Type 1 Diabetes MellitusPhase 1/2NCT02239354

A Safety, Tolerability, and Efficacy Study of VC-01™ Combination Product in Subjects With Type I Diabetes Mellitus

A Phase 1/2 study testing VC-01™ Combination Product in people with Type 1 Diabetes Mellitus, run by ViaCyte at 2 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The purpose of this trial is to test if VC-01™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for two years. It will also test if VC-01 is an effective treatment for subjects with Type 1 Diabetes. Read more about this study

Who is it for?: Ages 18 to 55 years
Conditions the study is for: Type 1 Diabetes Mellitus
What is being tested?: VC-01™ Combination Product
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 19 participants
Where: 2 sitesin 2 countries
Study dates: Sep 2014to Dec 2017
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 55 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Men and women (non-pregnant and non-childbearing potential)
Diagnosis of type 1 diabetes mellitus for at least 3 years
Stable diabetic treatment
Willingness to use a continuous glucose meter
Acceptable candidate for implantation

Who cannot join

Advanced complications associated with diabetes
Immunosuppressive therapy

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

United States1 site

University of California at San Diego
San Diego, California

Other trials in United States

Canada1 site

University of Alberta Hospital
Edmonton, Alberta, T6G 2B7

Other trials in Canada

About this study

Official title: A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study With Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01™ Combination Product in Subjects With Type 1 Diabetes Mellitus

Conditions: Type 1 Diabetes Mellitus
Being tested: VC-01™ Combination Product
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.

VC-01™ Combination ProductCombination product
Biologic and Device

Study groups in NCT02239354
Study group	Role	What they receive
Cohort 1	Experimental	2 VC-01™ Combination Product implants Receives: Combination Product: VC-01™ Combination Product
Cohort 2	Experimental	4 or 6 VC-01™ Combination Product implants Receives: Combination Product: VC-01™ Combination Product

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Adverse Events Reported During the Study.
Measured: Thru the Month 24 Visit
Change in C-peptide
The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint.
Measured: Baseline to the Month 6 Visit

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2014Dec 2017

First posted12 Sep 2014 (estimated)
Study startSep 2014
Primary completionDec 2017
Study completionDec 2017

Why it stopped: Insufficient functional product engraftment

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 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 1/2
Primary purpose: Other
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 19 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02239354
Sponsor's protocol ID: VC01-101
Status last verified: Feb 2022
First submitted: 9 Sep 2014
Last updated: 24 Mar 2022
Results posted: Yes, on ClinicalTrials.gov

Publications

Satin LS, Soleimanpour SA, Walker EM. New Aspects of Diabetes Research and Therapeutic Development. Pharmacol Rev. 2021 Jul;73(3):1001-1015. doi: 10.1124/pharmrev.120.000160.
PubMed 34193595
Saber N, Bruin JE, O'Dwyer S, Schuster H, Rezania A, Kieffer TJ. Sex Differences in Maturation of Human Embryonic Stem Cell-Derived beta Cells in Mice. Endocrinology. 2018 Apr 1;159(4):1827-1841. doi: 10.1210/en.2018-00048.
PubMed 29420708
Pase C, Mathias AD, Garcia CD, Garcia Rodrigues C. Using Social Media for the Promotion of Education and Consultation in Adolescents Who Have Undergone Kidney Transplant: Protocol for a Randomized Control Trial. JMIR Res Protoc. 2018 Jan 9;7(1):e3. doi: 10.2196/resprot.8065.
PubMed 29317381

