Bone Marrow vs Liver as Site for Islet Transplantation (IsletBOM2)

This study is ongoing, but not recruiting participants.
Ministry of Health, Italy
Information provided by (Responsible Party):
Piemonti Lorenzo, Ospedale San Raffaele Identifier:
First received: October 31, 2012
Last updated: July 21, 2015
Last verified: July 2015
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.

Condition Intervention Phase
Type 1 Diabetes
Biological: Human pancreatic islet transplantation
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Bone Marrow vs Liver as Site for Islet Transplantation in Patients With Type 1 Diabetes: Pilot Study to Evaluate Efficacy

Resource links provided by NLM:

Further study details as provided by Ospedale San Raffaele:

Primary Outcome Measures:
  • Insulin secretion under stimulation [ Time Frame: month 12 post-Tx ] [ Designated as safety issue: No ]
    basal and -10 to 120 min time course of glucose, C-pep levels and insulin derived from the mixed meal tolerance test

Secondary Outcome Measures:
  • Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE) [ Time Frame: throughout the study up to 1 year after first transplant ] [ Designated as safety issue: Yes ]
  • Insulin requirement [ Time Frame: month 1, 3, 6, 9, 12 post- transplant ] [ Designated as safety issue: No ]
    Change in average daily insulin requirements

  • Islet function [ Time Frame: month 1, 3, 6, 9, 12 post-Tx ] [ Designated as safety issue: No ]
    change in HbA1c, Transplant Estimated Function (TEF) and fasting C-peptide levels

Estimated Enrollment: 12
Study Start Date: June 2012
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: A: intraportal islet infusion
Patients will received islet into the liver through the portal venous circulation (standard procedure)
Biological: Human pancreatic islet transplantation
Experimental: B: intra BM islet infusion
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.
Biological: Human pancreatic islet transplantation

Detailed 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.

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT01722682

Ospedale San Raffaele
Milan, Italy, 20132
Sponsors and Collaborators
Ospedale San Raffaele
Ministry of Health, Italy
Principal Investigator: Lorenzo Piemonti, MD Ospedale San Raffaele
  More Information

Additional Information:
Responsible Party: Piemonti Lorenzo, Head of Beta Cell Biology Unit, CO-Director Human Islet Transplantation Program (ITP), Ospedale San Raffaele Identifier: NCT01722682     History of Changes
Other Study ID Numbers: IsletBOM2 
Study First Received: October 31, 2012
Last Updated: July 21, 2015
Health Authority: Italy: Centro Nazionale Trapianti (CNT)

Keywords provided by Ospedale San Raffaele:

Additional relevant MeSH terms:
Diabetes Mellitus, Type 1
Autoimmune Diseases
Diabetes Mellitus
Endocrine System Diseases
Glucose Metabolism Disorders
Immune System Diseases
Metabolic Diseases processed this record on May 26, 2016