A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus

February 26, 2026 updated by: Otsuka Pharmaceutical Factory, Inc.

A Phase I/IIa, Single Site, Open-Label, Ascending Dose Study to Evaluate the Safety and Efficacy of OPF-310 [Encapsulated Porcine Islet Cells for Xenotransplantation] in Subjects With Type 1 Diabetes Mellitus

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 Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

13

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • University of Illinois Hospital & Health Sciences System
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Subject must be aged 35 to 65 years of age inclusive, at the time of signing the informed consent.
  2. Subject has an established diagnosis of type 1 diabetes mellitus (T1DM)(in accordance with the American Diabetes Association's criteria), with a minimum duration since diagnosis of 5 years.

  3. If one of the following criteria (either a or b) applies:

    1. Subject has unstable T1DM, not achieving adequate control after receiving CLS (CGM:Dexcom G6, insulin pump: Omnipod® 5 or t:slim X2) under care of a qualified diabetes team for at least 6 months prior to enrollment.
    2. Subject has unstable T1DM, not achieving adequate control after receiving CLS (CGM:Dexcom G7, insulin pump: Omnipod® 5, t:slim X2, iLet Bionic Pancreas or The Tandem Mobi System) under care of a qualified diabetes team for at least 6 months prior to enrollment.

  4. If one of the following criteria (either a, b or c) applies:

    1. Subject has had a Level 3 (severe) hypoglycemic episode (defined as having cognitive impairment requiring external assistance for recovery) at least three times within the 1 year prior to enrollment recorded in the medical record or patient log.
    2. Subject has had a Level 3 (severe) hypoglycemic episode at least once within the 1 year prior to enrollment and demonstrates a Clarke Score ≥4, assessed by trained study personnel. The SHE(s) and Clarke Score must be recorded in the medical record or patient log.
    3. Subject has had TBR >1% at glucose levels below 70mg/dL and demonstrates a Clarke Score≥4, assessed by trained study personnel. TBR data used for screening and Clarke score must be recorded in the medical record or patient log.
  5. Subject has C-peptide <0.3 ng/mL following a mixed meal tolerance test or undetectable fasting C-peptide.
  6. Hemoglobin A1C (HbA1c) ≤ 9.0
  7. Contraceptive use must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  8. Subject who can agree to cooperate with lifetime follow-up after transplantation.
  9. Subject is capable of providing signed informed consent

Exclusion Criteria:

  1. Previous history of insulin resistance (defined as an average insulin dose requirement ≥ 0.8 unit/kg/day for 1 week prior to enrollment).
  2. Subject has latent autoimmune diabetes in adults (LADA), ketosis-prone (Flatbush) diabetes, or maturity onset diabetes of the young (MODY).
  3. CRP ≥ 10 mg/L.
  4. Clinically unstable thyroid disease (thyroid stimulating hormone (TSH)< the lower limit of the normal range of TSH at the site.) Patients with subclinical hyperthyroidism can be rescreened once TSH levels normalize due to treatment or other factors. In addition, patients with transiently abnormal TSH levels may undergo rescreening only once during the screening period.
  5. History of malignancies within the past 5 years, excluding basal and squamous cell carcinoma
  6. Positive serologies or nucleic acid testing for human immunodeficiency virus (HIV), hepatitis C, and hepatitis B.
  7. Active or untreated proliferative diabetic retinopathy. Subjects may be rescreened once they are successfully treated.

  8. Serious comorbid conditions that are likely to affect participation in the study, including:

    1. Within the last 12 months, peripheral vascular disease with previous amputation.
    2. History of New York Heart Association (NYHA) class II, III or IV congestive heart failure (CHF) and/or chronic atrial fibrillation.
    3. Chronic obstructive pulmonary disease (COPD) or asthma with previous hospitalization for decompensation; a requirement for mechanical ventilation at any stage; or long- term treatment with oral corticosteroids.
    4. Macroalbuminuria (> 300 mg albumin/gm creatinine).
    5. Estimated glomerular filtration rate (eGFR) cut-off of < 30 ml/min for all per Kidney Disease Improving Global Outcomes (KDOQI) and Kidney Disease Outcomes Quality Initiative (KDIGO) consensus.
  9. Use of warfarin or other anticoagulant therapy (except aspirin), or prothrombin time and international normalized ratio (PT-INR) > 1.5
  10. Adrenal insufficiency being treated with corticosteroids
  11. Previous pan-peritonitis

  12. Previous cardiovascular or cerebrovascular disease. NOTE: For the purposes of this exclusion criterion, "previous cardiovascular disease" is defined as the presence of co-existing cardiac disease, characterized by any of the following conditions:

    1. Recent myocardial infarction (within past one year), or
    2. Angiographic evidence of non-correctable coronary artery disease, or
    3. Evidence of ischemia on functional cardiac exam (with a stress echo test recommended for subjects with a history of ischemic disease), or
    4. Heart failure > NYHAII
  13. Patients with hematopoietic stem cell abnormalities (e.g., aplastic anemia, myelodysplastic syndrome)
  14. Patients who received a blood transfusion in the previous 90 days, are anticipated to undergo surgery during the 1-year study period that may require transfusion, or have donated blood within the previous 90 days.
  15. Previous receipt of an organ, skin allograft, or other tissue transplant from an allogeneic human or animal donor.
  16. Treatment with immunosuppressive medication.
  17. Previous abdominal surgery, excluding uncomplicated appendectomy, cholecystectomy, exploratory laparoscopy and hernia repair performed prior to 12 weeks prior to enrollment.
  18. Treatment with any hypoglycemic medication prescribed for glycemic control, other than insulin therapy.
  19. Treatment with acetaminophen or hydroxycarbamide.
  20. Use of any investigational products within 12 weeks of enrollment (before entering run-in) or 5 half-lives of the investigational product, whichever is greater.
  21. Subject has history of allergy to antibiotics (Amphotericin B, Cefazolin, Ciprofloxacin, Gentamicin), which are used during manufacture of OPF-310.
  22. Previous history of insulin allergy (including porcine insulin), pork product allergy or alginate/seaweed allergy.
  23. Panel reactive antibodies (PRA) > 80 %.
  24. Active drug, substance or alcohol addiction.
  25. Body mass index (BMI) >27 kg/m2.
  26. Any other condition that, in the opinion of the Investigator, may interfere with adherence to the study protocol, including dementia, psychiatric disorder, medical condition, or a history of non-adherence to appointments or treatments

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: OPF-310
13 patients will be transplanted OPF-310.
Combination product: OPF-310

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Subjects Reaching the Efficacy Goal
Time Frame: One year after transplant
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.
One year after transplant

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage reduction in nocturnal hypoglycemic event rate
Time Frame: 12 week, 24 week 52 weeks after transplant
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)
Time Frame: 12 week, 24 week 52 weeks after transplant
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
Time Frame: 12 week, 24 week 52 weeks after transplant
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
Time Frame: 12 week, 24 week 52 weeks after transplant
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.
Time Frame: 12 week, 24 week 52 weeks after transplant
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
Time Frame: 12 week, 24 week 52 weeks after transplant
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
Time Frame: One year after transplant
One year after transplant
Percentage of subjects with an HbA1c ≤ 6.5 % assessed at 52 weeks post-transplant
Time Frame: One year after transplant
One year after transplant
Percentage of subjects with positive porcine C-peptide qualitatively assessed by digital ELISA assay
Time Frame: One year after transplant
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
Time Frame: For one year after transplant
Assesment of glucose metabolism function
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
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.
Time Frame: For one year after transplant
Patient Quality of Life Assessment with DDS and HFS
For one year after transplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Otsuka Pharmaceutical Factory, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 10, 2025

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

August 20, 2024

First Submitted That Met QC Criteria

August 26, 2024

First Posted (Actual)

August 28, 2024

Study Record Updates

Last Update Posted (Actual)

March 2, 2026

Last Update Submitted That Met QC Criteria

February 26, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 215-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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