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

• ClinicalTrials.gov Identifier: NCT05210530
• Recruitment Status: Completed
• First Posted: January 27, 2022
• Last Update Posted: June 26, 2023
• Sponsor: CRISPR Therapeutics AG
• Collaborator: ViaCyte
• Information provided by (Responsible Party): CRISPR Therapeutics ( CRISPR Therapeutics AG )

Tracking Information

• First Submitted Date: December 7, 2021
• First Posted Date: January 27, 2022
• Last Update Posted Date: June 26, 2023
• Actual Study Start Date: January 24, 2022
• Actual Primary Completion Date: January 19, 2023 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: January 18, 2022): 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 ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 18, 2022)

• 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 ]
• 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 ]
• Incidence of new alloreactive antibodies found in the blood of patients post implantation. [ Time Frame: 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 ]
• The percentage of viable graft cells per unit using immunohistochemical staining. [ Time Frame: 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 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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)
• 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.
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Intervention

Combination Product: VCTX210A unit

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

Study Arms

Experimental: VCTX210A combination product

Up to seven (7) units will be implanted

Intervention: Combination Product: VCTX210A unit

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 23, 2023): 7
• Original Estimated Enrollment (submitted: January 18, 2022): 10
• Actual Study Completion Date: January 19, 2023
• Actual Primary Completion Date: January 19, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada

Administrative Information

• NCT Number: NCT05210530
• Other Study ID Numbers: VCTX210A-101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• 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

• IPD Sharing Statement: Not Provided
• Current Responsible Party: CRISPR Therapeutics ( CRISPR Therapeutics AG )
• Original Responsible Party: Same as current
• Current Study Sponsor: CRISPR Therapeutics AG
• Original Study Sponsor: Same as current
• Collaborators: ViaCyte

Investigators

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

• PRS Account: CRISPR Therapeutics
• Verification Date: June 2023