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

Primary Purpose

Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders

Status
Completed
Phase
Phase 1
Locations
Canada
Study Type
Interventional
Intervention
VCTX210A unit
Sponsored by
CRISPR Therapeutics AG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diabetes Mellitus focused on measuring Type 1 Diabetes, T1D, Allogeneic, Combination Device, CRISPR-Cas9, Cell Therapy, T1DM, Diabetes, VCTX

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Alberta
  • University of British Columbia
  • LMC Manna

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VCTX210A combination product

Arm Description

Up to seven (7) units will be implanted

Outcomes

Primary Outcome Measures

Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.

Secondary Outcome Measures

Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
The percentage of viable graft cells per unit using immunohistochemical staining.
The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.

Full Information

First Posted
December 7, 2021
Last Updated
June 23, 2023
Sponsor
CRISPR Therapeutics AG
Collaborators
ViaCyte
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1. Study Identification

Unique Protocol Identification Number
NCT05210530
Brief Title
An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D
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)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
January 24, 2022 (Actual)
Primary Completion Date
January 19, 2023 (Actual)
Study Completion Date
January 19, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CRISPR Therapeutics AG
Collaborators
ViaCyte

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diabetes Mellitus, Diabetes Mellitus, Type 1, Glucose Metabolism Disorders, Metabolic Disease, Endocrine System Diseases, Autoimmune Diseases, Immune System Diseases
Keywords
Type 1 Diabetes, T1D, Allogeneic, Combination Device, CRISPR-Cas9, Cell Therapy, T1DM, Diabetes, VCTX

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VCTX210A combination product
Arm Type
Experimental
Arm Description
Up to seven (7) units will be implanted
Intervention Type
Combination Product
Intervention Name(s)
VCTX210A unit
Intervention Description
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device
Primary Outcome Measure Information:
Title
Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.
Time Frame
From implantation up to 6 months post implantation
Secondary Outcome Measure Information:
Title
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.
Time Frame
From implantation up to 6 months post implantation
Title
Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.
Time Frame
From implantation up to 6 months post implantation
Title
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Time Frame
From implantation up to 6 months post implantation
Title
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Time Frame
From implantation up to 6 months post implantation
Title
The percentage of viable graft cells per unit using immunohistochemical staining.
Time Frame
From implantation up to 6 months post implantation
Title
The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.
Time Frame
From implantation up to 6 months post implantation

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Manasi Jaiman, MD, MPH
Organizational Affiliation
ViaCyte
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Sandeep Soni, MD
Organizational Affiliation
CRISPR Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
Country
Canada
Facility Name
University of British Columbia
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
LMC Manna
City
Toronto
State/Province
Ontario
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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