This study is First In Human study for Encapsulated Porcine Islet Cells for Xenotransplantation (OPF-310). The purpose of this study to assess the safety, tolerability, and efficacy of OPF-310 transplantation and to define the recommended Phase 2 dose (RP2D) in adult subjects with unstable Type 1 Diabetes Mellitus (T1DM) and a level 3 (severe) hypoglycemic episode at least three times within the 1 year prior to enrollment despite treatment with a closed loop system (CLS) for at least 6 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Dose(Part1): 6,000 islet equivalents (IEQ)/kg or 12,000 islet equivalents (IEQ)/kg Dose(Part2): Recommended Phase II Dose(RP2D), which will be determined based on the data of Part1 * In Part1, three subjects will be enrolled into the first dosing cohort (Cohort 1: 6,000 IEQ/kg) and they will undergo safety monitoring. Three subjects in Cohort 2 will be dosed with 12,000 IEQ/kg and will undergo safety monitoring. * In Part2, 7 subjects will be enrolled into the Part 2 dose-expansion part.
University of Illinois Hospital & Health Sciences System
Chicago, Illinois, United States
RECRUITINGPercentage of Subjects Reaching the Efficacy Goal
A successful primary endpoint was defined as achieve both an HbA1c \< 7 % and a reduction of at least 0.5% from baseline, and absence of a Level 3 (severe) hypoglycemic episode from 12 weeks to 52 weeks post-transplant.
Time frame: One year after transplant
Percentage reduction in nocturnal hypoglycemic event rate
Change from baseline in nocturnal hypoglycemic event rate specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Percentage improvement in time below range (<70 mg/dl) by continuous glucose monitoring (CGM)
Change from baseline in the CGM data specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Percentage improvement in time in range (70-180 mg/dl) by CGM
Change from baseline in the CGM data specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Percentage improvement in time above range (>180 mg/dl) by CGM
Change from baseline in the CGM data specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Change in mean amplitude of glycemic excursion (MAGE) by CGM.
Change from baseline in MAGE based on the CGM data specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Percentage reduction in daily average of insulin use
Change from baseline in insulin use specified in the protocol
Time frame: 12 week, 24 week 52 weeks after transplant
Percentage of subjects with an HbA1c < 7.0 % assessed at 52 weeks post-transplant
Time frame: One year after transplant
Percentage of subjects with an HbA1c ≤ 6.5 % assessed at 52 weeks post-transplant
Time frame: One year after transplant
Percentage of subjects with positive porcine C-peptide qualitatively assessed by digital ELISA assay
Time frame: One year after transplant
Values of porcine C-peptide, human c-peptide, and glucose during mixed meal tolerance test (MMTT) and Intravenous Glucose Tolerance Test (IVGTT) at each measuring point
Assesment of glucose metabolism function
Time frame: For one year after transplant
Psychological impact as assessed by the Diabetes Distress Scale (DDS) and the Hypoglycemic Fear Survey (HFS) at each measuring point.
Patient Quality of Life Assessment with DDS and HFS
Time frame: For one year after transplant
Percentage of subjects with improved awareness of hypoglycemia, as defined by a change in Clarke questionnaire score from ≥ 4 to < 4.
Time frame: For one year after transplant
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