Health TrialsbyHealthPanel

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

ConditionsDiabetes 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
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age35-70
SponsorOtsuka Pharmaceutical Factory, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • OPF-310

    Combination 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.

Treatment groups

13 Participants
are divided into 1 treatment group
Group A: OPF-310Experimental treatment 1 intervention

Trial outcomes

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.

Time frame
One year after transplant

Secondary outcomes

1

Percentage reduction in nocturnal hypoglycemic event rate

Change from baseline in nocturnal hypoglycemic event rate specified in the protocol

Time frame
12 week, 24 week 52 weeks after transplant
2

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

Time frame
12 week, 24 week 52 weeks after transplant
3

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

Change from baseline in the CGM data specified in the protocol

Time frame
12 week, 24 week 52 weeks after transplant
4

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

Change from baseline in the CGM data specified in the protocol

Time frame
12 week, 24 week 52 weeks after transplant

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Otsuka Pharmaceutical Factory, Inc.

Lead sponsor

Source ClinicalTrials.gov
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