Type 1 Diabetes Mellitus With HypoglycemiaPhase 1/2NCT03163511
A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness
A Phase 1/2 study testing VC-02 Combination Product in people with Type 1 Diabetes Mellitus With Hypoglycemia, run by ViaCyte at 10 sites in 3 countries.
Trial summary
VC02-101 will evaluate an experimental cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes and Hypoglycemia Unawareness. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Type 1 Diabetes Mellitus With Hypoglycemia
- What is being tested?
- VC-02 Combination Product
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 10 sitesin 3 countries
- Study dates
- 6 Jul 2017to 19 Oct 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Men and non-pregnant women
- Diagnosis of T1DM for a minimum of five (5) years
- Hypoglycemia unawareness or significant glycemic lability
- Stable diabetic treatment
- Willingness to use a continuous glucose meter
- Acceptable candidate for implantation
Who cannot join
- History of islet cell, kidney, and/or pancreas transplant.
- Six (6) or more severe, unexplained hypoglycemic events within six (6) months of enrollment
- Uncontrolled or untreated thyroid disease or adrenal insufficiency
- Diabetic complications such as severe kidney disease or renal dysfunction, proliferative retinopathy, diabetic foot ulcers, amputations attributable to diabetes, and/or severe peripheral neuropathy
- Non-compliance with current anti-diabetic regimen
- Detectable stimulated serum C-peptide during screening period assessment.
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
United States7 sites
Johns Hopkins University
Baltimore, Maryland, 21205
Ohio State University
Columbus, Ohio, 43203
City of Hope National Medical Center
Duarte, California, 91010
UCLA-UCI Alpha Stem Cell Clinic
Irvine, California, 92697
University of Minnesota
Minneapolis, Minnesota, 55455
UC Davis - Alpha Stem Cell Clinic
Sacramento, California, 95817
University of California San Diego
San Diego, California, 92121
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 VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness
VC02-101 will evaluate an experimental cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes and Hypoglycemia Unawareness.
Read the detailed description
The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and Hypoglycemia Unawareness and maintained safely for up to two years. It will also test if VC-02 is an effective treatment for these subjects.
- Being tested
- VC-02 Combination Product
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VC-02 Combination ProductCombination product
Also called PEC-Direct
PEC-01 cells loaded into a Delivery Device
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | VC-02 Combination Product:
Up to ten (10) VC-02-20 implants and up to two (2) VC-02-300 implants Receives: Combination Product: VC-02 Combination Product |
| Cohort 2 | Experimental | VC-02 Combination Product; Up to twelve units implanted of which up to ten (10) are VC-02-300 implants and the rest are VC-02-20 implants. Receives: Combination Product: VC-02 Combination Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of All AEs in Cohort 1 Participants
Incidence of all AEs and evaluation of causality related to VC-02 combination product, the surgical procedures, and the immunosuppressive drug regimen.
Measured: Enrollment (Visit 3, Day 1) through the Month 4 Visit
Change in C-peptide for Cohort 2 Subjects
Change from baseline to Week 26 in C-peptide area under the curve from 0 to 4 hours following a mixed meal tolerance test.
Measured: Baseline to Week 26 Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2017
- Study start6 Jul 2017
- Primary completion13 Oct 2023
- Study completion19 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Type 1 Diabetes Mellitus With HypoglycemiaStudies 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 1/2
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- There are two Cohorts in this study design. Cohort 1 was to enroll up to 15 subjects total. After Cohort 1 enrollment was completed, Cohort 2 was to enroll up to an additional 60 subjects.
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- ViaCyte
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM), Horizon 2020 - European Commission, Vertex Pharmaceuticals Incorporated
- Responsible party
- Sponsor
- Investigators
- Gautham Marigowda, Study director, Vice President, Clinical Development, Vertex
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03163511
- Sponsor's protocol ID
- VC02-101
- Status last verified
- Nov 2024
- First submitted
- 19 May 2017
- Last updated
- 3 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Keymeulen B, De Groot K, Jacobs-Tulleneers-Thevissen D, Thompson DM, Bellin MD, Kroon EJ, Daniels M, Wang R, Jaiman M, Kieffer TJ, Foyt HL, Pipeleers D. Encapsulated stem cell-derived beta cells exert glucose control in patients with type 1 diabetes. Nat Biotechnol. 2024 Oct;42(10):1507-1514. doi: 10.1038/s41587-023-02055-5. Epub 2023 Nov 27.
PubMed 38012450Shapiro AMJ, Thompson D, Donner TW, Bellin MD, Hsueh W, Pettus J, Wilensky J, Daniels M, Wang RM, Brandon EP, Jaiman MS, Kroon EJ, D'Amour KA, Foyt HL. Insulin expression and C-peptide in type 1 diabetes subjects implanted with stem cell-derived pancreatic endoderm cells in an encapsulation device. Cell Rep Med. 2021 Dec 2;2(12):100466. doi: 10.1016/j.xcrm.2021.100466. eCollection 2021 Dec 21.
PubMed 35028608Ramzy A, Thompson DM, Ward-Hartstonge KA, Ivison S, Cook L, Garcia RV, Loyal J, Kim PTW, Warnock GL, Levings MK, Kieffer TJ. Implanted pluripotent stem-cell-derived pancreatic endoderm cells secrete glucose-responsive C-peptide in patients with type 1 diabetes. Cell Stem Cell. 2021 Dec 2;28(12):2047-2061.e5. doi: 10.1016/j.stem.2021.10.003.
