A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus (VC01-103)
Primary Purpose
Type 1 Diabetes
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
VC-01 Combination Product
Sponsored by
About this trial
This is an interventional other trial for Type 1 Diabetes
Eligibility Criteria
Inclusion Criteria:
- Men and non-pregnant women
- Diagnosis of T1DM for a minimum of 3 years.
- Stable, optimized diabetic regimen
- Acceptable candidate for implant and explant procedures.
- Willing and able to comply with protocol requirements.
- Meet insulin dosing requirements per protocol
Exclusion Criteria:
• Advanced complications associated with diabetes
Sites / Locations
- AMCR Institute
- Diablo Clinical Research
- Atlanta Diabetes Associates
- Texas Diabetes & Endocrinology
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Sentinel units (aka Cohort 1)
Dose-finding units (aka Cohort 2)
Arm Description
VC-01 Combination Product; Up to ten (10) VC-01 sentinels
VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels
Outcomes
Primary Outcome Measures
Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models
Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models).
Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT
Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04678557
Brief Title
A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus
Acronym
VC01-103
Official Title
An Open-Label Phase 1/2 Study to Evaluate the Safety, Engraftment, and Efficacy of VC-01™Combination Product in Subjects With Type 1 Diabetes Mellitus [T1DM]
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Terminated
Why Stopped
Insufficient functional product engraftment
Study Start Date
June 25, 2019 (Actual)
Primary Completion Date
November 12, 2021 (Actual)
Study Completion Date
November 19, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ViaCyte
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.
Detailed Description
This trial will test if VC-01 combination product can be implanted and maintained with safety, tolerability, and efficacy for up to Month 12/Week 52.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
There are two Cohorts in this study design. Cohort 1 (Phase 1) enrolls up to 30 subjects total. After Cohort 1 enrollment is completed, Cohort 2 enrollment then occurs with up to an additional 40 subjects. A total of up to 70 subjects will be enrolled under this Phase 1/2 protocol.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
31 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Sentinel units (aka Cohort 1)
Arm Type
Experimental
Arm Description
VC-01 Combination Product; Up to ten (10) VC-01 sentinels
Arm Title
Dose-finding units (aka Cohort 2)
Arm Type
Experimental
Arm Description
VC-01 Combination Product; Up to twelve units implanted of which up to nine (9) are VC-01-DF (dose-finding) implants and the rest are VC-01 sentinels
Intervention Type
Combination Product
Intervention Name(s)
VC-01 Combination Product
Other Intervention Name(s)
PEC-Encap
Intervention Description
PEC-01 cells loaded into an Encaptra Drug Delivery System
Primary Outcome Measure Information:
Title
Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models
Description
Through histology, the potential for functional engraftment of VC-01 combination product could be assessed in Cohort 1 subjects. Explanted sentinel units from subjects were processed and stained for markers identifying the number of graft cell nuclei (i.e., signifying the cells were viable). The percentage of viable graft cells in these explanted units were then compared to the percentage of viable graft cells from the pre-clinical (i.e., animal) models. These pre-clinical models are based on animal studies performed at ViaCyte (ref: internal study report). The data in Outcome Measure Data Table represent the % of viable graft cells present in the explanted sentinels compared to what was expected based on the animal model at Weeks 4, 8, 12, and 26 (e.g., At Week 4, there was an average of 18.27% viable graft cells in the participant's explants compared to what was expected based on the animal models).
Time Frame
Weeks 4, 8, 12 and 26
Title
Cohort 2: The Change in AUC (Area Under Curve) From Baseline to Week 26 in C-peptide During 4-hour MMTT
Description
Evaluation of clinical efficacy of VC-01 combination product in Cohort 2 subjects was intended by measuring C-peptide levels during a 4-hour Mixed Meal Tolerance Test (MMTT). Blood glucose and C-peptide data were collected from subjects at timepoints 0, 30, 60, 90, 120, 180, 240 minutes after ingestion of a "meal" (i.e., BOOST drink). These C-peptide data points could be used to create the AUC calculation. If the implanted units contained mature, insulin-producing cells, stimulated C-peptide levels would be expected to increase over the time course in reaction to the meal.
Time Frame
To Week 26
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Men and non-pregnant women
Diagnosis of T1DM for a minimum of 3 years.
Stable, optimized diabetic regimen
Acceptable candidate for implant and explant procedures.
Willing and able to comply with protocol requirements.
Meet insulin dosing requirements per protocol
Exclusion Criteria:
• Advanced complications associated with diabetes
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Manasi Jaiman, MD
Organizational Affiliation
ViaCyte, Inc.
Official's Role
Principal Investigator
Facility Information:
Facility Name
AMCR Institute
City
Escondido
State/Province
California
ZIP/Postal Code
92025
Country
United States
Facility Name
Diablo Clinical Research
City
Walnut Creek
State/Province
California
ZIP/Postal Code
94598
Country
United States
Facility Name
Atlanta Diabetes Associates
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Texas Diabetes & Endocrinology
City
Austin
State/Province
Texas
ZIP/Postal Code
78731
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus
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