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

Primary Purpose

Type 1 Diabetes

Status
Completed
Phase
Phase 1
Locations
Italy
Study Type
Interventional
Intervention
Human pancreatic islet transplantation
Sponsored by
Ospedale San Raffaele
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Type 1 Diabetes focused on measuring Diabetes

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Ospedale San Raffaele

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

A: intraportal islet infusion

B: intra BM islet infusion

Arm Description

Patients will received islet into the liver through the portal venous circulation (standard procedure)

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.

Outcomes

Primary Outcome Measures

Insulin secretion under stimulation
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)
Insulin requirement
Change in average daily insulin requirements
Islet function
change in HbA1c, Transplant Estimated Function (TEF) and fasting C-peptide levels

Full Information

First Posted
October 31, 2012
Last Updated
November 2, 2020
Sponsor
Ospedale San Raffaele
Collaborators
Ministry of Health, Italy
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1. Study Identification

Unique Protocol Identification Number
NCT01722682
Brief Title
Bone Marrow vs Liver as Site for Islet Transplantation
Acronym
IsletBOM2
Official Title
Bone Marrow vs Liver as Site for Islet Transplantation in Patients With Type 1 Diabetes: Pilot Study to Evaluate Efficacy
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Completed
Study Start Date
June 2012 (undefined)
Primary Completion Date
September 2017 (Actual)
Study Completion Date
December 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Ospedale San Raffaele
Collaborators
Ministry of Health, Italy

4. Oversight

Data Monitoring Committee
Yes

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type 1 Diabetes
Keywords
Diabetes

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
A: intraportal islet infusion
Arm Type
Active Comparator
Arm Description
Patients will received islet into the liver through the portal venous circulation (standard procedure)
Arm Title
B: intra BM islet infusion
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Human pancreatic islet transplantation
Primary Outcome Measure Information:
Title
Insulin secretion under stimulation
Description
basal and -10 to 120 min time course of glucose, C-pep levels and insulin derived from the mixed meal tolerance test
Time Frame
month 12 post-Tx
Secondary Outcome Measure Information:
Title
Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE)
Time Frame
throughout the study up to 1 year after first transplant
Title
Insulin requirement
Description
Change in average daily insulin requirements
Time Frame
month 1, 3, 6, 9, 12 post- transplant
Title
Islet function
Description
change in HbA1c, Transplant Estimated Function (TEF) and fasting C-peptide levels
Time Frame
month 1, 3, 6, 9, 12 post-Tx

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lorenzo Piemonti, MD
Organizational Affiliation
Ospedale San Raffaele
Official's Role
Principal Investigator
Facility Information:
Facility Name
Ospedale San Raffaele
City
Milan
ZIP/Postal Code
20132
Country
Italy

12. IPD Sharing Statement

Citations:
PubMed Identifier
30130323
Citation
Maffi P, Nano R, Monti P, Melzi R, Sordi V, Mercalli A, Pellegrini S, Ponzoni M, Peccatori J, Messina C, Nocco A, Cardillo M, Scavini M, Magistretti P, Doglioni C, Ciceri F, Bloem SJ, Roep BO, Secchi A, Piemonti L. Islet Allotransplantation in the Bone Marrow of Patients With Type 1 Diabetes: A Pilot Randomized Trial. Transplantation. 2019 Apr;103(4):839-851. doi: 10.1097/TP.0000000000002416.
Results Reference
result
Links:
URL
http://www.hsr.it/ricerca/divisioni-centri-istituti-e-programmi-di-ricerca/islet-trasplantation-program-itp/
Description
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Bone Marrow vs Liver as Site for Islet Transplantation

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