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

NCT04678557 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: VC01-103
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 4

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.

Conditions

Type 1 Diabetes

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (2)

Sentinel units (aka Cohort 1)

EXPERIMENTAL

VC-01 Combination Product; Up to ten (10) VC-01 sentinels

Combination Product: VC-01 Combination Product

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

Combination Product: VC-01 Combination Product

Interventions

VC-01 Combination Product COMBINATION_PRODUCT

PEC-01 cells loaded into an Encaptra Drug Delivery System

Also known as: PEC-Encap

Dose-finding units (aka Cohort 2); Sentinel units (aka Cohort 1)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Advanced complications associated with diabetes

Sponsors & Collaborators

• ViaCyte: lead

Study Sites (4)

AMCR Institute

Escondido, California, 92025, United States

Location

Diablo Clinical Research

Walnut Creek, California, 94598, United States

Location

Atlanta Diabetes Associates

Atlanta, Georgia, 30318, United States

Location

Texas Diabetes & Endocrinology

Austin, Texas, 78731, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Limitations and Caveats

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.

Results Point of Contact

• Title: Medical Information
• Organization: Vertex

medicalinfo@vrtx.com

617-341-6777

Study Officials

• Manasi Jaiman, MD

  ViaCyte, Inc.

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 30, 2020
• First Posted: December 22, 2020
• Study Start: June 25, 2019
• Primary Completion: November 12, 2021
• Study Completion: November 19, 2021
• Last Updated: October 18, 2023
• Results First Posted: October 18, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)