Clinical trial NCT05565248: An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D
Official title: An Open-Label, First-in-Human Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Disease
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases
Keywords: Type 1 Diabetes
T1D
Allogeneic
Combination Device
CRISPR-Cas9
Cell Therapy
T1DM
Diabetes
VCTX
Lead sponsor: CRISPR Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: ViaCyte
First posted: 2022-10-04
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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.

Eligibility 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

Primary outcomes:
measure: 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.
timeFrame: From implantation up to 12 months post implantation
measure: Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.
timeFrame: From implantation up to 12 months post implantation
Secondary outcomes:
measure: Incidence of adverse events reported in patients implanted with VCTX211 units.
timeFrame: From implantation up to 12 months post implantation
measure: Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline.
timeFrame: From implantation up to 12 months post implantation
measure: Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline.
timeFrame: From implantation up to 12 months post implantation
measure: Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline.
timeFrame: From implantation up to 12 months post implantation
measure: 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.
timeFrame: From implantation up to 12 months post implantation
measure: Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft.
timeFrame: From implantation up to 12 months post implantation
measure: Incidence of new alloreactive antibodies found in the blood of patients post implantation.
timeFrame: From implantation up to 12 months post implantation
measure: Incidence of new autoreactive antibodies found in the blood of patients post implantation.
timeFrame: From implantation up to 12 months post implantation
measure: The percentage of viable graft cells per unit using immunohistochemical staining.
timeFrame: From implantation up to 12 months post implantation
measure: The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.
timeFrame: From implantation up to 12 months post implantation
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: University of British Columbia
state: British Columbia
Study officials:
affiliation: ViaCyte
name: Manasi Jaiman, MD, MPH
role: STUDY_DIRECTOR
affiliation: CRISPR Therapeutics
name: Sandeep Soni, MD
role: STUDY_DIRECTOR
References: