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

NCT03163511 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: VC02-101
• Study Type: interventional
• Target: 49
• Locations: 3 countries
• Sites: 10

Brief 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.

Conditions

Type 1 Diabetes Mellitus With Hypoglycemia

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline to Week 26 Visit

Study Arms (2)

Cohort 1

EXPERIMENTAL

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

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.

Combination Product: VC-02 Combination Product

Interventions

VC-02 Combination Product COMBINATION_PRODUCT

PEC-01 cells loaded into a Delivery Device

Also known as: PEC-Direct

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• ViaCyte: lead
• California Institute for Regenerative Medicine (CIRM): collaborator
• Horizon 2020 - European Commission: collaborator
• Vertex Pharmaceuticals Incorporated: collaborator

Study Sites (10)

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

UCLA-UCI Alpha Stem Cell Clinic

Irvine, California, 92697, United States

Location

UC Davis - Alpha Stem Cell Clinic

Sacramento, California, 95817, United States

Location

University of California San Diego

San Diego, California, 92121, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Ohio State University

Columbus, Ohio, 43203, United States

Location

University Hospital Brussels

Brussels, Belgium

Location

University of Alberta

Edmonton, Alberta, Canada

Location

University of British Columbia

Vancouver, British Columbia, Canada

Location

Related Publications (3)

• 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.

  PMID: 38012450 DERIVED

• 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.

  PMID: 35028608 DERIVED

• 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.

  PMID: 34861146 DERIVED

Results Point of Contact

• Title: Gautham Marigowda
• Organization: Vertex Pharmaceuticals, Inc

gautham_marigowda@vrtx.com

617-961-0568

Study Officials

• Gautham Marigowda

  Vice President, Clinical Development, Vertex

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 19, 2017
• First Posted: May 23, 2017
• Study Start: July 6, 2017
• Primary Completion: October 13, 2023
• Study Completion: October 19, 2023
• Last Updated: December 3, 2024
• Results First Posted: December 3, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2); BE (1); US (7)