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

• ClinicalTrials.gov Identifier: NCT03163511
• Recruitment Status: Active, not recruiting
• First Posted: May 23, 2017
• Last Update Posted: May 26, 2023
• Sponsor: ViaCyte
• Collaborators: California Institute for Regenerative Medicine (CIRM); Horizon 2020 - European Commission
• Information provided by (Responsible Party): ViaCyte

Tracking Information

• First Submitted Date: May 19, 2017
• First Posted Date: May 23, 2017
• Last Update Posted Date: May 26, 2023
• Actual Study Start Date: July 6, 2017
• Estimated Primary Completion Date: August 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 25, 2020)

• Incidence of all adverse events reported for Cohort 1 subjects [ Time Frame: Thru the Month 4 Visit ]
  AEs through four months.
• Change in C-peptide for Cohort 2 subjects [ Time Frame: Baseline to the Month 6 Visit ]
  Change in C-peptide from baseline.

Original Primary Outcome Measures (submitted: May 19, 2017)

• Incidence of all adverse events reported for Cohort 1 subjects [ Time Frame: Thru the Month 4 Visit ]
• Change in C-peptide for Cohort 2 subjects [ Time Frame: Baseline to the Month 6 Visit ]

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Safety, Tolerability, and Efficacy Study of VC-02™ Combination Product in Subjects With Type 1 Diabetes Mellitus and Hypoglycemia Unawareness
• Official Title: 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
• 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.
• 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
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

There are two Cohorts in this study design. Cohort 1 enrolls up to 15 subjects total. After Cohort 1 enrollment is completed, Cohort 2 enrollment then occurs with up to an additional 60 subjects.

Masking: None (Open Label)
Primary Purpose: Other

• Condition: Type 1 Diabetes Mellitus With Hypoglycemia

Intervention

Combination Product: VC-02 Combination Product

PEC-01 cells loaded into a Delivery Device

Other Name: PEC-Direct

Study Arms

• Experimental: Cohort 1
  VC-02 Combination Product; Up to six (6) VC-02-20 implants and up to two (2) VC-02-300 implants
  Intervention: Combination Product: VC-02 Combination Product
• 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.
  Intervention: Combination Product: VC-02 Combination Product

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.

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: May 24, 2023): 49
• Original Estimated Enrollment (submitted: May 19, 2017): 55
• Estimated Study Completion Date: September 2024
• Estimated Primary Completion Date: August 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Canada, United States

Administrative Information

• NCT Number: NCT03163511
• Other Study ID Numbers: VC02-101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: ViaCyte
• Original Responsible Party: Same as current
• Current Study Sponsor: ViaCyte
• Original Study Sponsor: Same as current

Collaborators

• California Institute for Regenerative Medicine (CIRM)
• Horizon 2020 - European Commission

Investigators

• Principal Investigator: Manasi Jaiman, MD, MPH; Chief Medical Officer, ViaCyte

• PRS Account: ViaCyte
• Verification Date: May 2023