IsletBOM2: Bone Marrow vs Liver as Site for Islet Transplantation
Study Details
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 |
|---|---|---|
|
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
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
- Insulin secretion under stimulation [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
- Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE) [throughout the study up to 1 year after first transplant]
- Insulin requirement [month 1, 3, 6, 9, 12 post- transplant]
Change in average daily insulin requirements
- Islet function [month 1, 3, 6, 9, 12 post-Tx]
change in HbA1c, Transplant Estimated Function (TEF) and fasting C-peptide levels
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Ospedale San Raffaele | Milan | Italy | 20132 |
Sponsors and Collaborators
- Ospedale San Raffaele
- Ministry of Health, Italy
Investigators
- Principal Investigator: Lorenzo Piemonti, MD, Ospedale San Raffaele
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- IsletBOM2