Clinical Trial

Disease: Type 1 Diabetes, T1D, (NCT05210530)

Disease info:

Diabetes is a chronic disease that affects the bodies ability to metabolise food. Individuals with diabetes fail to make sifficient insulin, or cannnot utilise insulin made in the body efficiently to break down sugar.

Type 1 diabetes (T1D) is an autoimmune disease that results in destruction of the insulin-producing beta cells of the pancreas. The resulting insulin deficiency leads to rising blood glucose levels that over time can lead to a range of complications involving the heart, eyes, kidneys, nerves, gums and teeth. Approximately 5-10% of diebetes patients have type 1.

T1D may debut at any age but it usually begins in childhood or early adulthood, and is fatal if untreated. Symptoms usually develop rapidly and patients are required to take insulin every day. The standard current treatment is regular blood glucose monitoring and subcutaneous administration of modified human insulin expressed in heterologous hosts, e.g., E. coli.

The exact genetic causes of T1D are currently unknown, as the disease is multifactorial, but several risk factors have been identified, with certain variants of the HLA-DQA1HLA-DQB1, and HLA-DRB1 genes being associated with the disease. 

Frequency:
Approximately 1 out of every 300 individuals will develop type 1 diabetes by the age of 18 in the United States and Europe. The rate is much lower in Asia and South America with only 1 in 1 million new cases per year.
Official title:
An Open-Label, First-In-Human Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
Who:

Investigators: Manasi Jaiman, Sandeep Soni

Partners:
Locations:

Canada, Alberta

University of Alberta, Edmonton, Alberta, Canada

 

Canada, British Columbia

University of British Columbia, Vancouver, British Columbia, Canada

 

Canada, Ontario

LMC Manna, Toronto, Ontario, Canada

Study start:
Jan. 24, 2022
Enrollment:
10
Gene editing method:
CRISPR-Cas9
Type of edit:
Disruption and insertion
Gene:
Undisclosed
Delivery method:
Undisclosed - Ex-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed

Description

VCTX210A combination product (unit) comprises 2 components: (1) allogeneic pancreatic endoderm cells (PEC210A) genetically modified using Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain the PEC210A cells.

Last updated: Apr. 20, 2024
close
Search CRISPR Medicine