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

Primary Purpose

Type 1 Diabetes

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VX-264
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Type 1 Diabetes

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria: Clinical history of T1D with greater than (>) 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.

Sites / Locations

  • UHealth Diabetes Research InstituteRecruiting
  • University of ChicagoRecruiting
  • Massachusetts General HospitalRecruiting
  • University of Pittsburgh Medical Center MontefioreRecruiting
  • University of Alberta, EdmontonRecruiting
  • Toronto General Hospital (TGH)Recruiting
  • Leiden UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-264

Arm Description

Outcomes

Primary Outcome Measures

Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)

Secondary Outcome Measures

Part C: Change in peak C-peptide during MMTT
Part C: Change in Average Total Daily Insulin Dose
Part C: Proportion of Participants who are Insulin Independent at One Point in Time
Part C: Change in HbA1c values

Full Information

First Posted
March 16, 2023
Last Updated
October 17, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05791201
Brief Title
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

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 16, 2023 (Actual)
Primary Completion Date
May 2026 (Anticipated)
Study Completion Date
May 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VX-264
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
VX-264
Intervention Description
Allogeneic human stem cell-derived islets.
Primary Outcome Measure Information:
Title
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
Title
Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
Time Frame
From Baseline and at Day 90
Secondary Outcome Measure Information:
Title
Part C: Change in peak C-peptide during MMTT
Time Frame
From Baseline up to 24 months
Title
Part C: Change in Average Total Daily Insulin Dose
Time Frame
From Baseline up to 24 months
Title
Part C: Proportion of Participants who are Insulin Independent at One Point in Time
Time Frame
From Day 180 up to Day 365
Title
Part C: Change in HbA1c values
Time Frame
From Baseline up to 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Clinical history of T1D with greater than (>) 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
UHealth Diabetes Research Institute
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Individual Site Status
Recruiting
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Pittsburgh Medical Center Montefiore
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Alberta, Edmonton
City
Edmonton
Country
Canada
Individual Site Status
Recruiting
Facility Name
Toronto General Hospital (TGH)
City
Toronto
Country
Canada
Individual Site Status
Recruiting
Facility Name
Leiden University
City
Leiden
Country
Netherlands
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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

Learn more about this trial

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

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