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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Type 1 Diabetes Mellitus With Hypoglycemia

Intervention / Treatment
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* Combination Product: VC-02 Combination Product


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Interventional 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)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1<br>VC-02 Combination Product; Up to six (6) VC-02-20 implants and up to two (2) VC-02-300 implants | Combination Product: VC-02 Combination Product<br>* PEC-01 cells loaded into a Delivery Device<br>* Other names: PEC-Direct;|
| Experimental: Cohort 2<br>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<br>* PEC-01 cells loaded into a Delivery Device<br>* Other names: PEC-Direct;|

What is the study measuring?
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Primary Outcome Measures

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