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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01722682
Recruitment Status : Active, not recruiting
First Posted : November 7, 2012
Last Update Posted : February 14, 2017
Information provided by (Responsible Party):

Study Description
Brief 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.

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Biological: Human pancreatic islet transplantation Phase 1 Phase 2

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Study Start Date : June 2012
Estimated Primary Completion Date : September 2017
Estimated Study Completion Date : December 2017

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U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :
  1. Insulin secretion under stimulation [ Time Frame: month 12 post-Tx ]
    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 :
  1. Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE) [ Time Frame: throughout the study up to 1 year after first transplant ]
  2. Insulin requirement [ Time Frame: month 1, 3, 6, 9, 12 post- transplant ]
    Change in average daily insulin requirements

  3. Islet function [ Time Frame: month 1, 3, 6, 9, 12 post-Tx ]
    change in HbA1c, Transplant Estimated Function (TEF) and fasting C-peptide levels

Eligibility Criteria

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
ClinicalTrials.gov Identifier: NCT01722682     History of Changes
Other Study ID Numbers: IsletBOM2
First Posted: November 7, 2012    Key Record Dates
Last Update Posted: February 14, 2017
Last Verified: February 2017

Keywords provided by Piemonti Lorenzo, Ospedale San Raffaele:

Additional relevant MeSH terms:
Diabetes Mellitus, Type 1
Diabetes Mellitus
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases