Diabetes Mellitus, Type 1Phase 3NCT04786262
A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes
A Phase 3 study testing VX-880 and VX-017 in people with Diabetes Mellitus, Type 1 and Impaired Hypoglycemic Awareness, run by Vertex Pharmaceuticals Incorporated at 29 sites in 10 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia
- What is being tested?
- VX-880 and VX-017
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 29 sitesin 10 countries
- Study dates
- 29 Mar 2021to 31 Dec 2031
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- -Prior islet cell transplant, organ transplant, or cell therapy
- Other protocol-defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
29 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
29 sites in 10 countries
United States12 sites, 10 recruiting
- Completed
Johns Hopkins University
Baltimore, Maryland, 21287
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
- Completed
Northwestern Organ Transplant Center
Chicago, Illinois, 60611
- Recruiting
University of Chicago
Chicago, Illinois, 60637
- Recruiting
Baylor Scott and White Research Institute
Dallas, Texas, 75246
- Recruiting
City of Hope
Duarte, California, 91010
- Recruiting
University of Wisconsin
Madison, Wisconsin, 53792
- Recruiting
UHealth Diabetes Research Institute
Miami, Florida, 33136
- Recruiting
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Recruiting
University of Pittsburgh Medical Center Montefiore
Pittsburgh, Pennsylvania, 15213
- Recruiting
VCU Medical Center, Richmond
Richmond, Virginia, 23298
- Recruiting
University of California San Francisco
San Francisco, California, 94143
Canada4 sites, 4 recruiting
- Recruiting
University of Alberta, Edmonton
Edmonton
- Recruiting
McGill University Health Centre
Montreal
- Recruiting
Toronto General Hospital (TGH)
Toronto
- Recruiting
Vancouver General Hospital
Vancouver
United Kingdom4 sites, 4 recruiting
- Recruiting
Royal Infirmary of Edinburgh
Edinburgh
- Recruiting
Churchill Hospital
Headington, Oxford
- Recruiting
Cardiovascular, Metabolic Medicine and Sciences, King's College London
London
- Recruiting
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne
France2 sites, 2 recruiting
- Recruiting
CHU Lille
Lille
- Recruiting
Centre de recherche en Biomédecine de Strasbourg
Strasbourg
Saudi Arabia2 sites, 2 recruiting
- Recruiting
King Abdullah International Medical Research Center (KAIMRC) - Riyadh - Endocrinology
Riyadh
- Recruiting
King Faisal Specialist Hospital & Research Centre - Riyadh - Endocrinology
Riyadh
Germany1 site, 1 recruiting
- Recruiting
Dresden Center for Islet Transplantation
Dresden
About this study
Official title: 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.
- Being tested
- VX-880, VX-017
- Medical subject headings
- Diabetes Mellitus, Type 1, Hypoglycemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-880Biological
Also called Zimislecel, Formerly known as STx-02
Infused into the hepatic portal vein.
VX-017Biological
Infused into the hepatic portal vein.
| Study group | Role | What they receive |
|---|---|---|
| VX-880 | Experimental | Not described Receives: Biological: VX-880 |
| VX-017 | Experimental | Not described Receives: Biological: VX-017 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Measured: 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)
Measured: 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)
Measured: From VX-017 infusion to end of study (at least 5 years)
Secondary outcomes (6)
Parts B and C: Proportion of Participants who are Insulin Independent
Measured: 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%
Measured: 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)
Measured: At 1 year after VX-880 infusion
Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year
Measured: 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)
Measured: 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)
Measured: At Day 90 after VX-017 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2021
- Study start29 Mar 2021
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 57 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04786262
- Sponsor's protocol ID
- VX20-880-101
- Other IDs
- 2024-513929-23-00
- Status last verified
- Aug 2026
- First submitted
- 3 Mar 2021
- Last updated
- 1 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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/
Publications
Brown ME, Barra JM, Pina MR, Proia J, Brusko TM, Russ HA. Human stem cell-derived beta cells expressing an optimized CD155 reduce cytotoxic immune cell function for application in type 1 diabetes. Sci Adv. 2025 Nov 7;11(45):eadx9755. doi: 10.1126/sciadv.adx9755. Epub 2025 Nov 7.
PubMed 41202120Reichman 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.
PubMed 40544428
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04786262
- Acronym / sponsor ID
- VX20-880-101
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 57 participants (estimated)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 29 Mar 2021 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2031 (estimated)
- First posted
- 8 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VX-880 | Experimental | Biological: VX-880 |
| VX-017 | Experimental | Biological: VX-017 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (2 listed)
- -Prior islet cell transplant, organ transplant, or cell therapy
- Other protocol-defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-880Also: Zimislecel, Formerly known as STx-02Infused into the hepatic portal vein. | Biological | VX-880 |
| VX-017Infused into the hepatic portal vein. | Biological | VX-017 |
Primary outcome
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)
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)
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)
6 secondary outcomes
Parts B and C: Proportion of Participants who are Insulin Independent
Time frame: 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
Parts B and C: Change From Baseline in Glycated Hemoglobin (HbA1c)
Time frame: 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)
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)
Part D: Change from baseline in peak C-peptide during mixed-meal tolerance test (MMTT)
Time frame: At Day 90 after VX-017 infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
- Derived from this study: PubMed 41202120Brown ME, Barra JM, Pina MR, Proia J, Brusko TM, Russ HA. Human stem cell-derived beta cells expressing an optimized CD155 reduce cytotoxic immune cell function for application in type 1 diabetes. Sci Adv. 2025 Nov 7;11(45):eadx9755. doi: 10.1126/sciadv.adx9755. Epub 2025 Nov 7.
- Derived from this study: PubMed 40544428Reichman 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.
- Individual participant dataSharing planned: No. 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/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
29 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing VX-880 and VX-017 in people with Diabetes Mellitus, Type 1 and Impaired Hypoglycemic Awareness, run by Vertex Pharmaceuticals Incorporated at 29 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Canada, United Kingdom, France, Saudi Arabia and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical Information (617-341-6777, medicalinfo@vrtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04786262, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.