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

February 5, 2024 updated by: Vertex Pharmaceuticals Incorporated

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

17

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
      • Edmonton, Canada
        • Recruiting
        • University of Alberta, Edmonton
      • Toronto, Canada
        • Recruiting
        • Toronto General Hospital (TGH)
  • Germany
      • Dresden, Germany
        • Recruiting
        • Dresden Center for Islet Transplantation
  • Italy
      • Milan, Italy
        • Recruiting
        • IRCCS Ospedale San Raffaele
  • Netherlands
      • Leiden, Netherlands
        • Recruiting
        • Leiden University
  • Switzerland
      • Geneve, Switzerland
        • Recruiting
        • Hopiteaux Universitaires de Geneve
  • United Kingdom
      • Headington, United Kingdom
        • Recruiting
        • Churchill Hospital
  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • UHealth Diabetes Research Institute
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Hospital of the University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • University of Pittsburgh Medical Center Montefiore

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VX-264
Drug: VX-264
Allogeneic human stem cell-derived islets.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 16, 2023

Primary Completion (Estimated)

May 1, 2026

Study Completion (Estimated)

May 1, 2026

Study Registration Dates

First Submitted

March 16, 2023

First Submitted That Met QC Criteria

March 16, 2023

First Posted (Actual)

March 30, 2023

Study Record Updates

Last Update Posted (Actual)

February 7, 2024

Last Update Submitted That Met QC Criteria

February 5, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX22-264-101
  • 2022-003318-35 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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