Clinical trial NCT03163511: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Type 1 Diabetes Mellitus With Hypoglycemia
Lead sponsor: ViaCyte
Sponsor class: INDUSTRY
Collaborators: name: California Institute for Regenerative Medicine (CIRM)
name: Horizon 2020 - European Commission
name: Vertex Pharmaceuticals Incorporated
First posted: 2017-05-23
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: 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 purpose: OTHER

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.

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.

Primary outcomes:
measure: Incidence of All AEs in Cohort 1 Participants
timeFrame: Enrollment (Visit 3, Day 1) through the Month 4 Visit
measure: Change in C-peptide for Cohort 2 Subjects
timeFrame: Baseline to Week 26 Visit
Secondary outcomes:
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Irvine
country: United States
facility: UCLA-UCI Alpha Stem Cell Clinic
state: California
city: Sacramento
country: United States
facility: UC Davis - Alpha Stem Cell Clinic
state: California
city: San Diego
country: United States
facility: University of California San Diego
state: California
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Brussel
country: Belgium
facility: University Hospital Brussels
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: University of British Columbia
state: British Columbia
Study officials:
affiliation: Vice President, Clinical Development, Vertex
name: Gautham Marigowda
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.