Autoimmune Diseases, Diabetes Mellitus, Diabetes Mellitus, Type 1, Endocrine System Diseases, Glucose Metabolism Disorders, Immune System Diseases, Metabolic Disease
Conditions
Keywords
Type 1 Diabetes, T1D, Allogeneic, Combination Device, CRISPR-Cas9, Cell Therapy, T1DM, Diabetes, VCTX
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.
Interventions
COMBINATION_PRODUCTVCTX210A unit
CRISPR-Cas9 genetically modified PEC210A cells loaded into a delivery device
Sponsors
ViaCyte
CRISPR Therapeutics AG
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
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
Design outcomes
Primary
| 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. | From implantation up to 6 months post implantation |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| Incidence of new alloreactive antibodies found in the blood of patients 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 innate immune cells within the graft. | From implantation up to 6 months post implantation |
| The percentage of viable graft cells per unit using immunohistochemical staining. | 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. | From implantation up to 6 months post implantation |
| Incidence of new autoreactive antibodies found in the blood of patients post implantation. | From implantation up to 6 months post implantation |
Countries
Canada
Outcome results
None listed