Clinical Trial

Disease: Type 1 Diabetes, T1D, (NCT05565248)

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, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
Who:

Contact

Name: Clinical Trials

Phone Number: +1 (877) 214-4634

Email: MedicalAffairs@crisprtx.com

 

 

Partners:
Locations:

Canada, Alberta

University of Alberta, Edmonton, Alberta, Canada

Contact: Sara Qureshi saira@ualberta.ca   

 

Canada, British Columbia

University of British Columbia, Vancouver, British Columbia, Canada

Contact: Study Coordinator  
 

Study start:
Jan. 20, 2023
Enrollment:
40 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Disruption and insertion
Gene:
Delivery method:
- Ex-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D.

VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster 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 PEC211 cells.

Last updated: Dec. 27, 2023
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