A Study to Evaluate Safety, Engraftment, and Efficacy of VC-01 in Subjects With T1 Diabetes Mellitus (VC01-103)

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]

VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.

Study Overview

• Status: Terminated
• Conditions: Type 1 Diabetes
• Intervention / Treatment: Combination product: VC-01 Combination Product

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.

• Study Type: Interventional
• Enrollment (Actual): 31
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Escondido, California, United States, 92025
      • AMCR Institute
    • Walnut Creek, California, United States, 94598
      • Diablo Clinical Research
  • Georgia
    • Atlanta, Georgia, United States, 30318
      • Atlanta Diabetes Associates
  • Texas
    • Austin, Texas, United States, 78731
      • Texas Diabetes & Endocrinology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Sentinel units (aka Cohort 1); VC-01 Combination Product; Up to ten (10) VC-01 sentinels	Combination product: VC-01 Combination Product; PEC-01 cells loaded into an Encaptra Drug Delivery System; Other Names: PEC-Encap
Experimental: 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	Combination product: VC-01 Combination Product; PEC-01 cells loaded into an Encaptra Drug Delivery System; Other Names: PEC-Encap

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: ViaCyte
• Investigators: Principal Investigator: Manasi Jaiman, MD, ViaCyte, Inc.

Publications and helpful links

Helpful Links

• PEC-Encap VC-01

Study record dates

Study Major Dates

• Study Start (Actual): June 25, 2019
• Primary Completion (Actual): November 12, 2021
• Study Completion (Actual): November 19, 2021

Study Registration Dates

• First Submitted: November 30, 2020
• First Submitted That Met QC Criteria: December 16, 2020
• First Posted (Actual): December 22, 2020

Study Record Updates

• Last Update Posted (Actual): October 18, 2023
• Last Update Submitted That Met QC Criteria: September 28, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: VC01-103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No