A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes

Phase 1

Active, not recruiting

• Conditions: Type 1 Diabetes
• Interventions: Drug: VX-264

• Registration Number: NCT05791201
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-03-16
• Study First Submitted QC: 2023-03-16
• Study First Posted: 2023-03-30
• Study Start: 2023-05-16
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-16
• Last Update Posted: 2025-05-21
• Primary Completion: 2026-05-29
• Study Completion: 2026-05-29

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 7

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
VX-264	VX-264	-

Primary Outcome Measures

Name	Time	Method
Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	From Day 1 up to 24 months
Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)	From Baseline and at Day 90

Secondary Outcome Measures

Name	Time	Method
Part C: Proportion of Participants who are Insulin Independent at One Point in Time	From Day 180 up to Day 365
Part C: Change in HbA1c values	From Baseline up to 24 months
Part C: Change in Average Total Daily Insulin Dose	From Baseline up to 24 months
Part C: Change in peak C-peptide during MMTT	From Baseline up to 24 months

Trial Locations

Locations (16)

UHealth Diabetes Research Institute; 🇺🇸 Miami, Florida, United States

Montreal Clinical Research Institute; 🇨🇦 Montreal, Canada

Toronto General Hospital (TGH); 🇨🇦 Toronto, Canada

Vancouver General Hospital; 🇨🇦 Vancouver, Canada

University of Chicago; 🇺🇸 Chicago, Illinois, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Hospital of the University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center Montefiore; 🇺🇸 Pittsburgh, Pennsylvania, United States

University of Wisconsin; 🇺🇸 Madison, Wisconsin, United States

University of Alberta, Edmonton; 🇨🇦 Edmonton, Canada

Dresden Center for Islet Transplantation; 🇩🇪 Dresden, Germany

IRCCS Ospedale San Raffaele; 🇮🇹 Milan, Italy

Leiden University; 🇳🇱 Leiden, Netherlands

Hopiteaux Universitaires de Geneve; 🇨🇭 Geneve, Switzerland

Churchill Hospital; 🇬🇧 Headington, United Kingdom

Royal Victoria Infirmary; 🇬🇧 Newcastle Upon Tyne, United Kingdom