NCT06575426

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_1

Timeline
14mo left

Started Jun 2025

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress44%
Jun 2025Jun 2027

First Submitted

Initial submission to the registry

August 20, 2024

Completed
8 days until next milestone

First Posted

Study publicly available on registry

August 28, 2024

Completed
10 months until next milestone

Study Start

First participant enrolled

June 10, 2025

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2027

Last Updated

March 2, 2026

Status Verified

February 1, 2026

Enrollment Period

2.1 years

First QC Date

August 20, 2024

Last Update Submit

February 26, 2026

Conditions

Diabetes Mellitus, Type 1HypoglycemiaIslet Cell TransplantationType 1 DiabetesType 1 Diabetes MellitusT1DT1DMT1DM - Type 1 Diabetes MellitusType 1 Diabetes (T1D)Severe HypoglycemiaXenotransplantationHypoglycemic EpisodeIslet Transplantation in Diabetes Mellitus Type 1Glucose Metabolism Disorders (Including Diabetes Mellitus)Immune System DiseasesAutoimmune DiseasesMetabolic Disease

Keywords

Diabetes MellitusDiabetes Mellitus, Type1T1DMHypoglycemiaislet cell transplantationpig islet cell transplantationXenotransplantationPorcine islet cell transplantationType 1 Diabetes MellitusType 1 Diabetes

Outcome Measures

Primary Outcomes (1)

  • 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.

    One year after transplant

Secondary Outcomes (12)

  • Percentage reduction in nocturnal hypoglycemic event rate

    12 week, 24 week 52 weeks after transplant

  • Percentage improvement in time below range (<70 mg/dl) by continuous glucose monitoring (CGM)

    12 week, 24 week 52 weeks after transplant

  • Percentage improvement in time in range (70-180 mg/dl) by CGM

    12 week, 24 week 52 weeks after transplant

  • Percentage improvement in time above range (>180 mg/dl) by CGM

    12 week, 24 week 52 weeks after transplant

  • Change in mean amplitude of glycemic excursion (MAGE) by CGM.

    12 week, 24 week 52 weeks after transplant

  • +7 more secondary outcomes

Study Arms (1)

OPF-310

EXPERIMENTAL

13 patients will be transplanted OPF-310.

Combination Product: OPF-310

Interventions

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.

OPF-310

Eligibility Criteria

Age35 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subject must be aged 35 to 65 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:
  • 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

You may not qualify if:

  • 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. In addition, patients with transiently abnormal TSH levels may undergo rescreening only once during the screening period.
  • 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.
  • 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
  • +19 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Illinois Hospital & Health Sciences System

Chicago, Illinois, 60612, United States

RECRUITING

MeSH Terms

Conditions

Diabetes Mellitus, Type 1HypoglycemiaGlucose Metabolism DisordersImmune System DiseasesAutoimmune DiseasesMetabolic DiseasesDiabetes Mellitus

Condition Hierarchy (Ancestors)

Nutritional and Metabolic DiseasesEndocrine System Diseases

Central Study Contacts

For participant-focused inquiries, please contact:

CONTACT

847-500-9204opf-310_autoreply@opf-america.com

For physician or professional inquiries, please contact:

CONTACT

847-200-6731opf-310_project_team@opf-america.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: A Phase I/IIa, Single site, Open-Label, Ascending Dose Study
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 20, 2024

First Posted

August 28, 2024

Study Start

June 10, 2025

Primary Completion (Estimated)

June 30, 2027

Study Completion (Estimated)

June 30, 2027

Last Updated

March 2, 2026

Record last verified: 2026-02

Locations

US(1)