Type 1 Diabetes Mellitus
Conditions
Brief summary
The purpose of this trial is to test if VC-01™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for two years. It will also test if VC-01 is an effective treatment for subjects with Type 1 Diabetes.
Interventions
COMBINATION_PRODUCTVC-01™ Combination Product
Biologic and Device
Sponsors
California Institute for Regenerative Medicine (CIRM)
ViaCyte
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* Men and women (non-pregnant and non-childbearing potential) * Diagnosis of type 1 diabetes mellitus for at least 3 years * Stable diabetic treatment * Willingness to use a continuous glucose meter * Acceptable candidate for implantation
Exclusion criteria
* Advanced complications associated with diabetes * Immunosuppressive therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Adverse Events Reported During the Study. | Thru the Month 24 Visit | — |
| Change in C-peptide | Baseline to the Month 6 Visit | The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint. |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1 Up to 2 VC-01-250™ Combination Product implants
Up to 6 VC-01-20 sentinel units
VC-01™ Combination Product: Biologic and Device | 19 |
| Cohort 2 Up to 4 or 6 VC-01™ Combination Product implants
Up to 3 VC-01-20 sentinel units
VC-01™ Combination Product: Biologic and Device | 0 |
| Total | 19 |
Baseline characteristics
| Characteristic | Total | Cohort 1 |
|---|---|---|
| Age, Continuous | 29.0 years STANDARD_DEVIATION 11.92 | 29.0 years STANDARD_DEVIATION 11.92 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 18 Participants | 18 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 19 Participants | 19 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 18 Participants | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 19 | 0 / 0 |
| other Total, other adverse events | 18 / 19 | 0 / 0 |
| serious Total, serious adverse events | 2 / 19 | 0 / 0 |
Outcome results
Primary
Change in C-peptide
The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint.
Time frame: Baseline to the Month 6 Visit
Population: No subjects were enrolled in Cohort 2. Therefore, no data was collected for this endpoint.
Primary
Number of Adverse Events Reported During the Study.
Time frame: Thru the Month 24 Visit
Population: The Safety Analysis Set (SAS) will include all T1DM subjects who were enrolled into the study and in whom an implant surgery was attempted, regardless if any VC-01-250 units or sentinel-sized units were actually implanted. This includes all subjects in both Cohort 1 and Cohort 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Cohort 1 | Number of Adverse Events Reported During the Study. | 114 events |