A Futility Trial of Sirolimus in Multiple System Atrophy

Multiple System Atrophy Clinical Trial

Official title: A Single Center Randomized,Double Blind, Placebo-controlled Futility Trial to Determine if Sirolimus is of Sufficient Promise to Slow the Progression of Multiple System Atrophy

Status Terminated

Clinical Trial 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Atrophy
• Multiple System Atrophy
• Shy-Drager Syndrome

• NCT number: NCT03589976
• Study type: Interventional
• Source: NYU Langone Health
• Status: Terminated
• Phase: Phase 2
• Start date: September 1, 2018
• Completion date: January 1, 2021