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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05210530
Recruitment Status : Active, not recruiting
First Posted : January 27, 2022
Last Update Posted : December 27, 2022
Sponsor:
Collaborator:
ViaCyte
Information provided by (Responsible Party):
CRISPR Therapeutics ( CRISPR Therapeutics AG )

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

Condition or disease Intervention/treatment Phase
Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Disease Endocrine System Diseases Autoimmune Diseases Immune System Diseases Combination Product: VCTX210A unit Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : January 24, 2022
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : April 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arms and Interventions
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Arm Intervention/treatment
Experimental: VCTX210A combination product
Up to ten (10) units will be implanted
 Combination Product: VCTX210A unit
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05210530


Locations
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Canada, Alberta
University of Alberta
Edmonton, Alberta, Canada
Canada, British Columbia
University of British Columbia
Vancouver, British Columbia, Canada
Canada, Ontario
LMC Manna
Toronto, Ontario, Canada
Sponsors and Collaborators
CRISPR Therapeutics AG
ViaCyte
Investigators
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Study Director: Manasi Jaiman, MD, MPH ViaCyte
Study Director: Sandeep Soni, MD CRISPR Therapeutics
More Information
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Responsible Party: CRISPR Therapeutics AG
ClinicalTrials.gov Identifier: NCT05210530    
Other Study ID Numbers: VCTX210A-101
First Posted: January 27, 2022    Key Record Dates
Last Update Posted: December 27, 2022
Last Verified: December 2022

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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
Keywords provided by CRISPR Therapeutics ( CRISPR Therapeutics AG ):
Type 1 Diabetes
T1D
Allogeneic
Combination Device
CRISPR-Cas9
 Cell Therapy
T1DM
Diabetes
VCTX
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Metabolic Diseases
Glucose Metabolism Disorders
 Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases