RECRUITING

A Study to Investigate Safety and Effectiveness of Pancreatic Cells Derived from Pigs (OPF-310) in Patients with Type 1 Diabetes Mellitus

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2025-04-01

Study Completion:2027-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06575426

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.

Ages Eligible for Study:35 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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. 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. 5. Subject has C-peptide \<0.3 ng/mL following a mixed meal tolerance test or undetectable fasting C-peptide. 6. Hemoglobin A1C (HbA1c) ≥ 7.5 and ≤ 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	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 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 non-insulin hypoglycemic medication not intended to be used as an adjunct to 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

Inclusion Criteria

Exclusion 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. 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.
5. Subject has C-peptide \<0.3 ng/mL following a mixed meal tolerance test or undetectable fasting C-peptide.
6. Hemoglobin A1C (HbA1c) ≥ 7.5 and ≤ 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

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
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 non-insulin hypoglycemic medication not intended to be used as an adjunct to 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

Contacts and Locations

Study Contact

Takayuki Hasegawa

CONTACT

8474661050

Takayuki.Hasegawa@otsuka-us.com

Yuka Ishizaki

CONTACT

8474661050

Yuka.Ishizaki@otsuka-us.com

Study Locations (Sites)

University of Illinois Hospital &Health Scences System

Chicago, Illinois, 60612

United States

Collaborators and Investigators

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 Registration Dates

Study Start Date2025-04-01

Study Completion Date2027-06-30

Study Record Updates

Study Start Date2025-04-01

Study Completion Date2027-06-30

Terms related to this study

Additional Relevant MeSH Terms

Diabetes Mellitus, Type 1
Hypoglycemia