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

NCT05791201 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: VX22-264-1012024-515583-32-00
• Study Type: interventional
• Target: 7
• Locations: 7 countries
• Sites: 16

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).

Conditions

Type 1 Diabetes

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (4)

• Part C: Change in peak C-peptide during MMTT

  From Baseline up to 24 months

• Part C: Change in Average Total Daily Insulin Dose

  From Baseline up to 24 months

• 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

Study Arms (1)

VX-264

EXPERIMENTAL

Drug: VX-264

Interventions

VX-264 DRUG

Allogeneic human stem cell-derived islets.

VX-264

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Prior islet cell transplant, organ transplant, or cell therapy

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (16)

UHealth Diabetes Research Institute

Miami, Florida, 33136, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh Medical Center Montefiore

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

University of Alberta, Edmonton

Edmonton, Canada

Location

Montreal Clinical Research Institute

Montreal, Canada

Location

Toronto General Hospital (TGH)

Toronto, Canada

Location

Vancouver General Hospital

Vancouver, Canada

Location

Dresden Center for Islet Transplantation

Dresden, Germany

Location

IRCCS Ospedale San Raffaele

Milan, Italy

Location

Leiden University

Leiden, Netherlands

Location

Hopiteaux Universitaires de Geneve

Geneva, Switzerland

Location

Churchill Hospital

Headington, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, United Kingdom

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2023
• First Posted: March 30, 2023
• Study Start: May 16, 2023
• Primary Completion (Estimated): May 29, 2026
• Study Completion (Estimated): May 29, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); GB (2); DE (1); CA (4); IT (1); NL (1); CH (1)