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

San Donato Group (GSD)

Status and phase: Completed; Phase 2; Phase 1

Conditions: Type 1 Diabetes

Treatments: Biological: Human pancreatic islet transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT01722682

IsletBOM2

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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9 participants in 2 patient groups

A: intraportal islet infusion

Active Comparator group

Description:

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

Treatment:

Biological: Human pancreatic islet transplantation

B: intra BM islet infusion

Experimental group

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.

Treatment:

Biological: Human pancreatic islet transplantation