A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness

NCT ID: NCT03163511

Last Updated: 2024-12-03

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

49 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-06

Study Completion Date

2023-10-19

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Type 1 Diabetes Mellitus With Hypoglycemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

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.
Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Cohort 1

VC-02 Combination Product:

Up to ten (10) VC-02-20 implants and up to two (2) VC-02-300 implants

Group Type EXPERIMENTAL

VC-02 Combination Product

Intervention Type COMBINATION_PRODUCT

PEC-01 cells loaded into a Delivery Device

Cohort 2

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.

Group Type EXPERIMENTAL

VC-02 Combination Product

Intervention Type COMBINATION_PRODUCT

PEC-01 cells loaded into a Delivery Device

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VC-02 Combination Product

PEC-01 cells loaded into a Delivery Device

Intervention Type COMBINATION_PRODUCT

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

PEC-Direct

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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

Exclusion Criteria

* 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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role collaborator

Horizon 2020 - European Commission

OTHER

Sponsor Role collaborator

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role collaborator

ViaCyte

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Gautham Marigowda

Role: STUDY_DIRECTOR

Vice President, Clinical Development, Vertex

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

City of Hope National Medical Center

Duarte, California, United States

Site Status

UCLA-UCI Alpha Stem Cell Clinic

Irvine, California, United States

Site Status

UC Davis - Alpha Stem Cell Clinic

Sacramento, California, United States

Site Status

University of California San Diego

San Diego, California, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

University Hospital Brussels

Brussels, , Belgium

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

University of British Columbia

Vancouver, British Columbia, Canada

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Belgium Canada

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 38012450 (View on PubMed)

Shapiro 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.

Reference Type DERIVED
PMID: 35028608 (View on PubMed)

Ramzy 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.

Reference Type DERIVED
PMID: 34861146 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

VC02-101

Identifier Type: -

Identifier Source: org_study_id