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NCT Number: NCT05210530

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

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

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Key information

Conditions

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

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Alberta, Edmonton, Alberta, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

VCTX210A unit

Combination Product

CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device

Primary outcomes

  1. 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 outcomes

  1. 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

  2. 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

  3. Incidence of new alloreactive antibodies found in the blood of patients post implantation.

    Time frame: From implantation up to 6 months post implantation

  4. Incidence of new autoreactive antibodies found in the blood of patients post implantation.

    Time frame: From implantation up to 6 months post implantation

  5. The percentage of viable graft cells per unit using immunohistochemical staining.

    Time frame: From implantation up to 6 months post implantation

  6. 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

Sponsors and collaborators

Lead sponsor

CRISPR Therapeutics AG

Industry

Collaborators

  • ViaCyte

Registry information

Official study 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)

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Jan 27, 2022
Registry last updated
Jun 26, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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