An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D

CRISPR Therapeutics AG7 enrolled

Overview

This is an open-label, multicenter, Phase 1 study evaluating the safety and tolerability of VCTX210A combination product in patients with T1D

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Disease Endocrine System Diseases Autoimmune Diseases Immune System Diseases

Interventions

VCTX210A unitCOMBINATION_PRODUCT

CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device

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, optimized diabetic 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 (3)

University of Alberta

Edmonton, Alberta, Canada

University of British Columbia

Vancouver, British Columbia, Canada

LMC Manna

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.