Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia
Conditions
Brief summary
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.
Interventions
BIOLOGICALVX-880
Infused into the hepatic portal vein.
BIOLOGICALVX-017
Infused into the hepatic portal vein.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * 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 Key
Exclusion criteria
-Prior islet cell transplant, organ transplant, or cell therapy Other protocol-defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | 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) | 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) | From VX-017 infusion to end of study (at least 5 years) |
Secondary
| Measure | Time frame |
|---|---|
| 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 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 |
Countries
Canada, France, Germany, Italy, Netherlands, Norway, Saudi Arabia, Switzerland, United Kingdom, United States
Contacts
CONTACTMedical Information
Outcome results
None listed