Table 1.
Overview of selected clinical trials in islet transplantation
| Trial ID | Patient N | Investigators | Transplant type | Transplant site | Induction | Maintenance | Status | Primary endpoint | Percentage of participants that achieved the primary endpoint | Insulin independent rate (at any point throughout the trial) | Year of publicationRef |
|---|---|---|---|---|---|---|---|---|---|---|---|
| N/A | N = 7 | Alberta | ITA | Liver | Daclizumab | Tacrolimus, sirolimus | Completed | N/A | N/A | 7/7 (100%) | 200014 |
| NCT00014911 | N = 36 | Multicenter (Alberta etc.) | ITA | Liver | Daclizumab | Tacrolimus, sirolimus | Completed | Insulin independence with adequate glycemic control 1 y after the final transplantation | 44% | 21/36 (58%) | 200615 |
| NCT00285194 | N = 6 | Minnesota | ITA | Liver | Anti‐CD3 | Tacrolimus, sirolimus | Completed | Safety, tolerability, immune activity, and pharmacokinetics of hOKT3γ1 (Ala‐Ala) antibody induction therapy | N/A | 4/6 (67%) | 200425 |
| N/A | N = 8 | Minnesota | ITA | Liver | ATG, daclizumab, etanercept | Tacrolimus, sirolimus or MMF | Completed | Proportion of recipients who achieve insulin independence in the first year after a single‐donor islet transplant | 100% | 8/8 (100%) | 200516 |
| NCT00434811 | N = 48 | Multicenter (Minnesota etc.) | ITA | Liver | ATG, daclizumab, etanercept | Tacrolimus, sirolimus or MMF | Completed | Achievement of HbA1c <7.0% at day 365 and freedom from severe hypoglycemic events from day 28 to day 365 after the first transplant | 87.5% | 25/48 (52.1%) | 201618 |
| ACTRN083020 | N = 17 | Australia, multicenter | ITA | Liver | ATG, daclizumab, etanercept | Tacrolimus, sirolimus, MMF | Completed | HbA1c of <7% and cessation of severe hypoglycemia | 82% | 9/17 (53%) | 201320 |
| NCT01148680 | N = 25 vs 24 (RCT) | France, multicenter | ITA and IAK | Liver | ATG, daclizumab, etanercept | Tacrolimus, MMF | Completed | Proportion of patients with a modified β‐score (in which an overall score of 0 was not allocated when stimulated C‐peptide was negative) of 6 or higher at 6 mo after first islet infusion | 64% vs 0% (Immediate transplantation group vs 6 mo after randomization in the insulin group) | 11/25 (44%) | 201821 |
| UMIN000003977 | N = 20 | Japan, multicenter | ITA and IAK | Liver | ATG, daclizumab, etanercept | Tacrolimus or cyclosporine, MMF | Recruiting | Achievement of HbA1c <7.4% at day 365 and freedom from severe hypoglycemic events from day 28 to day 365 after the first transplant | Not yet | Not yet | Not yet |
| NCT00468117 | N = 24 | Multicenter (Minnesota etc.) | IAK | Liver | ATG, daclizumab, etanercept | Tacrolimus, sirolimus or MMF | Completed | Proportion of patients with HbA1c </= 6.5% and an absence of severe hypoglycemic events or a reduction in HbA1c of at least 1 point and an absence of severe hypoglycemic events | Not yet | Not yet | Not yet |
| N/A | N = 8 | San Francisco | ITA | Liver | ATG | Efalizumab, sirolimus or MMF | Completed | N/A | N/A | 8/8 (100%) | 201026 |
| NCT01722682 | N = 9 | Italy | ITA | Bone marrow vs liver | Unknown | Unknown | Completed | Insulin secretion under stimulation | Not yet | Not yet | Not yet |
| NCT02213003 | N = 6 | Miami | ITA | Omentum | ATG, etanercept | Tacrolimus, MMF | Recruiting | HbA1c </= 6.5% and no severe hypoglycemia | Not yet | Not yet | Not yet |
| NCT02064309 | N = 4 | Uppsala | ITA | Subcutaneous, macroencapsulation devices | No immunosuppression | No immunosuppression | Completed | Safety of device, as evaluated by incidence of adverse events or serious adverse events judged probable or highly probable related to the device | N/A | 0/4 (0%) | 201827 |
| NCT02239354 | N = 65 | Multicenter (Alberta etc.) | hESC‐derived pancreatic beta cells | Subcutaneous, macroencapsulation devices | No immunosuppression | No immunosuppression | Active, not recruiting |
1. Incidence of all adverse events 2. Change in C‐peptide |
Not yet | Not yet | Not yet |
| NCT01739829 | N = 8 | Argentina | Porcine islets | Microencapsulation, intraperitoneal | No immunosuppression | No immunosuppression | Completed | Reduction in the unaware hypoglycemic event rate combined with no increase in HbA1c | 4/4 (100%; High‐dose group) | 0/8 (0%) | 201628 |
ATG, antithymocyte globulin; HbA1c, hemoglobin A1c; hESC, human embryonic stem cell; IAK, islet after kidney; ITA, islet transplant alone; MMF, mycophenolate mofetil; N/A, not applicable.