Type 1 DiabetesPhase 1/2NCT05791201
A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
A Phase 1/2 study testing VX-264 in people with Type 1 Diabetes, run by Vertex Pharmaceuticals Incorporated at 16 sites in 7 countries.
Trial summary
The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Type 1 Diabetes
- What is being tested?
- VX-264
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 16 sitesin 7 countries
- Study dates
- 16 May 2023to 12 Mar 2027
- Phase
- Phase 1/2
- 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 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
Who cannot join
- Prior islet cell transplant, organ transplant, or cell therapy
Trial locations and contacts
16 sites in 7 countries.
16 sites in 7 countries
United States6 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Chicago
Chicago, Illinois, 60637
University of Wisconsin
Madison, Wisconsin, 53792
UHealth Diabetes Research Institute
Miami, Florida, 33136
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
University of Pittsburgh Medical Center Montefiore
Pittsburgh, Pennsylvania, 15213
Canada4 sites
University of Alberta, Edmonton
Edmonton
Montreal Clinical Research Institute
Montreal
Toronto General Hospital (TGH)
Toronto
Vancouver General Hospital
Vancouver
United Kingdom2 sites
Royal Victoria Infirmary
Newcastle upon Tyne
Churchill Hospital
Oxford
About this study
Official title: A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).
- Conditions
- Type 1 Diabetes
- Being tested
- VX-264
- Medical subject headings
- Diabetes Mellitus, Type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-264Drug
Allogeneic human stem cell-derived islets.
| Study group | Role | What they receive |
|---|---|---|
| VX-264 | Experimental | Not described Receives: Drug: VX-264 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: From Day 1 up to 24 months
Part B: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
Measured: From Baseline and at Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2023
- Study start16 May 2023
- Primary completion12 Mar 2027 (estimated)
- Study completion12 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 DiabetesStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05791201
- Sponsor's protocol ID
- VX22-264-101
- Other IDs
- 2024-515583-32-00
- Status last verified
- Oct 2026
- First submitted
- 16 Mar 2023
- Last updated
- 9 Oct 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/
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
- ClinicalTrials.gov ID
- NCT05791201
- Acronym / sponsor ID
- VX22-264-101
- Conditions
- Type 1 Diabetes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 16 May 2023 (actual)
- Primary completion
- 12 Mar 2027 (estimated)
- Study completion
- 12 Mar 2027 (estimated)
- First posted
- 30 Mar 2023 (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.
The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VX-264 | Experimental | Drug: VX-264 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 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 (1 listed)
- Prior islet cell transplant, organ transplant, or cell therapy
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-264Allogeneic human stem cell-derived islets. | Drug | VX-264 |
Primary outcome
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
Part B: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
Time frame: From Baseline and at Day 90
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 9 Oct 2026.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
16 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VX-264 in people with Type 1 Diabetes, run by Vertex Pharmaceuticals Incorporated at 16 sites in 7 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 1 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada, United Kingdom, Germany, Italy and 2 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Oct 2026.