A Study to Investigate Safety and Effectiveness of Pancreatic Cells Derived From Pigs (OPF-310) in Patients With Type 1 Diabetes Mellitus
NCT Number: NCT06575426
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1|PHASE2
Conditions: Diabetes Mellitus, Type 1|Hypoglycemia
Interventions: DRUG: OPF-310
Sponsor: Otsuka Pharmaceutical Factory, Inc.
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 13
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-12-28
Completion Date: 2027-06-30

Summary:
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.

Primary Outcome Measures: Percentage of Subjects Reaching the Efficacy Goal, A successful primary endpoint was defined as hemoglobin A1C (HbA1c) \< 7.0% and absence of a Level 3 (severe) hypoglycemic episode from 12 weeks to 52 weeks post-transplant., One year after transplant
Secondary Outcome Measures: Percentage of subjects with reduction in HbA1c more than 1 %, Change from baseline in HbA1c specified in the protocol, 12 week, 24 week 52 weeks after transplant|Percentage reduction in nocturnal hypoglycemic event rate, Change from baseline in nocturnal hypoglycemic event rate specified in the protocol, 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, 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, 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, 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, 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, 12 week, 24 week 52 weeks after transplant|Percentage of subjects with an HbA1c < 7.0 % assessed at 52 weeks post-transplant, One year after transplant|Percentage of subjects with an HbA1c ≤ 6.5 % assessed at 52 weeks post-transplant, One year after transplant|Percentage of subjects with positive porcine C-peptide qualitatively assessed by digital ELISA assay, One year after transplant|Values of porcine C-peptide during mixed meal tolerance test (MMTT) and Intravenous Glucose Tolerance Test (IVGTT) at each measuring point, Assesment of glucose metabolism function, For one year after transplant|Values of human C-peptide during mixed meal tolerance test (MMTT) and Intravenous Glucose Tolerance Test (IVGTT) at each measuring point, Assesment of glucose metabolism function, For one year after transplant|Values of Glucose during mixed meal tolerance test (MMTT) and Intravenous Glucose Tolerance Test (IVGTT) at each measuring point, Assesment of glucose metabolism function, For one year after transplant|Psychological impact as assessed by the Diabetes Distress Scale (DDS) at each measuring point., Patient Quality of Life Assessment with DDS, For one year after transplant|Psychological impact as assessed by the Hypoglycemic Fear Survey (HFS) at each measuring point., Patient Quality of Life Assessment with HFS, 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., For one year after transplant