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

Phase 1

Recruiting

• Conditions: Diabetes Mellitus; Metabolic Disease; Immune System Diseases; Diabetes Mellitus, Type 1; Endocrine System Diseases; Glucose Metabolism Disorders; Autoimmune Diseases
• Interventions: Combination Product: VCTX211

• Registration Number: NCT05565248
• Lead Sponsor: CRISPR Therapeutics AG

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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Study Timeline

• Study First Submitted: 2022-09-30
• Study First Submitted QC: 2022-09-30
• Study First Posted: 2022-10-04
• Study Start: 2023-01-20
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-22
• Last Update Posted: 2024-05-23
• Primary Completion: 2025-04
• Study Completion: 2025-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Diagnosis of T1D for a minimum of 5 years
• Stable diabetes regimen for at least 3 months prior to enrollment.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
VCTX211 unit	VCTX211	-

Primary Outcome Measures

Name	Time	Method
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
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (2)

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

University of British Columbia; 🇨🇦 Vancouver, British Columbia, Canada