A Study to Investigate Safety and Effectiveness of Porcine Pancreatic Cells (OPF-310) in Patients With Type 1 Diabetes Mellitus
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 35 Years to 70 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Mentioned in the study description
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Mentioned in the study description
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This first study in humans tests OPF-310, pig pancreas cells placed inside a protective covering and transplanted into the body. It will check safety, how well the treatment works, and the dose for the next study. It is for adults ages 35–70 with hard-to-control type 1 diabetes and serious low blood sugar problems despite using an automated insulin pump system.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- You are 35–70 years old and have had type 1 diabetes for at least 5 years.
- Your diabetes is still hard to control after at least 6 months using one of the listed glucose sensor and automated insulin pump systems, with care from a diabetes team.
- You meet one low blood sugar rule: at least 3 severe episodes needing another person's help in the past year; at least 1 such episode plus a Clarke score of 4 or more; or blood sugar below 70 mg/dL more than 1% of the time plus a Clarke score of 4 or more. This score checks how well you notice low blood sugar.
- Your C-peptide test, which shows how much insulin your body makes, is below 0.3 ng/mL after a test meal or cannot be detected after fasting.
- Your A1C, a measure of average blood sugar, is 9.0 or lower.
- You follow local birth control rules for people in clinical studies.
- You agree to lifelong follow-up after the transplant.
- You can understand and sign the study consent form.
Who can't join
- You need an average insulin dose of at least 0.8 units/kg/day during the week before joining; have LADA, Flatbush diabetes, or MODY rather than type 1 diabetes; or have a body mass index (BMI) above 27.
- You have certain abnormal blood tests, unstable thyroid disease, HIV, hepatitis B or C, or cancer within the past 5 years other than basal or squamous cell skin cancer.
- You have active or untreated advanced diabetic eye disease, or certain serious heart, blood vessel, brain blood vessel, lung, or kidney problems.
- You have certain blood-forming cell disorders, adrenal glands that do not make enough hormones and need steroid treatment, or a past infection throughout the belly lining.
- You have had a donor organ or tissue transplant, certain belly surgeries, or a blood transfusion or blood donation in the past 90 days. Planned surgery that may need a transfusion during the study also rules you out.
- You take blood thinners other than aspirin, drugs that weaken the immune system, diabetes drugs other than insulin, acetaminophen, or hydroxycarbamide. Recent use of another study treatment also rules you out.
- You are allergic to insulin, pork products, alginate or seaweed, or any of these antibiotics used to make OPF-310: amphotericin B, cefazolin, ciprofloxacin, or gentamicin.
- You have an active drug or alcohol addiction, or another condition that the study doctor thinks could keep you from following study visits and treatment.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Subject must be aged 35 to 70 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. 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 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. 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 sites (1)
IL
- University of Illinois Hospital & Health Sciences System · Chicago, IL 60612Benito Valdepenas · Study contact · 312-355-3113 · bvalde2@uic.eduMore trials in Chicago