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A Futility Trial of Sirolimus in Multiple System Atrophy

NCT03589976 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 47

Last updated 2021-12-07

Study results available
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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

Interventions

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.

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • NYU Langone Health

    lead OTHER

Principal Investigators

  • Jose-Alberto Palma, MD, PhD · NYU Langone Health

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-09-01
Primary Completion
2020-11-20
Completion
2021-01-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03589976 on ClinicalTrials.gov