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

Diabetes Mellitus Clinical Trial

Official title:

An Open-Label, First-In-Human Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05210530
• Other study ID #: VCTX210A-101
• Status: Completed
• Phase: Phase 1
• Start date: January 24, 2022
• Est. completion date: January 19, 2023

Study information

• Verified date: June 2023
• Source: CRISPR Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: January 19, 2023
• Est. primary completion date: January 19, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Combination Product:
• VCTX210A unit
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
Canada	LMC Manna	Toronto	Ontario
Canada	University of British Columbia	Vancouver	British Columbia

Sponsors (2)

Lead Sponsor	Collaborator
CRISPR Therapeutics AG	ViaCyte

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.		From implantation up to 6 months post implantation
Secondary	Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.		From implantation up to 6 months post implantation
Secondary	Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.		From implantation up to 6 months post implantation
Secondary	Incidence of new alloreactive antibodies found in the blood of patients post implantation.		From implantation up to 6 months post implantation
Secondary	Incidence of new autoreactive antibodies found in the blood of patients post implantation.		From implantation up to 6 months post implantation
Secondary	The percentage of viable graft cells per unit using immunohistochemical staining.		From implantation up to 6 months post implantation
Secondary	The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.		From implantation up to 6 months post implantation