Failure to Preserve β-Cell Function With Mycophenolate Mofetil and Daclizumab Combined Therapy in Patients With New- Onset Type 1 Diabetes

Abstract Objective: This trial was to test whether mycophenolate mofetil (MMF) alone or with daclizumab (DZB) could arrest the loss of insulin-producing beta cells in subjects with new onset type 1 diabetes (T1D). Research Design and Methods: A multicenter, randomized, placebo-controlled, double-masked trial was initiated by Type 1 Diabetes TrialNet at 13 sites in North America and Europe. Subjects diagnosed with type 1 diabetes and with sufficient C-peptide within 3 months of diagnosis were randomized to either: MMF-alone, MMF and DZB, or placebo, and then followed for 2 years. The primary outcome was the geometric mean area under the curve (AUC) C-peptide from the 2 hour mixed meal tolerance test. Results: One hundred and twenty-six subjects were randomized and treated during the trial. The geometric mean C-peptide AUC at 2 years was unaffected by MMF-alone, or MMF and DZB, versus placebo. Adverse events were more frequent in the active therapy groups relative to the control group, but not significantly. Conclusions: Neither MMF-alone nor MMF in combination with DZB had an effect on the loss of C-peptide in subjects with new onset T1D. Higher doses or more targeted immunotherapies may be needed to affect the autoimmune process.