NCT01287221

Study of Rifampicin in Multiple System Atrophy

Double-Blind, Placebo-Controlled Study of Rifampicin in Multiple System Atrophy

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: Phillip Low · Academic / Other
• Sex: All
• Age: 30 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to determine whether Rifampicin was effective in slowing or reversing the progression of multiple system atrophy (MSA). Research studies indicate that there is an abnormality in protein synthesis and structure in parts of the brain responsible for MSA (protein misfolding) and the drug Rifampicin could potentially prevent or reverse this protein alteration. The study was done on participants with early MSA. The study consisted of taking the drug 2 times a day for 12 months. Participants underwent an evaluation of symptoms and function and will underwent a neurologic examination at the beginning of the study, at 6 months and at 12 months. They were also be contacted at 3 and 9 months by telephone. Studies were done at 10 participating sites.

Detailed description

MSA is a progressive, fatal disorder characterized by autonomic failure and parkinsonism and/or cerebellar involvement. Neuropathologically, MSA is characterized by glial cytoplasmic inclusions (GCI) of abnormally aggregated α-synuclein (α-syn). This was a study to test the hypothesis that Rifampicin, because of its ability to inhibit the formation of α-synuclein fibrils and disaggregate fibrils already formed, will delay progression or reverse neurologic and autonomic functions and symptoms in MSA. This approach has been proposed as a potential approach to treat parkinsonism and specifically, MSA. In an experimental model of MSA, it was hypothesized that Rifampicin would improve behavioral abnormalities of MSA and halt or reverse the pathological changes. The primary objective was to undertake a double-blind placebo-controlled clinical trial on the effect of Rifampicin on progression of neurological and autonomic failure in MSA. The Data Safety Monitoring Board (DSMB) recommended stopping the study after an interim analysis of the primary endpoint revealed that futility criteria were met.

Conditions

• Multiple System Atrophy

Interventions

Type	Name	Description
DRUG	Rifampicin	300 mg, 2 times daily
DRUG	placebo	placebo

Timeline

• Start date: 2011-03-01
• Primary completion: 2012-11-01
• Completion: 2013-01-01
• First posted: 2011-02-01
• Last updated: 2014-03-28
• Results posted: 2014-03-28

Locations

10 sites across 1 country: United States