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Phase 3CompletedNCT01897688

Single-center phase 3 islet transplantation in non-uremic T1D

Northwestern phase 3 donor-islet transplant study for non-uremic people with difficult T1D control. Now completed, with results posted in June 2026 — but of 10 people enrolled, only 2 were actually transplanted, so it is a very small transplant experience rather than a 10-patient efficacy readout.

Primary endpoints

  • To demonstrate the safety and efficacy of islet transplantation under alemtuzumab induction for treatment of Type-1 Diabetes (T1D) in subjects with hypoglycemia unawareness and a history of severe hypoglycemic episodes.

Results so far

Completed February 2025 (primary completion July 2021); results were first posted on ClinicalTrials.gov in June 2026. The headline number is small: 105 people were screened and 10 enrolled, but only 2 actually received islets — the other 8 withdrew before any transplant. The posted primary outcome (participants free of severe hypoglycaemic events two years after their final islet infusion) was met by 2 of the 2 transplanted participants. No deaths and no serious adverse events were reported among the 10 enrolled over 24 months. Read this as a very small transplant experience, not a 10-patient efficacy readout.

The full picture

What was tested

Islet transplantation takes the insulin-producing islet cells from a deceased donor's pancreas and infuses them into the recipient's liver, where they can start sensing glucose and releasing insulin again. This Northwestern University study was a single-centre phase 3 trial in adults (18-65) with type 1 diabetes who had hypoglycaemia unawareness and a history of severe lows, and who did not have kidney failure ("non-uremic") — so the transplant was being asked to justify itself on hypoglycaemia grounds alone, not as an add-on to a kidney transplant. Immune induction used alemtuzumab (Campath).1

What happened

The trial ran from June 2012, reached primary completion in July 2021, and completed in February 2025. Results were first posted to ClinicalTrials.gov in June 2026.2

The most important thing in the posted results is the participant flow. 105 people were screened and 10 were enrolled — but only 2 actually received an islet infusion. The other 8 withdrew without ever being transplanted. Getting a matched donor pancreas, staying eligible while waiting, and accepting lifelong immunosuppression are all hard, and this trial shows how much attrition that creates.2

The posted primary outcome — being free of severe hypoglycaemic events two years after the final islet transplant — was met by 2 of the 2 transplanted participants. Across all 10 enrolled participants, no deaths and no serious adverse events were reported over 24 months.2

How to read this

A denominator of two is not an efficacy result. It is consistent with the broader donor-islet literature — where transplantation reliably abolishes severe hypoglycaemia in the people who get it — but this trial on its own cannot tell you how well islet transplants work. What it does illustrate clearly is the bottleneck the field is trying to design around: donor supply and the burden of immunosuppression are what keep islet transplantation from scaling, not the biology of the transplanted cells. That is precisely the problem stem-cell-derived islets and encapsulation or hypoimmune approaches are built to solve.

References

  1. Northwestern University. A Phase 3 Single Center Study of Islet Transplantation in Non-uremic Diabetic Patients — protocol, eligibility and induction regimen. ClinicalTrials.gov, NCT01897688 (accessed 2026-07-14). https://clinicaltrials.gov/study/NCT01897688 ↩

  2. Northwestern University. NCT01897688 — posted results (participant flow, primary outcome measure, adverse events); study completed 2025-02, results first posted 2026-06-18. ClinicalTrials.gov (accessed 2026-07-14). https://clinicaltrials.gov/study/NCT01897688 ↩ ↩2 ↩3