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)

CRISPR Therapeutics AG2 sites in 1 country40 target enrollmentJanuary 20, 2023

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

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Diabetes Mellitus
Sponsor: CRISPR Therapeutics AG
Enrollment: 40
Locations: 2
Primary Endpoint: 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.
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

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

Detailed 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.

Registry

clinicaltrials.gov

Start Date

January 20, 2023

End Date

August 2025

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

CRISPR Therapeutics AG

Responsible Party

Sponsor

Eligibility Criteria

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

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.

Time Frame: From implantation up to 12 months post implantation

Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.

Time Frame: From implantation up to 12 months post implantation

Secondary Outcomes

Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline.(From implantation up to 12 months post implantation)
The percentage of viable graft cells per unit using immunohistochemical staining.(From implantation up to 12 months post implantation)
Incidence of adverse events reported in patients implanted with VCTX211 units.(From implantation up to 12 months post implantation)
Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline.(From implantation up to 12 months post implantation)
Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline.(From implantation up to 12 months post implantation)
Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline.(From implantation up to 12 months post implantation)
Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft.(From implantation up to 12 months post implantation)
Incidence of new alloreactive antibodies found in the blood of patients post implantation.(From implantation up to 12 months post implantation)
Incidence of new autoreactive antibodies found in the blood of patients post implantation.(From implantation up to 12 months post implantation)
The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.(From implantation up to 12 months post implantation)