Type 1 Diabetes
Conditions
Keywords
Diabetes
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.
Interventions
BIOLOGICALHuman pancreatic islet transplantation
Sponsors
Ministry of Health, Italy
Ospedale San Raffaele
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Italy
Outcome results
None listed