An Oligo-centered, Randomized, Double-blind, Placebo-controlled Clinical Trial to Assess the Efficacy, Safety, and Tolerability of Rifampicin in Patients with Multiple System Atrophy
This is a double-blind placebo-controlled randomized outpatient study comparing Rifampicin versus placebo on indices of neurologic function and autonomic function in participants with MSA. One hundred participants with MSA who will be randomly assigned to receive either placebo or 300 mg of Rifampicin two times a day for 12 months. The primary goal is to evaluate the effect of Rifampicin versus placebo on progression of neurologic disorders, including autonomic, cerebellar and parkinsonian features. This study grew out of the MSA PPG (NS44233; Principal Investigator: Dr. Phillip Low) and is being funded by NINDS (NS44233; U54 NS065736). The MSA PPG will provide the necessary infrastructure contained in Projects 1 (Dr. Sid Gilman) and 4 (Dr. Low) coordinated by the Administrative and Clinical Core to carry out the study. The MSA PPG will be responsible for studying 60 participants and the Consortium for 40 participants but the proportion could change depending on success of recruitment among the sites. This randomized, double-blind, placebo-controlled, oligo-center study will be carried out in five sites, with the option of adding five additional sites. The option of adding additional sites if recruitment in the first 15 months is slow. The aim is to complete recruitment within 2 years from commencement of the study.
Eligible participants, who signed the approved informed consent form at the screening visit, will be randomized in a 1:1 ratio to receive either Rifampicin 300 mg two times a day or matching placebo for 12 months. Capsules will be supplied by a central pharmacy (Mark F. Binkley, D.Ph.; Compounding Pharmacist; Health and Wellness Compounding Pharmacy, Nashville, TN 37203). The placebo and test drug will be identical in appearance.
Participants must meet all inclusion criteria in order to be eligible for the study:
1. Participants aged 30-80 years old with a diagnosis of Possible or Probable MSA of the parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) according to The Gilman Criteria (2008).
2. Participants who are less than 4 years from the time of documented MSA diagnosis.
3. Participants with an anticipated survival of at least 3 years in the opinion of the investigator.
4. Participants who are willing and able to give informed consent.
5. “Normal” cognition as assessed by MMSE. We will require a value >24