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

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.

Study Overview

• Status: Completed
• Conditions: Type 1 Diabetes Mellitus With Hypoglycemia
• Intervention / Treatment: Combination product: VC-02 Combination Product

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 Type: Interventional
• Enrollment (Actual): 49
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Brussel, Belgium
    • University Hospital Brussels
• Canada
  • Alberta
    • Edmonton, Alberta, Canada
      • University of Alberta
  • British Columbia
    • Vancouver, British Columbia, Canada
      • University of British Columbia
• United States
  • California
    • Duarte, California, United States, 91010
      • City of Hope National Medical Center
    • Irvine, California, United States, 92697
      • UCLA-UCI Alpha Stem Cell Clinic
    • Sacramento, California, United States, 95817
      • UC Davis - Alpha Stem Cell Clinic
    • San Diego, California, United States, 92121
      • University of California San Diego
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins University
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Ohio
    • Columbus, Ohio, United States, 43203
      • Ohio State University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 61 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Combination product: VC-02 Combination Product; PEC-01 cells loaded into a Delivery Device; Other Names: PEC-Direct
Experimental: Cohort 1; 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; PEC-01 cells loaded into a Delivery Device; Other Names: PEC-Direct

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: ViaCyte
• Collaborators: Vertex Pharmaceuticals Incorporated; California Institute for Regenerative Medicine (CIRM); Horizon 2020 - European Commission
• Investigators: Study Director: Gautham Marigowda, Vice President, Clinical Development, Vertex

Publications and helpful links

General Publications

• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (Actual): July 6, 2017
• Primary Completion (Actual): October 13, 2023
• Study Completion (Actual): October 19, 2023

Study Registration Dates

• First Submitted: May 19, 2017
• First Submitted That Met QC Criteria: May 19, 2017
• First Posted (Actual): May 23, 2017

Study Record Updates

• Last Update Posted (Actual): December 3, 2024
• Last Update Submitted That Met QC Criteria: November 7, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Metabolic Diseases; Autoimmune Diseases; Immune System Diseases; Glucose Metabolism Disorders; Hypoglycemia; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: VC02-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No