A Futility Trial of Sirolimus in Multiple System Atrophy
Status:
Terminated
Trial end date:
2021-01-01
Target enrollment:
Participant gender:
Summary
Single-center, randomized, placebo-controlled, phase-II, futility clinical trial to determine
if oral sirolimus is of sufficient promise to slow disease progression in MSA, prior to
embarking on a large-scale and costly phase III study to assess its efficacy. A futility
design under the null hypothesis assumes that sirolimus will slow the progression of the
disease, whereas the alternative hypothesis assumes no benefit of sirolimus. If the null
hypothesis is rejected (i.e., futility of sirolimus to slow progression of MSA), a major
phase III study will be discouraged, whereas non-futility will offer strong support for a
phase III trial to detect clinical efficacy.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)