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

• Sponsor: CRISPR Therapeutics AG (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05210530
• Collaborator: ViaCyte (Industry)
• Enrollment: 10
• Locations: 3
• Arm: 1
• Anticipated Duration (Months): 10.2
• Patients Per Site: 3.3
• Patients Per Site Per Month: 0.3

Study Details

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

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

• Study Type: Interventional
• Anticipated 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: Jan 24, 2022
• Anticipated Primary Completion Date: Dec 1, 2022
• Anticipated Study Completion Date: Dec 1, 2022

Arms and Interventions

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

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. [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. [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. [From implantation up to 6 months post implantation]

3. Incidence of new alloreactive antibodies found in the blood of patients post implantation. [From implantation up to 6 months post implantation]

4. Incidence of new autoreactive antibodies found in the blood of patients post implantation. [From implantation up to 6 months post implantation]

5. The percentage of viable graft cells per unit using immunohistochemical staining. [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. [From implantation up to 6 months post implantation]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	University of Alberta	Edmonton	Alberta	Canada
2	University of British Columbia	Vancouver	British Columbia	Canada
3	LMC Manna	Toronto	Ontario	Canada

Sponsors and Collaborators

• CRISPR Therapeutics AG
• ViaCyte

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: CRISPR Therapeutics AG
• ClinicalTrials.gov Identifier: NCT05210530
• Other Study ID Numbers: VCTX210A-101
• First Posted: Jan 27, 2022
• Last Update Posted: Apr 4, 2022
• Last Verified: Feb 1, 2022

• 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 AG: Type 1 Diabetes; T1D; Allogeneic; Combination Device; CRISPR-Cas9; Cell Therapy; T1DM; Diabetes; VCTX
• Additional relevant MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1; Metabolic Diseases; Glucose Metabolism Disorders; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Results

No Results Posted as of Apr 4, 2022