Type 1 DiabetesPhase 1/2VC01-103NCT04678557

A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus

A Phase 1/2 study testing VC-01 Combination Product in people with Type 1 Diabetes, run by ViaCyte at 4 sites in United States.

Terminated

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

Official record

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Trial summary

VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes. 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?: VC-01 Combination Product
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 31 participants
Where: 4 sitesUnited States
Study dates: 25 Jun 2019to 19 Nov 2021
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 3 years.
Stable, optimized diabetic regimen
Acceptable candidate for implant and explant procedures.
Willing and able to comply with protocol requirements.
Meet insulin dosing requirements per protocol

Who cannot join

Advanced complications associated with diabetes

Trial locations and contacts

4 sites in 1 country.

United States4 sites

Atlanta Diabetes Associates
Atlanta, Georgia, 30318
Texas Diabetes & Endocrinology
Austin, Texas, 78731
AMCR Institute
Escondido, California, 92025
Diablo Clinical Research
Walnut Creek, California, 94598

Other trials in United States

About this study

Official title: An Open-Label Phase 1/2 Study to Evaluate the Safety, Engraftment, and Efficacy of VC-01™Combination Product in Subjects With Type 1 Diabetes Mellitus [T1DM]

VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.

Read the detailed description

This trial will test if VC-01 combination product can be implanted and maintained with safety, tolerability, and efficacy for up to Month 12/Week 52.

Conditions: Type 1 Diabetes
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
Also called PEC-Encap
PEC-01 cells loaded into an Encaptra Drug Delivery System

Study groups in NCT04678557
Study group	Role	What they receive
Sentinel units (aka Cohort 1)	Experimental	VC-01 Combination Product; Up to ten (10) VC-01 sentinels Receives: Combination Product: VC-01 Combination Product
Dose-finding units (aka Cohort 2)	Experimental	VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels Receives: Combination Product: VC-01 Combination Product

What is being measured

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

Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models
Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models).
Measured: Weeks 4, 8, 12 and 26
Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT
Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.
Measured: To Week 26

Timeline

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

25 Jun 201919 Nov 2021

First posted22 Dec 2020
Study start25 Jun 2019
Primary completion12 Nov 2021
Study completion19 Nov 2021

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.
Other trials for Type 1 DiabetesStudies 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 (Phase 1) enrolls up to 30 subjects total. After Cohort 1 enrollment is completed, Cohort 2 enrollment then occurs with up to an additional 40 subjects. A total of up to 70 subjects will be enrolled under this Phase 1/2 protocol.
Masking: None (open label)
Enrolment: 31 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04678557
Sponsor's protocol ID: VC01-103
Acronym: VC01-103
Status last verified: Sep 2023
First submitted: 30 Nov 2020
Last updated: 18 Oct 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

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 trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label Phase 1/2 Study to Evaluate the Safety, Engraftment, and Efficacy of VC-01™Combination Product in Subjects With Type 1 Diabetes Mellitus [T1DM]
ClinicalTrials.gov ID: NCT04678557
Acronym / sponsor ID: VC01-103 / VC01-103
Conditions: Type 1 Diabetes
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Insufficient functional product engraftment
Enrolment: 31 participants (actual)
Lead sponsor: ViaCyteIndustry (pharma, biotech, devices)
Study start: 25 Jun 2019 (actual)
Primary completion: 12 Nov 2021 (actual)
Study completion: 19 Nov 2021 (actual)
First posted: 22 Dec 2020 (actual)
Results: Posted 18 Oct 2023

Clinical summary

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

VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.

Detailed description

This trial will test if VC-01 combination product can be implanted and maintained with safety, tolerability, and efficacy for up to Month 12/Week 52.

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 (Phase 1) enrolls up to 30 subjects total. After Cohort 1 enrollment is completed, Cohort 2 enrollment then occurs with up to an additional 40 subjects. A total of up to 70 subjects will be enrolled under this Phase 1/2 protocol.

Arm	Type	Receives
Sentinel units (aka Cohort 1)	Experimental	Combination Product: VC-01 Combination ProductVC-01 Combination Product; Up to ten (10) VC-01 sentinels
Dose-finding units (aka Cohort 2)	Experimental	Combination Product: VC-01 Combination ProductVC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels

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 3 years.
Stable, optimized diabetic regimen
Acceptable candidate for implant and explant procedures.
Willing and able to comply with protocol requirements.

Key exclusion criteria (1 listed)

Advanced complications associated with diabetes

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 ProductAlso: PEC-EncapPEC-01 cells loaded into an Encaptra Drug Delivery System	Combination product	Dose-finding units (aka Cohort 2), Sentinel units (aka Cohort 1)

Primary outcome

Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models
Time frame: Weeks 4, 8, 12 and 26
Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models).
Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT
Time frame: To Week 26
Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.

Results and safety

Results posted to the registry on 18 Oct 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
8 or 10 Sentinel Units Implanted (Cohort 1)	17	16
9 Dose Finding Units Implanted (Cohort 2)	14	0

Primary outcome: Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models

Time frame: Weeks 4, 8, 12 and 26. Mean (Standard Deviation). Unit: %cells in sentinels rel to animal models

Measure	Sentinel Units (Aka Cohort 1) n = 17
Week 4	18.27 (13.94)
Week 8	6.23 (4.63)
Week 12	7.58 (7.27)
Week 26	8.02 (8.09)

Population analysed: For each timepoint, explanted sentinel units containing PEC-01 cells from the 17 subjects were stained and evaluated for graft cell survival (i.e., viability). Not all subjects had units explanted at every timepoint, and units explanted that had "de minimus" graft cell survival (i.e., minimal, \< 500 cells, \< 4% of pre-clinical controls) could not be evaluated and were not included in the summary table.

Primary outcome: Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT

Time frame: To Week 26. Number. Unit: ng*h/mL

Measure	Dose-finding Units (Aka Cohort 2) n = 1
Value	0.325

Population analysed: Stimulated C-peptide data from the MMTT showed every value from baseline and Week 26 samples at all timepoints to be "undetectable" (\<0.1 ng/mL) except for one subject for whom the AUC could be calculated. Analyzing the data was deemed futile because the number of surviving graft cells was noted as insufficient for establishing the biological critical mass. The change from baseline to Week 26 in AUC for this one subject is provided below.

Adverse events

Time frame: Cohort 1 subjects reported adverse events over approximately 7 months (e.g., subjects implanted/treated for 6 months). Cohort 2 subjects reported adverse events over approximately 13 months (e.g., subjects implanted/treated for 12 months).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Sentinel Units (Aka Cohort 1)	1 of 17 (5.9%)	17 of 17 (100%)	0 of 17
Dose-finding Units (Aka Cohort 2)	3 of 14 (21.4%)	14 of 14 (100%)	0 of 14

Limitations and caveats (from the sponsor): Cohort 2 (Limitation): Stimulated C-peptide data from the MMTT at Baseline and Week 26 showed undetectable C-peptide (\<0.1 ng/mL) for all but one subject. For those subjects, AUC were not generated and assumed "zero" as serial undetectable C-peptide results cannot create a curve.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.

Manasi Jaiman, MD principal investigator

ViaCyte, Inc.

4 sites in 1 country: 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, run by ViaCyte at 4 sites in United States.

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 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: stopped early, as of the sponsor's last update on 18 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. 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 18 Oct 2023). 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?

Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models (time frame: Weeks 4, 8, 12 and 26). 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 NCT04678557.

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 18 Oct 2023.