Clinical trial NCT05791201: A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
Official title: A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Type 1 Diabetes
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2023-03-30
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).


Eligibility criteria: Key Inclusion Criteria:

Clinical history of T1D with greater than or equal to (\>=) 5 years duration Participant is on a stable diabetic treatment Consistent use of continuous glucose monitoring (CGM) for at least 4 weeks 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

Primary outcomes:
measure: Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From Day 1 up to 24 months
measure: Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
timeFrame: From Baseline and at Day 90
Secondary outcomes:
measure: Part C: Change in peak C-peptide during MMTT
timeFrame: From Baseline up to 24 months
measure: Part C: Change in Average Total Daily Insulin Dose
timeFrame: From Baseline up to 24 months
measure: Part C: Proportion of Participants who are Insulin Independent at One Point in Time
timeFrame: From Day 180 up to Day 365
measure: Part C: Change in HbA1c values
timeFrame: From Baseline up to 24 months
Other outcomes:
Locations:
city: Miami
country: United States
facility: UHealth Diabetes Research Institute
state: Florida
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center Montefiore
state: Pennsylvania
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Edmonton
country: Canada
facility: University of Alberta, Edmonton
city: Montreal
country: Canada
facility: Montreal Clinical Research Institute
city: Toronto
country: Canada
facility: Toronto General Hospital (TGH)
city: Vancouver
country: Canada
facility: Vancouver General Hospital
city: Dresden
country: Germany
facility: Dresden Center for Islet Transplantation
city: Milan
country: Italy
facility: IRCCS Ospedale San Raffaele
city: Leiden
country: Netherlands
facility: Leiden University
city: Geneve
country: Switzerland
facility: Hopiteaux Universitaires de Geneve
city: Headington
country: United Kingdom
facility: Churchill Hospital
city: Newcastle Upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
Study officials:
References: