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

CRISPR Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

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

Treatments: Combination Product: VCTX211

Study type: Interventional

Funder types: Industry

Identifiers

NCT05565248

VCTX211-101

Details and patient eligibility

About

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

Full description

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.

Enrollment: 40 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

VCTX211 unit

Experimental group

Treatment:

Combination Product: VCTX211