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

Phase 1

Terminated

• Conditions: Type 1 Diabetes
• Interventions: Combination Product: VC-01 Combination Product

• Registration Number: NCT04678557
• Lead Sponsor: ViaCyte

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.

Study Timeline

• Study Start: 2019-06-25
• Study First Submitted: 2020-11-30
• Study First Submitted QC: 2020-12-16
• Study First Posted: 2020-12-22
• Primary Completion: 2021-11-12
• Study Completion: 2021-11-19
• Results First Submitted: 2023-04-03
• Status Verified: 2023-09
• Results First Submitted QC: 2023-09-28
• Last Update Submitted: 2023-09-28
• Results First Posted: 2023-10-18
• Last Update Posted: 2023-10-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 31

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Sentinel units (aka Cohort 1)	VC-01 Combination Product	VC-01 Combination Product; Up to ten (10) VC-01 sentinels
Dose-finding units (aka Cohort 2)	VC-01 Combination Product	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

Primary Outcome Measures

Name	Time	Method
Cohort 1: The Percentage of Graft Cells Present at Post-implant Time Points Relative to Pre-clinical Models	Weeks 4, 8, 12 and 26	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	To Week 26	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.

Trial Locations

Locations (4)

AMCR Institute; 🇺🇸 Escondido, California, United States

Diablo Clinical Research; 🇺🇸 Walnut Creek, California, United States

Atlanta Diabetes Associates; 🇺🇸 Atlanta, Georgia, United States

Texas Diabetes & Endocrinology; 🇺🇸 Austin, Texas, United States