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Insufficient functional product engraftment
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VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sentinel units (aka Cohort 1) | Experimental | VC-01 Combination Product; Up to ten (10) VC-01 sentinels |
|
| Dose-finding units (aka Cohort 2) | Experimental | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VC-01 Combination Product | Combination Product | PEC-01 cells loaded into an Encaptra Drug Delivery System |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Weeks 4, 8, 12 and 26 |
| 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. | To Week 26 |
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Inclusion Criteria:
Exclusion Criteria:
• Advanced complications associated with diabetes
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| Name | Affiliation | Role |
|---|---|---|
| Manasi Jaiman, MD | ViaCyte, Inc. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| AMCR Institute | Escondido | California | 92025 | United States | ||
| Diablo Clinical Research |
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| ID | Title | Description |
|---|---|---|
| FG000 | 8 or 10 Sentinel Units Implanted (Cohort 1) | VC-01 combination product is comprised of PEC-01 cells loaded into an Encaptra Drug Delivery System. In Cohort 1 of this study, 8 or 10 VC-01 sentinel units were surgically implanted in each subject during a single procedure. Sentinel units are smaller versions of the product not intended to provide efficacy but are instead explanted at various timepoints over the course of the study and examined histologically. |
| FG001 | 9 Dose Finding Units Implanted (Cohort 2) | A total of 12 VC-01 units were implanted in each of the Cohort 2 subjects in a single surgical procedure. Of those units, 9 were VC-01 Dose Finding (DF) units. DF units are larger than sentinels and intended to remain implanted during the duration of participation in the trial to assess efficacy. The remaining 3 units implanted were the smaller VC-01 sentinel units intended for explant at various time points for analysis histologically. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Sentinel Units (Aka Cohort 1) | VC-01 Combination Product; Up to ten (10) VC-01 sentinels VC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System |
| BG001 | Dose-finding Units (Aka Cohort 2) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | For each timepoint, explanted sentinel units containing PEC-01 cells from the 17 subjects were stained and evaluated for graft cell survival (i.e., viability). Not all subjects had units explanted at every timepoint, and units explanted that had "de minimus" graft cell survival (i.e., minimal, < 500 cells, < 4% of pre-clinical controls) could not be evaluated and were not included in the summary table. | Posted | Mean | Standard Deviation | %cells in sentinels rel to animal models | Weeks 4, 8, 12 and 26 |
Cohort 1 subjects reported adverse events over approximately 7 months (e.g., subjects implanted/treated for 6 months). Cohort 2 subjects reported adverse events over approximately 13 months (e.g., subjects implanted/treated for 12 months).
AE/SAE Definitions used are the same as the ClinicalTrials.gov definitions.
Systematic Assessment of AE Collection was used (e.g., regular investigator assessment was required at each study visit per protocol). In addition, routine monitoring visits to the site provided 100% source document verification of the AE Collection.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Sentinel Units (Aka Cohort 1) | VC-01 Combination Product; Up to ten (10) VC-01 sentinels VC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vanishing Twin Syndrome | Pregnancy, puerperium and perinatal conditions | MedDRA 22.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
Cohort 2 (Limitation): Stimulated C-peptide data from the MMTT at Baseline and Week 26 showed undetectable C-peptide (<0.1 ng/mL) for all but one subject. For those subjects, AUC were not generated and assumed "zero" as serial undetectable C-peptide results cannot create a curve.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Vertex | 617-341-6777 | medicalinfo@vrtx.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 29, 2020 | Mar 28, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 15, 2021 | Mar 28, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D003922 | Diabetes Mellitus, Type 1 |
| ID | Term |
|---|---|
| D003920 | Diabetes Mellitus |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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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.
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|
| Walnut Creek |
| California |
| 94598 |
| United States |
| Atlanta Diabetes Associates | Atlanta | Georgia | 30318 | United States |
| Texas Diabetes & Endocrinology | Austin | Texas | 78731 | United States |
| Safety Evaluation |
|
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
VC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Body Mass Index | Mean | Standard Deviation | kg/m^2 |
|
| Duration of Type 1 Diabetes | Mean | Standard Deviation | years |
|
| VC01 sentinels (contains PEC-01 cells) |
| VC01 sentinels (contains PEC-01 cells) |
|
|
|
| Primary | 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. | Stimulated C-peptide data from the MMTT showed every value from baseline and Week 26 samples at all timepoints to be "undetectable" (<0.1 ng/mL) except for one subject for whom the AUC could be calculated. Analyzing the data was deemed futile because the number of surviving graft cells was noted as insufficient for establishing the biological critical mass. The change from baseline to Week 26 in AUC for this one subject is provided below. | Posted | Number | ng*h/mL | To Week 26 |
|
|
|
| 0 |
| 17 |
| 1 |
| 17 |
| 17 |
| 17 |
| EG001 | Dose-finding Units (Aka Cohort 2) | 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 VC-01 Combination Product: PEC-01 cells loaded into an Encaptra Drug Delivery System | 0 | 14 | 3 | 14 | 14 | 14 |
| Small Intestinal Obstruction | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
|
| Hypoglycemia | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
|
| Subarachnoid hemorrhage | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
|
| Complication of Device Removal | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Implant Site Erythema | General disorders | MedDRA 22.0 | Systematic Assessment |
|
| Implant Site Hypoaesthesia | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Infusion Site Haemorrhage | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Postoperative wound infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Incision site complication | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site erythema | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site haematoma | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
|
| Incision site hypoaesthesia | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site pain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site pruritis | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site rash | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Incision site swelling | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Muscle strain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural discomfort | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural haematoma | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural oedema | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Post procedural swelling | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Procedural nausea | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Seroma | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Migraine | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Tension headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
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| D004700 | Endocrine System Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |