An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D
Study Details
Study Description
Brief Summary
This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 1/Phase 2 |
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.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: VCTX211 unit
|
Combination Product: VCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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]
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
No locations specified.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.- VCTX211-101