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Type 1 Diabetes Mellitus trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study With Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01™ Combination Product in Subjects With Type 1 Diabetes Mellitus
ClinicalTrials.gov ID: NCT02239354
Acronym / sponsor ID: VC01-101
Conditions: Type 1 Diabetes Mellitus
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Insufficient functional product engraftment
Enrolment: 19 participants (actual)
Lead sponsor: ViaCyteIndustry (pharma, biotech, devices)
Collaborators: California Institute for Regenerative Medicine (CIRM)
Study start: Sep 2014 (actual)
Primary completion: Dec 2017 (actual)
Study completion: Dec 2017 (actual)
First posted: 12 Sep 2014 (estimated)
Results: Posted 24 Mar 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Other
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Cohort 1	Experimental	Combination Product: VC-01™ Combination Product2 VC-01™ Combination Product implants
Cohort 2	Experimental	Combination Product: VC-01™ Combination Product4 or 6 VC-01™ Combination Product implants

Who may qualify

Ages: 18 to 55 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (5 listed)

Men and women (non-pregnant and non-childbearing potential)
Diagnosis of type 1 diabetes mellitus for at least 3 years
Stable diabetic treatment
Willingness to use a continuous glucose meter
Acceptable candidate for implantation

Key exclusion criteria (2 listed)

Advanced complications associated with diabetes
Immunosuppressive 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
VC-01™ Combination ProductBiologic and Device	Combination product	Cohort 1, Cohort 2

Primary outcome

Number of Adverse Events Reported During the Study.
Time frame: Thru the Month 24 Visit
Change in C-peptide
Time frame: Baseline to the Month 6 Visit
The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint.

Results and safety

Results posted to the registry on 24 Mar 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1	19	9
Cohort 2	0	0

Primary outcome: Number of Adverse Events Reported During the Study.

Time frame: Thru the Month 24 Visit. Number. Unit: events

Measure	Cohort 1 n = 19
Value	114

Population analysed: The Safety Analysis Set (SAS) will include all T1DM subjects who were enrolled into the study and in whom an implant surgery was attempted, regardless if any VC-01-250 units or sentinel-sized units were actually implanted. This includes all subjects in both Cohort 1 and Cohort 2.

Primary outcome: Change in C-peptide

Time frame: Baseline to the Month 6 Visit

Population analysed: No subjects were enrolled in Cohort 2. Therefore, no data was collected for this endpoint.

Adverse events

Time frame: AEs are recorded on the CRF from Visit 2 (Week-3) through the last subject visit (out to two years). At the conclusion of the trial, any ongoing AEs were followed up for 28 days after the explantation procedure.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1	2 of 19 (10.5%)	18 of 19 (94.7%)	0 of 19
Cohort 2	0 of 0	0 of 0	0

Limitations and caveats (from the sponsor): Study did not proceed to Cohort 2 to evaluate efficacy.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 34193595Satin LS, Soleimanpour SA, Walker EM. New Aspects of Diabetes Research and Therapeutic Development. Pharmacol Rev. 2021 Jul;73(3):1001-1015. doi: 10.1124/pharmrev.120.000160.
Derived from this study: PubMed 29420708Saber N, Bruin JE, O'Dwyer S, Schuster H, Rezania A, Kieffer TJ. Sex Differences in Maturation of Human Embryonic Stem Cell-Derived beta Cells in Mice. Endocrinology. 2018 Apr 1;159(4):1827-1841. doi: 10.1210/en.2018-00048.
Derived from this study: PubMed 29317381Pase C, Mathias AD, Garcia CD, Garcia Rodrigues C. Using Social Media for the Promotion of Education and Consultation in Adolescents Who Have Undergone Kidney Transplant: Protocol for a Randomized Control Trial. JMIR Res Protoc. 2018 Jan 9;7(1):e3. doi: 10.2196/resprot.8065.

Related trials for Type 1 Diabetes Mellitus

Not yet recruitingPhase 3
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Obesity, Overweight
RecruitingPhase 1
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Active, not recruitingPhase 2
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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.

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing VC-01™ Combination Product in people with Type 1 Diabetes Mellitus, run by ViaCyte at 2 sites in 2 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 5 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: stopped early, as of the sponsor's last update on 24 Mar 2022. 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 United States, 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 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 24 Mar 2022). 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?

Number of Adverse Events Reported During the Study. (time frame: Thru the Month 24 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 NCT02239354, 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 24 Mar 2022.