NCT04786262

Brief Summary

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

Trial Health

88
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
52

participants targeted

Target at below P25 for phase_3

Timeline
51mo left

Started Mar 2021

Longer than P75 for phase_3

Geographic Reach
10 countries

29 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress55%
Mar 2021Jun 2030

First Submitted

Initial submission to the registry

March 3, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 8, 2021

Completed
21 days until next milestone

Study Start

First participant enrolled

March 29, 2021

Completed
6.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2027

Expected
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2030

Last Updated

March 17, 2026

Status Verified

March 1, 2026

Enrollment Period

6.3 years

First QC Date

March 3, 2021

Last Update Submit

March 13, 2026

Conditions

Diabetes Mellitus, Type 1Impaired Hypoglycemic AwarenessSevere Hypoglycemia

Outcome Measures

Primary Outcomes (2)

  • Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

    From VX-880 infusion to end of study (up to 5 years)

  • Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)

    1 year after achieving insulin independence

Secondary Outcomes (5)

  • Parts B and C: Proportion of Participants who are Insulin Independent

    At 1 year after VX-880 infusion

  • Parts B and C: Proportion of Participants Free from SHEs with a Glycosylated Hemoglobin (HbA1c) less than (<) 7.0%

    At 1 year after VX-880 infusion

  • Parts B and C: Change From Baseline in Glycosylated Hemoglobin (HbA1c)

    At 1 year after VX-880 infusion

  • Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year

    From VX-880 infusion to end of study (up to 5 years)

  • Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

    From Signing of informed consent to end of study (up to 5 years)

Study Arms (1)

VX-880

EXPERIMENTAL
Biological: VX-880

Interventions

VX-880BIOLOGICAL

Infused into the hepatic portal vein.

Also known as: Zimislecel, Formerly known as STx-02
VX-880

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (29)

City of Hope

Duarte, California, 91010, United States

RECRUITING

University of California San Francisco

San Francisco, California, 94143, United States

RECRUITING

UHealth Diabetes Research Institute

Miami, Florida, 33136, United States

RECRUITING

Northwestern Organ Transplant Center

Chicago, Illinois, 60611, United States

COMPLETED

University of Chicago

Chicago, Illinois, 60637, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Pittsburgh Medical Center Montefiore

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Baylor Scott and White Research Institute

Dallas, Texas, 75246, United States

RECRUITING

VCU Medical Center, Richmond

Richmond, Virginia, 23298, United States

RECRUITING

University of Wisconsin

Madison, Wisconsin, 53792, United States

RECRUITING

University of Alberta, Edmonton

Edmonton, Canada

RECRUITING

McGill University Health Centre

Montreal, Canada

RECRUITING

Toronto General Hospital (TGH)

Toronto, Canada

RECRUITING

Vancouver General Hospital

Vancouver, Canada

RECRUITING

CHU Lille

Lille, France

RECRUITING

Centre de recherche en Biomédecine de Strasbourg

Strasbourg, France

RECRUITING

Dresden Center for Islet Transplantation

Dresden, Germany

RECRUITING

IRCCS Ospedale San Raffaele

Milan, Italy

RECRUITING

Leiden University

Leiden, Netherlands

RECRUITING

Oslo University Hospital

Oslo, Norway

RECRUITING

King Abdullah International Medical Research Center (KAIMRC) - Riyadh - Endocrinology

Riyadh, Saudi Arabia

RECRUITING

King Faisal Specialist Hospital & Research Centre - Riyadh - Endocrinology

Riyadh, Saudi Arabia

RECRUITING

Hopiteaux Universitaires de Geneve

Geneva, Switzerland

RECRUITING

Churchill Hospital

Headington, Oxford, United Kingdom

RECRUITING

Royal Infirmary of Edinburgh

Edinburgh, United Kingdom

RECRUITING

Cardiovascular, Metabolic Medicine and Sciences, King's College London

London, United Kingdom

RECRUITING

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, United Kingdom

RECRUITING

Related Publications (1)

  • Reichman TW, Markmann JF, Odorico J, Witkowski P, Fung JJ, Wijkstrom M, Kandeel F, de Koning EJP, Peters AL, Mathieu C, Kean LS, Bruinsma BG, Wang C, Mascia M, Sanna B, Marigowda G, Pagliuca F, Melton D, Ricordi C, Rickels MR; VX-880-101 FORWARD Study Group. Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes. N Engl J Med. 2025 Sep 4;393(9):858-868. doi: 10.1056/NEJMoa2506549. Epub 2025 Jun 20.

    PMID: 40544428DERIVED

MeSH Terms

Conditions

Diabetes Mellitus, Type 1Hypoglycemia

Condition Hierarchy (Ancestors)

Diabetes MellitusGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System Diseases

Central Study Contacts

Medical Information

CONTACT

617-341-6777medicalinfo@vrtx.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 3, 2021

First Posted

March 8, 2021

Study Start

March 29, 2021

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

June 30, 2030

Last Updated

March 17, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

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

Locations

SA(2)CH(1)GB(4)NO(1)DE(1)FR(2)IT(1)CA(4)US(12)NL(1)