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

June 23, 2023 updated by: CRISPR Therapeutics AG

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

7

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • University of Alberta
    • British Columbia
      • Vancouver, British Columbia, Canada
        • University of British Columbia
    • Ontario
      • Toronto, Ontario, Canada
        • LMC Manna

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VCTX210A combination product
Up to seven (7) units will be implanted
Combination product: VCTX210A unit
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
From implantation up to 6 months post implantation

Secondary Outcome Measures

Outcome Measure
Time Frame
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
From implantation up to 6 months post implantation
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
From implantation up to 6 months post implantation
Incidence of new alloreactive antibodies found in the blood of patients post implantation.
Time Frame: From implantation up to 6 months post implantation
From implantation up to 6 months post implantation
Incidence of new autoreactive antibodies found in the blood of patients post implantation.
Time Frame: From implantation up to 6 months post implantation
From implantation up to 6 months post implantation
The percentage of viable graft cells per unit using immunohistochemical staining.
Time Frame: From implantation up to 6 months post implantation
From implantation up to 6 months post implantation
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
From implantation up to 6 months post implantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CRISPR Therapeutics AG

Collaborators

ViaCyte

Investigators

  • Study Director: Manasi Jaiman, MD, MPH, ViaCyte
  • Study Director: Sandeep Soni, MD, CRISPR Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 24, 2022

Primary Completion (Actual)

January 19, 2023

Study Completion (Actual)

January 19, 2023

Study Registration Dates

First Submitted

December 7, 2021

First Submitted That Met QC Criteria

January 18, 2022

First Posted (Actual)

January 27, 2022

Study Record Updates

Last Update Posted (Actual)

June 26, 2023

Last Update Submitted That Met QC Criteria

June 23, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VCTX210A-101

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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