NCT03589976

A Futility Trial of Sirolimus in Multiple System Atrophy

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: NYU Langone Health · Academic / Other
• Sex: All
• Age: 30 Years – 80 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Multiple System Atrophy

Interventions

Type	Name	Description
DRUG	Sirolimus 2 MG	Dose will be adjusted throughout this trial based on sirolimus plasma levels and the presence of drug-related adverse events. The maximum dose will be 6mg/day.
OTHER	Placebo	Patients receiving placebo will undergo analog sham level measurements and the number of tablets will be also adjusted to maintain the blinding of the trial.

Timeline

• Start date: 2018-09-01
• Primary completion: 2020-11-20
• Completion: 2021-01-01
• First posted: 2018-07-18
• Last updated: 2021-12-07
• Results posted: 2021-12-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study