Diabetes MellitusPhase 1NCT05210530
An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D
A Phase 1 study testing VCTX210A unit in people with Diabetes Mellitus and Diabetes Mellitus, Type 1, run by CRISPR Therapeutics AG at 3 sites in Canada.
Trial summary
This is an open-label, multicenter, Phase 1 study evaluating the safety and tolerability of VCTX210A combination product in patients 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?
- VCTX210A unit
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 3 sitesCanada
- Study dates
- 24 Jan 2022to 19 Jan 2023
- 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, optimized diabetic 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
3 sites in 1 country.
Canada3 sites
University of Alberta
Edmonton, Alberta
LMC Manna
Toronto, Ontario
University of British Columbia
Vancouver, British Columbia
About this study
Official title: An Open-Label, First-In-Human Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
This is an open-label, multicenter, Phase 1 study evaluating the safety and tolerability of VCTX210A combination product in patients with T1D
Read the detailed description
VCTX210A combination product (unit) comprises 2 components: (1) allogeneic pancreatic endoderm cells (PEC210A) genetically modified using Clustered 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 the PEC210A cells.
- Conditions
- Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders, Metabolic Disease, Endocrine System Diseases, Autoimmune Diseases
- Being tested
- VCTX210A unit
- 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.
VCTX210A unitCombination product
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device
| Study group | Role | What they receive |
|---|---|---|
| VCTX210A combination product | Experimental | Up to seven (7) units will be implanted Receives: Combination Product: VCTX210A unit |
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 VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.
Measured: From implantation up to 6 months post implantation
Secondary outcomes (6)
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.
Measured: From implantation up to 6 months post implantation
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.
Measured: From implantation up to 6 months post implantation
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Measured: From implantation up to 6 months post implantation
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Measured: From implantation up to 6 months post implantation
The percentage of viable graft cells per unit using immunohistochemical staining.
Measured: From implantation up to 6 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 6 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 posted27 Jan 2022
- Study start24 Jan 2022
- Primary completion19 Jan 2023
- Study completion19 Jan 2023
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 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
- 7 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
- NCT05210530
- Sponsor's protocol ID
- VCTX210A-101
- Status last verified
- Jun 2023
- First submitted
- 7 Dec 2021
- Last updated
- 26 Jun 2023
- 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 42023429Ellis CE, Mojibian M, Ida S, Fung VCW, Skovso S, McIver E, O'Dwyer S, Webber TD, Braam MJS, Saber N, Sasaki S, Lynn FC, Kieffer TJ, Levings MK. Human A2-CAR T Cells Reject HLA-A2 + Human Islets Transplanted Into Mice Without Inducing Graft-versus-host Disease. Transplantation. 2023 Sep 1;107(9):e222-e233. doi: 10.1097/TP.0000000000004709. Epub 2023 Aug 21.
PubMed 37528526
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2023. 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 and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
- ClinicalTrials.gov ID
- NCT05210530
- Acronym / sponsor ID
- VCTX210A-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
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- CRISPR Therapeutics AGIndustry (pharma, biotech, devices)
- Collaborators
- ViaCyte
- Study start
- 24 Jan 2022 (actual)
- Primary completion
- 19 Jan 2023 (actual)
- Study completion
- 19 Jan 2023 (actual)
- First posted
- 27 Jan 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 and tolerability of VCTX210A combination product in patients with T1D
Detailed description
VCTX210A combination product (unit) comprises 2 components: (1) allogeneic pancreatic endoderm cells (PEC210A) genetically modified using Clustered 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 the PEC210A cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VCTX210A combination product | Experimental | Combination Product: VCTX210A unitUp to seven (7) units will be implanted |
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, optimized diabetic 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 |
|---|---|---|
| VCTX210A unitCRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device | Combination product | VCTX210A combination product |
Primary outcome
Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.
Time frame: From implantation up to 6 months post implantation
6 secondary outcomes
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.
Time frame: From implantation up to 6 months post implantation
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.
Time frame: From implantation up to 6 months post implantation
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Time frame: From implantation up to 6 months post implantation
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Time frame: From implantation up to 6 months post implantation
The percentage of viable graft cells per unit using immunohistochemical staining.
Time frame: From implantation up to 6 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 6 months post implantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2023.
- 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.
- Derived from this study: PubMed 37528526Ellis CE, Mojibian M, Ida S, Fung VCW, Skovso S, McIver E, O'Dwyer S, Webber TD, Braam MJS, Saber N, Sasaki S, Lynn FC, Kieffer TJ, Levings MK. Human A2-CAR T Cells Reject HLA-A2 + Human Islets Transplanted Into Mice Without Inducing Graft-versus-host Disease. Transplantation. 2023 Sep 1;107(9):e222-e233. doi: 10.1097/TP.0000000000004709. Epub 2023 Aug 21.
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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
3 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 VCTX210A unit in people with Diabetes Mellitus and Diabetes Mellitus, Type 1, run by CRISPR Therapeutics AG at 3 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: finished, as of the sponsor's last update on 26 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units. (time frame: From implantation up to 6 months post implantation). 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 NCT05210530, 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 26 Jun 2023.