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, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
Detailed description
VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster 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 PEC211 cells.
Interventions
COMBINATION_PRODUCTVCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device
Sponsors
CRISPR Therapeutics AG
ViaCyte
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 diabetes 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 VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units. | From implantation up to 12 months post implantation |
| Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline. | From implantation up to 12 months post implantation |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of adverse events reported in patients implanted with VCTX211 units. | From implantation up to 12 months post implantation |
| Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline. | From implantation up to 12 months post implantation |
| Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline. | From implantation up to 12 months post implantation |
| Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline. | From implantation up to 12 months post implantation |
| Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline. | From implantation up to 12 months post implantation |
| Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft. | From implantation up to 12 months post implantation |
| Incidence of new alloreactive antibodies found in the blood of patients post implantation. | From implantation up to 12 months post implantation |
| Incidence of new autoreactive antibodies found in the blood of patients post implantation. | From implantation up to 12 months post implantation |
| The percentage of viable graft cells per unit using immunohistochemical staining. | From implantation up to 12 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 12 months post implantation |
Countries
Canada
Contacts
STUDY_DIRECTORManasi Jaiman, MD, MPH
ViaCyte
STUDY_DIRECTORSandeep Soni, MD
CRISPR Therapeutics
Outcome results
None listed