PubMed 34861146
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2024. 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 VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness
- ClinicalTrials.gov ID
- NCT03163511
- Acronym / sponsor ID
- VC02-101
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- ViaCyteIndustry (pharma, biotech, devices)
- Collaborators
- California Institute for Regenerative Medicine (CIRM), Horizon 2020 - European Commission, Vertex Pharmaceuticals Incorporated
- Study start
- 6 Jul 2017 (actual)
- Primary completion
- 13 Oct 2023 (actual)
- Study completion
- 19 Oct 2023 (actual)
- First posted
- 23 May 2017 (actual)
- Results
- Posted 3 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
VC02-101 will evaluate an experimental cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes and Hypoglycemia Unawareness.
Detailed description
The purpose of this trial is to test if VC-02™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and Hypoglycemia Unawareness and maintained safely for up to two years. It will also test if VC-02 is an effective treatment for these subjects.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- There are two Cohorts in this study design. Cohort 1 was to enroll up to 15 subjects total. After Cohort 1 enrollment was completed, Cohort 2 was to enroll up to an additional 60 subjects.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Combination Product: VC-02 Combination ProductVC-02 Combination Product: Up to ten (10) VC-02-20 implants and up to two (2) VC-02-300 implants |
| Cohort 2 | Experimental | Combination Product: VC-02 Combination ProductVC-02 Combination Product; Up to twelve units implanted of which up to ten (10) are VC-02-300 implants and the rest are VC-02-20 implants. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Men and non-pregnant women
- Diagnosis of T1DM for a minimum of five (5) years
- Hypoglycemia unawareness or significant glycemic lability
- Stable diabetic treatment
- Willingness to use a continuous glucose meter
Key exclusion criteria (6 listed)
- History of islet cell, kidney, and/or pancreas transplant.
- Six (6) or more severe, unexplained hypoglycemic events within six (6) months of enrollment
- Uncontrolled or untreated thyroid disease or adrenal insufficiency
- Diabetic complications such as severe kidney disease or renal dysfunction, proliferative retinopathy, diabetic foot ulcers, amputations attributable to diabetes, and/or severe peripheral neuropathy
- Non-compliance with current anti-diabetic regimen
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 |
|---|---|---|
| VC-02 Combination ProductAlso: PEC-DirectPEC-01 cells loaded into a Delivery Device | Combination product | Cohort 1, Cohort 2 |
Primary outcome
Incidence of All AEs in Cohort 1 Participants
Time frame: Enrollment (Visit 3, Day 1) through the Month 4 Visit
Incidence of all AEs and evaluation of causality related to VC-02 combination product, the surgical procedures, and the immunosuppressive drug regimen.
Change in C-peptide for Cohort 2 Subjects
Time frame: Baseline to Week 26 Visit
Change from baseline to Week 26 in C-peptide area under the curve from 0 to 4 hours following a mixed meal tolerance test.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38012450Keymeulen B, De Groot K, Jacobs-Tulleneers-Thevissen D, Thompson DM, Bellin MD, Kroon EJ, Daniels M, Wang R, Jaiman M, Kieffer TJ, Foyt HL, Pipeleers D. Encapsulated stem cell-derived beta cells exert glucose control in patients with type 1 diabetes. Nat Biotechnol. 2024 Oct;42(10):1507-1514. doi: 10.1038/s41587-023-02055-5. Epub 2023 Nov 27.
- Derived from this study: PubMed 35028608Shapiro AMJ, Thompson D, Donner TW, Bellin MD, Hsueh W, Pettus J, Wilensky J, Daniels M, Wang RM, Brandon EP, Jaiman MS, Kroon EJ, D'Amour KA, Foyt HL. Insulin expression and C-peptide in type 1 diabetes subjects implanted with stem cell-derived pancreatic endoderm cells in an encapsulation device. Cell Rep Med. 2021 Dec 2;2(12):100466. doi: 10.1016/j.xcrm.2021.100466. eCollection 2021 Dec 21.
- Derived from this study: PubMed 34861146Ramzy A, Thompson DM, Ward-Hartstonge KA, Ivison S, Cook L, Garcia RV, Loyal J, Kim PTW, Warnock GL, Levings MK, Kieffer TJ. Implanted pluripotent stem-cell-derived pancreatic endoderm cells secrete glucose-responsive C-peptide in patients with type 1 diabetes. Cell Stem Cell. 2021 Dec 2;28(12):2047-2061.e5. doi: 10.1016/j.stem.2021.10.003.
- Individual participant dataSharing planned: No
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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.
Gautham Marigowda study director
Vice President, Clinical Development, Vertex
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VC-02 Combination Product in people with Type 1 Diabetes Mellitus With Hypoglycemia, run by ViaCyte at 10 sites in 3 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 6 inclusion and 6 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 3 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada, Belgium. 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?
Yes. Results are posted in the registry (first posted 3 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 All AEs in Cohort 1 Participants (time frame: Enrollment (Visit 3, Day 1) through the Month 4 Visit). 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 NCT03163511, which also lists 3 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 3 Dec 2024; AzTrials last refreshed its copy on 6 Nov 2026.