Overview

This trial is active, not recruiting.

Condition type 1 diabetes
Treatment human pancreatic islet transplantation
Phase phase 1/phase 2
Sponsor Ospedale San Raffaele
Collaborator Ministry of Health, Italy
Start date June 2012
End date November 2016
Trial size 12 participants
Trial identifier NCT01722682, IsletBOM2

Summary

The goal of this study is to evaluate safety and efficacy of bone marrow (BM) as site for pancreatic islet transplantationin humans. Our hypothesis is that BM represents a better site than liver (currently the location of choice for this procedure) thanks to its potential capacity to favor islet engraftment. To address our hypothesis we propose herein a randomized phase II trial to compare BM and liver as sites for islet transplantation in T1D patients.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Active Comparator)
Patients will received islet into the liver through the portal venous circulation (standard procedure)
human pancreatic islet transplantation
(Experimental)
Patients will received an intra BM islet infusion at the level of the iliac crest. The direct intra BM administration will be performed following the same procedures that our institution utilizes for administration of cord-blood cells in patients with acute leukemia (Lancet Oncol. 2008;9:831). The procedure is easy and reproducible: a standard needle for BM aspiration is inserted in the iliac crest and cells are gently infused.
human pancreatic islet transplantation

Primary Outcomes

Measure
Insulin secretion under stimulation
time frame: month 12 post-Tx

Secondary Outcomes

Measure
Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE)
time frame: throughout the study up to 1 year after first transplant
Insulin requirement
time frame: month 1, 3, 6, 9, 12 post- transplant
Islet function
time frame: month 1, 3, 6, 9, 12 post-Tx

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - diabetic patients eligible for pancreatic islet transplantation based on local accepted practice and guidelines. This includes at least: a)clinical history compatible with T1D with insulin-dependence for >5 years; b) undetectable stimulated (arginine or MMTT) C-peptide levels (<0.3 ng/mL) in the 12 months before transplant c)presence of severe hypoglycaemic events Exclusion Criteria: - presence of hematologic disease

Additional Information

Official title Bone Marrow vs Liver as Site for Islet Transplantation in Patients With Type 1 Diabetes: Pilot Study to Evaluate Efficacy
Principal investigator Lorenzo Piemonti, MD
Description The study is a phase II, single center, open label, pilot study. We will recruit 12 patients with T1D to be randomly (1:1) assigned to receive islet either into the liver through the portal venous circulation (standard procedure; arm A, n=6) or directly into the BM at the level of the iliac crest (arm B, n=6). Patients will be selected from those eligible for islet Tx based on local practice and guidelines.
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Ospedale San Raffaele.