, and o peak stimulated C-peptide level during MMTT <50 pmol/L (<0.15 ng/mL)
- •Consistent use of CGM for at least 4 weeks before Screening and willingness to use only the study-provided CGM for the duration of the study.
- •Body habitus supportive of implantation of the planned number of VX- 264 units per study part and/or cohort.
排除标准
- •Prior islet cell transplant, organ transplant, or cell therapy.
- •Advanced complications associated with diabetes including untreated proliferative retinopathy, diabetic nephropathy, skin ulcers, or amputations attributable to diabetes.
- •Insulin requirement: a) Parts A and B >40 U/day, or <10 U/day b) Part C: >55 U/day, or <10 U/day c) >0.8 U/kg/day.
- •Subjects with ≥2 or more episodes of severe hypoglycemia in the 12 months prior to signing of informed consent at Screening.
- •Previous abdominal or abdominal wall surgery, or history of peritonitis, that can impact VX-264 placement or put the patient at higher risk of surgical complications.
结局指标
主要结局
Part A: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues.
Part A: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues.
Part B: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12- lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues.
Part B: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12- lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues.
Part C: Change from baseline in peak C-peptide during MMTT at Day 90.
Part C: Change from baseline in peak C-peptide during MMTT at Day 90.
次要结局
- Part C: Change from baseline in peak C-peptide during MMTT over time
- Part C: Change from baseline in C-peptide AUC during MMTT over time
- Part C: Proportion of subjects with peak C-peptide ≥100, ≥200, ≥400, and ≥ 1000 pmol/L (≥0.30, ≥0.60, ≥1.21, and ≥3.02 ng/mL) during MMTT over time
- Part C: Change from baseline in average total daily insulin dose over time
- Part C: Proportion of subjects with a reduction of at least 50% insulin dose from baseline and HbA1c <7.0% at Day 365
- Part C: Proportion of subjects who are insulin independent at one point in time between Day 180 and Day 365
- Part C: Change from baseline on metabolic control: HbA1c values; continuous glucose monitoring (CGM)-derived time-in-range (TIR) over time; glucose variability (CGM, CV%); and glucose management indicator (GMI)
- Part C: Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12- lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues