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

Diabetes Mellitus Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05565248
• Other study ID #: VCTX211-101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: January 20, 2023
• Est. completion date: August 2025

Study information

• Verified date: March 2023
• Source: CRISPR Therapeutics
• Contact: Clinical Trials
• Phone: +1 (877) 214-4634
• Email: MedicalAffairs[@]crisprtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: August 2025
• Est. primary completion date: April 2025
• 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 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

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:
• VCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device

Locations

Country	Name	City	State
Canada	University of Alberta	Edmonton	Alberta
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 VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.		From implantation up to 12 months post implantation
Primary	Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.		From implantation up to 12 months post implantation
Secondary	Incidence of adverse events reported in patients implanted with VCTX211 units.		From implantation up to 12 months post implantation
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Incidence of new alloreactive antibodies found in the blood of patients post implantation.		From implantation up to 12 months post implantation
Secondary	Incidence of new autoreactive antibodies found in the blood of patients post implantation.		From implantation up to 12 months post implantation
Secondary	The percentage of viable graft cells per unit using immunohistochemical staining.		From implantation up to 12 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 12 months post implantation