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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05791201
Recruitment Status : Recruiting
First Posted : March 30, 2023
Last Update Posted : May 2, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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).

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Drug: VX-264 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 17 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
Actual Study Start Date : May 16, 2023
Estimated Primary Completion Date : May 2026
Estimated Study Completion Date : May 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arms and Interventions
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Arm Intervention/treatment
Experimental: VX-264 Drug: VX-264
Allogeneic human stem cell-derived islets.


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
  2. 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 Measures :
  1. Part C: Change in peak C-peptide during MMTT [ Time Frame: From Baseline up to 24 months ]
  2. Part C: Change in Average Total Daily Insulin Dose [ Time Frame: From Baseline up to 24 months ]
  3. Part C: Proportion of Participants who are Insulin Independent at One Point in Time [ Time Frame: From Day 180 up to Day 365 ]
  4. Part C: Change in HbA1c values [ Time Frame: From Baseline up to 24 months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Other protocol defined Inclusion/Exclusion criteria may apply.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05791201


Contacts
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Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

Locations
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United States, Florida
UHealth Diabetes Research Institute Recruiting
Miami, Florida, United States, 33136
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
United States, Pennsylvania
Hospital of the University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
University of Pittsburgh Medical Center Montefiore Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Canada
University of Alberta, Edmonton Recruiting
Edmonton, Canada
Toronto General Hospital (TGH) Recruiting
Toronto, Canada
Germany
Dresden Center for Islet Transplantation Recruiting
Dresden, Germany
Italy
IRCCS Ospedale San Raffaele Recruiting
Milan, Italy
Netherlands
Leiden University Recruiting
Leiden, Netherlands
Switzerland
Hopiteaux Universitaires de Geneve Recruiting
Geneve, Switzerland
United Kingdom
Churchill Hospital Recruiting
Headington, United Kingdom
Royal Victoria Infirmary Recruiting
Newcastle Upon Tyne, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05791201    
Other Study ID Numbers: VX22-264-101
2022-003318-35 ( EudraCT Number )
First Posted: March 30, 2023    Key Record Dates
Last Update Posted: May 2, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
 Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases