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Abstract: FR-OR40

Safety and Preliminary Efficacy Results of a Novel Mesenchymal Stromal Cell Therapy in Diabetic Kidney Disease: The Multicenter, Randomized, Placebo-Controlled, Phase-1b/2a NEPHSTROM Clinical Trial

Session Information

Category: Diabetic Kidney Disease

  • 602 Diabetic Kidney Disease: Clinical


  • Griffin, Matthew D., Regenerative Medicine Institute (REMEDI), School of Medicine, NUI, Galway, Ireland
  • Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Bergamo, Italy
  • Cockwell, Paul, University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom
  • Maxwell, Alexander P., Queen's University Belfast Centre for Public Health, Belfast, Belfast, United Kingdom
  • Perico, Norberto, Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Bergamo, Italy
  • Ruggenenti, Piero Luigi, Aziende Socio Sanitarie Territoriale Papa Giovanni XXIII, Bergamo, Italy
  • Introna, Martino, Aziende Socio Sanitarie Territoriale Papa Giovanni XXIII, Bergamo, Lombardia, Italy
  • Finnerty, Andrew Anthony, National University of Ireland Galway (NUIG), Galway, Ireland
  • Smythe, Jon, National Head of Cellular and Molecular Therapies, Oxford, United Kingdom
  • Fibbe, Willem E., Leiden University Medical Center, Leiden, Netherlands
  • Elliman, Stephen Joseph, Orbsen Therapeutics Ltd, Galway, Ireland
  • O'Brien, Timothy, Regenerative Medicine Institute (REMEDI), School of Medicine, NUI, Galway, Ireland

Group or Team Name

  • The NEPHSTROM Trial Consortium

Mesenchymal stromal cells (MSC), by targeting individual or combined effects of renoprotective processes, are candidates for treatment of diabetic kidney disease (DKD).


NEPHSTROM is a randomized, placebo-controlled, double blind, dose-escalation phase 1b/2a clinical trial of next-generation bone marrow-derived, antibody-purified allo-CD362+ MSC (ORBCEL-M) in adults with type 2 diabetes, DKD and eGFR 25-55 ml/min/1.73 m2 with evidence of or risk of progressive eGFR decline. We report the experience with the first dose cohort, consisting of 16 subjects [single i.v. infusion of ORBCEL-M 80x106 cell (n=12, group A) or placebo (n=4, group B)], enrolled at 3 sites in Italy, Ireland and UK, and followed-up for 18 months.


All randomized patients were negative for anti-HLA antibodies at study entry. Mean baseline measured GFR (mGFR, iohexol clearance) and estimated GFR (eGFR, CKD-EPI equation) were comparable between the two groups. The trial intervention was well tolerated and safe (primary outcome), with one quickly-resolved infusion reaction (in the placebo group) and no subsequent SAEs ascribed to trial product. Two patients in group A died of cell product unrelated causes between 12 and 18 months. Serial serum assays for anti-HLA antibodies indicated low-level allo-immune sensitization in a patient from month 3. The median annual rate of renal function decline by mGFR (secondary outcome) was numerically lower in group A than group B; by eGFR this was statistically significant (Table). Blood pressure, glycemic and lipid profiles and spot morning urinary albumin creatinine ratio were comparable in the two groups during the 18 month follow-up.


In summary, for subjects enrolled into the first (low-dose) cohort of the NEPHSTROM trial, the safety and tolerability of ORBCEL-M was established. Over 18 months rate of decline of eGFR was less for recipients of cells compared to placebo.

 ORBCEL-MPlaceboP value*
mGFR (ml/min/1.73m2 per year)-3.8 [-5.7, -2.1]-7.5 [-13.9, +1.0]0.467
eGFR - CKD-EPI (ml/min/1.73m2 per year)-2.6 [-4.2, -0.3]-8.7 [-11.4, -4.6]0.034

Values are median [IQR]. *Wilcoxon rank sum ORBCEL-M vs Placebo