Diabetes Mellitus, Type 1Phase 1/2NCT06575426
A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus
A Phase 1/2 study testing OPF-310 in people with Diabetes Mellitus, Type 1 and Hypoglycemia, run by Otsuka Pharmaceutical Factory, Inc. at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 35 to 70 years
- Conditions the study is for
- Diabetes Mellitus, Type 1, Hypoglycemia, Islet Cell Transplantation
- What is being tested?
- OPF-310
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Jun 2025to 30 Jun 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject must be aged 35 to 70 years of age inclusive, at the time of signing the informed consent.
- 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.
- If one of the following criteria (either a or b) applies:
- 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.
- 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.
- If one of the following criteria (either a, b or c) applies:
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- 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.
- 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.
- 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.
- Subject has C-peptide <0.3 ng/mL following a mixed meal tolerance test or undetectable fasting C-peptide.
- Hemoglobin A1C (HbA1c) ≤ 9.0
- Contraceptive use must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- Subject who can agree to cooperate with lifetime follow-up after transplantation.
- Subject is capable of providing signed informed consent
Who cannot join
- Previous history of insulin resistance (defined as an average insulin dose requirement ≥ 0.8 unit/kg/day for 1 week prior to enrollment).
- Subject has latent autoimmune diabetes in adults (LADA), ketosis-prone (Flatbush) diabetes, or maturity onset diabetes of the young (MODY).
- CRP ≥ 10 mg/L.
- 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.
- History of malignancies within the past 5 years, excluding basal and squamous cell carcinoma
- Positive serologies or nucleic acid testing for human immunodeficiency virus (HIV), hepatitis C, and hepatitis B.
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- Active or untreated proliferative diabetic retinopathy. Subjects may be rescreened once they are successfully treated.
- Serious comorbid conditions that are likely to affect participation in the study, including:
- Within the last 12 months, peripheral vascular disease with previous amputation.
- History of New York Heart Association (NYHA) class II, III or IV congestive heart failure (CHF) and/or chronic atrial fibrillation.
- 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.
- Macroalbuminuria (> 300 mg albumin/gm creatinine).
- 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.
- Use of warfarin or other anticoagulant therapy (except aspirin), or prothrombin time and international normalized ratio (PT-INR) > 1.5
- Adrenal insufficiency being treated with corticosteroids
- Previous pan-peritonitis
- 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:
- Recent myocardial infarction (within past one year), or
- Angiographic evidence of non-correctable coronary artery disease, or
- Evidence of ischemia on functional cardiac exam (with a stress echo test recommended for subjects with a history of ischemic disease), or
- Heart failure > NYHAII For subjects aged 65 to <70 years who do not meet Exclusion Criterion 12 but have a history of cardiovascular or cerebrovascular disease related to the conditions above, a cardiology consultation (and consultation with other relevant specialists, as appropriate) will be required during the screening period to confirm suitability for general anesthesia and laparoscopic surgery.
- Patients with hematopoietic stem cell abnormalities (e.g., aplastic anemia, myelodysplastic syndrome)
- 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.
- Previous receipt of an organ, skin allograft, or other tissue transplant from an allogeneic human or animal donor.
- Treatment with immunosuppressive medication.
- Previous abdominal surgery, excluding uncomplicated appendectomy, cholecystectomy, exploratory laparoscopy and hernia repair performed prior to 12 weeks prior to enrollment.
- Treatment with any hypoglycemic medication prescribed for glycemic control, other than insulin therapy.
- Treatment with acetaminophen or hydroxycarbamide.
- 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.
- Subject has history of allergy to antibiotics (Amphotericin B, Cefazolin, Ciprofloxacin, Gentamicin), which are used during manufacture of OPF-310.
- Previous history of insulin allergy (including porcine insulin), pork product allergy or alginate/seaweed allergy.
- Panel reactive antibodies (PRA) > 80 %.
- Active drug, substance or alcohol addiction.
- Body mass index (BMI) >27 kg/m2.
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
For participant-focused inquiries, please contact: contact
For physician or professional inquiries, please contact: contact
United States1 site, 1 recruiting
- Recruiting
University of Illinois Hospital & Health Sciences System
Chicago, Illinois, 60612
Benito Valdepenas, 312-355-3113, bvalde2@uic.edu
About this study
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
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.
- Conditions
- Diabetes Mellitus, Type 1, Hypoglycemia, Islet Cell Transplantation, Type 1 Diabetes, Type 1 Diabetes Mellitus, T1D
- Being tested
- OPF-310
- Keywords
- Diabetes Mellitus, Diabetes Mellitus, Type1, T1DM, Hypoglycemia, islet cell transplantation, pig islet cell transplantation, Xenotransplantation, Porcine islet cell transplantation, Type 1 Diabetes Mellitus, Type 1 Diabetes
- Medical subject headings
- Diabetes Mellitus, Type 1, Hypoglycemia, Glucose Metabolism Disorders, Immune System Diseases, Autoimmune Diseases, Metabolic Diseases, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OPF-310Combination product
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.
| Study group | Role | What they receive |
|---|---|---|
| OPF-310 | Experimental | 13 patients will be transplanted OPF-310. Receives: Combination Product: OPF-310 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage 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.
Measured: One year after transplant
Secondary outcomes (12)
Percentage reduction in nocturnal hypoglycemic event rate
Change from baseline in nocturnal hypoglycemic event rate specified in the protocol
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 12 week, 24 week 52 weeks after transplant
Percentage of subjects with an HbA1c < 7.0 % assessed at 52 weeks post-transplant
Measured: One year after transplant
Percentage of subjects with an HbA1c ≤ 6.5 % assessed at 52 weeks post-transplant
Measured: One year after transplant
Percentage of subjects with positive porcine C-peptide qualitatively assessed by digital ELISA assay
Measured: 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
Measured: 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
Measured: 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.
Measured: For one year after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2024
- Study start10 Jun 2025
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A Phase I/IIa, Single site, Open-Label, Ascending Dose Study
- Masking
- None (open label)
- Enrolment
- 13 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Otsuka Pharmaceutical Factory, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06575426
- Sponsor's protocol ID
- 215-001
- Status last verified
- Aug 2026
- First submitted
- 20 Aug 2024
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes