Minimal Islet Transplant at Diabetes Onset

Summary

This is a prospective phase 2, single-arm, mono-center pilot study. It has been designed to investigate whether giving the combination therapy consisting of minimal islet transplantation (1500 EIQ/Kg body weight), Thymoglobulin® (ATG), Rapamune® (rapamycin) and Neulasta® (pegfilgastrim) to patients with Type 1 Diabetes (T1D) at onset is safe and secondarily, if it will preserve insulin production. It will involve 6 patients with new-onset T1D. Each patient will be involved in the study for a screening period and a post-islet transplantation study period of 52±2 weeks, to include 1 treatment cycles of 12 weeks, assessment during treatment and 5 follow-up visits scheduled at weeks 2±1 (14 days), 4±1 (month 1), 12±2 (month 3), 26±2 (month 6) and 52±2 (month 12).

Conditions

• Diabetes Mellitus, Type 1

Interventions

BIOLOGICAL

Human pancreatic islet

One thousand and five hundred (1,500) equivalent islet for Kg of body weight, isolated from a single brain-dead donor, will be infused into the patient's liver.

DRUG

ATG

ATG will be administered IV (central vein) at a total dose of 6 mg/kg up to day 6 post-transplant

DRUG

Pegylated G-CSF

Pegylated G-CSF (6 mg/dose) will be administered SC every 2 weeks for 6 doses (12 weeks) beginning after the last ATG infusion

DRUG

Rapamycin

Rapamycin will be administered orally at a starting dose of 0.2 mg/kg once a day, then targeted to blood trough level of 8-10 ng/mL and suspended one month after transplant.

Sponsors & Collaborators

• Italian Diabetes Foundation

  collaborator OTHER

• Ospedale San Raffaele

  lead OTHER

Principal Investigators

• Lorenzo Piemonti, MD · IRCCS Ospedale San Raffaele

• Emanuele Bosi, MD · IRCCS Ospedale San Raffaele

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

45 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2015-04-30

Primary Completion

2018-05-31

Completion

2023-12-31

Countries

• Italy

Study Locations