Diabetes MellitusPhase 1NCT05565248
An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D
A Phase 1 study testing VCTX211 in people with Diabetes Mellitus and Diabetes Mellitus, Type 1, run by CRISPR Therapeutics AG at 2 sites in Canada.
Trial summary
This is an open-label, multicenter, Phase 1 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders
- What is being tested?
- VCTX211
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 2 sitesCanada
- Study dates
- 20 Jan 2023to 8 Aug 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Diagnosis of T1D for a minimum of 5 years
- Stable diabetes regimen for at least 3 months prior to enrollment.
Who cannot join
- Medical history of islet cell, kidney, and/or pancreas transplant
- Occurrence of 2 or more severe, unexplained hypoglycemic events within 6 months prior to enrollment
- Known causes of diabetes other than T1D
- Immunosuppressant therapy in the previous 30 days and/or requirements for chronic immunosuppressive therapy during the study
- Prior treatment with gene therapy or edited product
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
University of Alberta
Edmonton, Alberta
University of British Columbia
Vancouver, British Columbia
About this study
Official title: An Open-Label, First-in-Human Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
This is an open-label, multicenter, Phase 1 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
Read the detailed description
VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster Regularly Interspaced Short Palindromic Repeats/ CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain PEC211 cells.
- Conditions
- Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders, Metabolic Disease, Endocrine System Diseases, Autoimmune Diseases
- Being tested
- VCTX211
- Keywords
- Type 1 Diabetes, T1D, Allogeneic, Combination Device, CRISPR-Cas9, Cell Therapy, T1DM, Diabetes, VCTX
- Medical subject headings
- Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders, Metabolic Diseases, Endocrine System Diseases, Autoimmune Diseases, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VCTX211Combination product
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device
| Study group | Role | What they receive |
|---|---|---|
| VCTX211 unit | Experimental | Not described Receives: Combination Product: VCTX211 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.
Measured: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.
Measured: From implantation up to 12 months post implantation
Secondary outcomes (10)
Incidence of adverse events reported in patients implanted with VCTX211 units.
Measured: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline.
Measured: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline.
Measured: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline.
Measured: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline.
Measured: From implantation up to 12 months post implantation
Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft.
Measured: From implantation up to 12 months post implantation
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Measured: From implantation up to 12 months post implantation
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Measured: From implantation up to 12 months post implantation
The percentage of viable graft cells per unit using immunohistochemical staining.
Measured: From implantation up to 12 months post implantation
The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.
Measured: From implantation up to 12 months post implantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2022
- Study start20 Jan 2023
- Primary completion8 Aug 2025
- Study completion8 Aug 2025
Why it stopped: Patients to be followed up in the VCTX-201 study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Diabetes MellitusStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- CRISPR Therapeutics AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ViaCyte
- Responsible party
- Sponsor
- Investigators
- Manasi Jaiman, MD, MPH, Study director, ViaCyteSandeep Soni, MD, Study director, CRISPR Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05565248
- Sponsor's protocol ID
- VCTX211-101
- Status last verified
- May 2026
- First submitted
- 30 Sep 2022
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Lin TM, Chang HF, Lin TC, Lin CH, Sun YL, Lin CS. Gene Therapy and Gene Editing in Type 1 Diabetes: CRISPR-Based beta-Cell Replacement and Treg Immune Modulation Approaches. Diabetes Obes Metab. 2026 Jul;28(7):5476-5491. doi: 10.1111/dom.70800. Epub 2026 Apr 23.
PubMed 42023429
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, First-in-Human Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
- ClinicalTrials.gov ID
- NCT05565248
- Acronym / sponsor ID
- VCTX211-101
- Conditions
- Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders, Metabolic Disease, Endocrine System Diseases, Autoimmune Diseases
- Keywords
- Type 1 Diabetes, T1D, Allogeneic, Combination Device, CRISPR-Cas9, Cell Therapy, T1DM, Diabetes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Patients to be followed up in the VCTX-201 study
- Enrolment
- 5 participants (actual)
- Lead sponsor
- CRISPR Therapeutics AGIndustry (pharma, biotech, devices)
- Collaborators
- ViaCyte
- Study start
- 20 Jan 2023 (actual)
- Primary completion
- 8 Aug 2025 (actual)
- Study completion
- 8 Aug 2025 (actual)
- First posted
- 4 Oct 2022 (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 is an open-label, multicenter, Phase 1 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
Detailed description
VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster Regularly Interspaced Short Palindromic Repeats/ CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain PEC211 cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VCTX211 unit | Experimental | Combination Product: VCTX211 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of T1D for a minimum of 5 years
- Stable diabetes regimen for at least 3 months prior to enrollment.
Key exclusion criteria (5 listed)
- Medical history of islet cell, kidney, and/or pancreas transplant
- Occurrence of 2 or more severe, unexplained hypoglycemic events within 6 months prior to enrollment
- Known causes of diabetes other than T1D
- Immunosuppressant therapy in the previous 30 days and/or requirements for chronic immunosuppressive therapy during the study
- Prior treatment with gene therapy or edited product
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 |
|---|---|---|
| VCTX211CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device | Combination product | VCTX211 unit |
Primary outcome
Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.
Time frame: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.
Time frame: From implantation up to 12 months post implantation
10 secondary outcomes
Incidence of adverse events reported in patients implanted with VCTX211 units.
Time frame: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline.
Time frame: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline.
Time frame: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline.
Time frame: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline.
Time frame: From implantation up to 12 months post implantation
Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft.
Time frame: From implantation up to 12 months post implantation
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Time frame: From implantation up to 12 months post implantation
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Time frame: From implantation up to 12 months post implantation
The percentage of viable graft cells per unit using immunohistochemical staining.
Time frame: From implantation up to 12 months post implantation
The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.
Time frame: From implantation up to 12 months post implantation
Results and safety
The study was stopped early (reason given: Patients to be followed up in the VCTX-201 study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2026.
- Derived from this study: PubMed 42023429Lin TM, Chang HF, Lin TC, Lin CH, Sun YL, Lin CS. Gene Therapy and Gene Editing in Type 1 Diabetes: CRISPR-Based beta-Cell Replacement and Treg Immune Modulation Approaches. Diabetes Obes Metab. 2026 Jul;28(7):5476-5491. doi: 10.1111/dom.70800. Epub 2026 Apr 23.
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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.
Manasi Jaiman, MD, MPH study director
ViaCyte
Sandeep Soni, MD study director
CRISPR Therapeutics
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VCTX211 in people with Diabetes Mellitus and Diabetes Mellitus, Type 1, run by CRISPR Therapeutics AG at 2 sites in Canada.
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 2 inclusion and 5 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: stopped early, as of the sponsor's last update on 7 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Patients to be followed up in the VCTX-201 study). Results are not available in the source record.
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?
Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units. (time frame: From implantation up to 12 months post implantation). 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?
On the ClinicalTrials.gov record for NCT05565248, which also lists 1 linked publication.
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 7 May 2026.