A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes

August 27, 2026 updated by: Vertex Pharmaceuticals Incorporated

A Master Protocol to Evaluate the Safety, Tolerability, and Efficacy of Islet Cell Replacement Therapies in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia: A Phase 1/2/3 Study of VX-880 and a Phase 1/2 Study of VX-017

This study will evaluate the safety, tolerability and efficacy of VX-880 infusion and VX-017 infusion in participants with type 1 diabetes (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

57

Phase

  • Phase 3

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
      • Montreal, Canada
        • Recruiting
        • McGill University Health Centre
      • Toronto, Canada
        • Recruiting
        • Toronto General Hospital (TGH)
      • Vancouver, Canada
        • Recruiting
        • Vancouver General Hospital
  • France
      • Lille, France
        • Recruiting
        • CHU Lille
      • Strasbourg, France
        • Recruiting
        • Centre de recherche en Biomédecine de Strasbourg
  • Germany
      • Dresden, Germany
        • Recruiting
        • Dresden Center for Islet Transplantation
  • Italy
      • Milan, Italy
        • Recruiting
        • IRCCS Ospedale San Raffaele
  • Netherlands
      • Leiden, Netherlands
        • Recruiting
        • Leiden University
  • Norway
      • Oslo, Norway
        • Recruiting
        • Oslo University Hospital
  • Saudi Arabia
      • Riyadh, Saudi Arabia
        • Recruiting
        • King Abdullah International Medical Research Center (KAIMRC) - Riyadh - Endocrinology
      • Riyadh, Saudi Arabia
        • Recruiting
        • King Faisal Specialist Hospital & Research Centre - Riyadh - Endocrinology
  • Switzerland
      • Geneva, Switzerland
        • Completed
        • Hopiteaux Universitaires de Geneve
  • United Kingdom
      • Edinburgh, United Kingdom
        • Recruiting
        • Royal Infirmary of Edinburgh
      • London, United Kingdom
        • Recruiting
        • Cardiovascular, Metabolic Medicine and Sciences, King's College London
      • Newcastle upon Tyne, United Kingdom
        • Recruiting
        • The Newcastle upon Tyne Hospitals NHS Foundation Trust
    • Oxford
      • Headington, Oxford, United Kingdom
        • Recruiting
        • Churchill Hospital
  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California San Francisco
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • UHealth Diabetes Research Institute
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago
      • Chicago, Illinois, United States, 60611
        • Completed
        • Northwestern Organ Transplant Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Completed
        • Johns Hopkins University
    • 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
    • Texas
      • Dallas, Texas, United States, 75246
        • Recruiting
        • Baylor Scott and White Research Institute
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • VCU Medical Center, Richmond
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Recruiting
        • University of Wisconsin

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 >=5 years of duration of insulin dependence
  • At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment
  • Stable diabetic treatment
  • Consistent use of continuous glucose monitor (CGM) for at least 3 months 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: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VX-880
Biological: VX-880
Infused into the hepatic portal vein.
Other Names:
  • Formerly known as STx-02
  • Zimislecel
Experimental: VX-017
Biological: VX-017
Infused into the hepatic portal vein.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-880 infusion to end of study (at least 5 years)
From VX-880 infusion to end of study (at least 5 years)
Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)
Time Frame: 1 year after achieving insulin independence (following VX-880 infusion)
1 year after achieving insulin independence (following VX-880 infusion)
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-017 infusion to end of study (at least 5 years)
From VX-017 infusion to end of study (at least 5 years)

Secondary Outcome Measures

Outcome Measure
Time Frame
Parts B and C: Proportion of Participants who are Insulin Independent
Time Frame: At 1 year after VX-880 infusion
At 1 year after VX-880 infusion
Parts B and C: Proportion of Participants Free from SHEs with a Glycated Hemoglobin (HbA1c) less than (<) 7.0%
Time Frame: SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion
SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion
Parts B and C: Change From Baseline in Glycated Hemoglobin (HbA1c)
Time Frame: At 1 year after VX-880 infusion
At 1 year after VX-880 infusion
Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year
Time Frame: From VX-880 infusion to end of study (at least 5 years)
From VX-880 infusion to end of study (at least 5 years)
Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-880 infusion up to end of study (at least 5 years)
From VX-880 infusion up to end of study (at least 5 years)
Part D: Change from baseline in peak C-peptide during mixed-meal tolerance test (MMTT)
Time Frame: At Day 90 after VX-017 infusion
At Day 90 after VX-017 infusion

Collaborators and Investigators

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

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

March 29, 2021

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2031

Study Registration Dates

First Submitted

March 3, 2021

First Submitted That Met QC Criteria

March 3, 2021

First Posted (Actual)

March 8, 2021

Study Record Updates

Last Update Posted (Actual)

September 1, 2026

Last Update Submitted That Met QC Criteria

August 27, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX20-880-101
  • 2024-513929-23-00 (Other Identifier: EU Trial (CTIS) 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/our-science/clinical-trials-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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