A Phase 1/2 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
- Conditions
- Type 1 Diabetes Mellitus with Impaired Hypoglycemic Awareness and Severe HypoglycemiaMedDRA version: 20.0Level: PTClassification code 10012601Term: Diabetes mellitusSystem Organ Class: 10027433 - Metabolism and nutrition disordersMedDRA version: 21.1Level: LLTClassification code 10081605Term: Severe hypoglycemiaSystem Organ Class: 10027433 - Metabolism and nutrition disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2022-002292-11-IT
- Lead Sponsor
- VERTEX PHARMACEUTICALS INCORPORATED
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 17
1. Clinical history and laboratory evidence of T1D
2. 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.
3. Reduced awareness of hypoglycemia at Screening
4. Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening
5. Compatible blood group (A or AB)
Please refer to Section 8.1 of the Protocol for additional inclusion criteria
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
1. Prior islet cell transplant, organ transplant, or cell therapy
2. Advanced complications associated with diabetes including untreated proliferative retinopathy, skin ulcers, or amputations attributable to diabetes.
3. 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%
4. 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.
5. Negative screen for Epstein-Barr virus (EBV) by immunoglobulin G (IgG) determination
Please refer to Section 8.2 of the Protocol for additional exclusion criteria
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluate the efficacy of VX-880 infusion in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia;Secondary Objective: - Evaluate the efficacy of VX-880 infusion on insulin independence<br>- Evaluate VX-880 islet cell function over time<br>- Evaluate the safety and tolerability of VX-880 infusion in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia;Primary end point(s): Part A<br>• Safety and tolerability assessments<br>Part B<br>• Safety and tolerability assessments<br>• Number of subjects with peak C-peptide =100 pmol/L during mixed meal tolerance test (MMTT)<br>Part C<br>• Proportion of subjects free of severe hypoglycemic events (SHEs) from Day 90 through Day 365 (inclusive) and with either a glycosylated hemoglobin (HbA1c) <7.0% or a =1% reduction in HbA1c from baseline (at one time point) between Day 180 and Day 365 after VX-880 infusion;Timepoint(s) of evaluation of this end point: Please refer to the protocol
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Proportion of subjects who are insulin independent (at one timepoint) between Day 180 and Day 365 after VX-880 infusion<br>• Proportion of subjects with peak C-peptide =100 pmol/L during MMTT over time.<br>• Safety and tolerability assessments;Timepoint(s) of evaluation of this end point: Please refer to the protocol