An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D

Phase 2RecruitingNCT05565248

CRISPR Therapeutics AG40 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Disease

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of T1D for a minimum of 5 years * Stable diabetes regimen for at least 3 months prior to enrollment. Exclusion Criteria: * Medical history of islet cell, kidney, and/or pancreas transplant * Occurrence of 2 or more severe, unexplained hypoglycemic events within 6 months prior to enrollment * Known causes of diabetes other than T1D * Immunosuppressant therapy in the previous 30 days and/or requirements for chronic immunosuppressive therapy during the study * Prior treatment with gene therapy or edited product

Locations (2)

University of Alberta

Edmonton, Alberta, Canada

RECRUITING

University of British Columbia

Vancouver, British Columbia, Canada

RECRUITING

Outcomes

Primary Outcomes

Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.