DISEASE CLASSIFICATION(S):
Amyotrophic Lateral Sclerosis(ALS)
NAME OF CLINICAL TRIAL/STUDY:
Minocycline - A Multicenter, Phase 3 Trial in ALS
TRIAL RESULTS:
December 2007
Paul Gordon, who coordinated this trial, spoke about the negative results at the 18th International Symposium on ALS/MND, held in Toronto Dec. 1-3, 2007.
He noted that the decline in function, as measured by the ALS Functional Rating Scale (Revised), or ALSFRS-R, was 25 percent faster in participants taking minocycline than it was in those taking a placebo (inert, look-alike substance). However, there was no difference in survival time in the minocycline versus the placebo groups.
The rate of ALSFRS decline wasn’t dependent on the dose of minocycline, or on whether or not people were also taking riluzole (Rilutek).
For details, see the article listed in Publications, below.
May 2007
Disappointing results of this trial of minocycline were announced May 1, 2007, at the American Academy of Neurology (AAN) annual meeting in Boston.
The trial, which had MDA funding, tested high-dose minocycline (400 milligrams per day) versus a placebo (inert substance) in 412 people with ALS at 31 U.S. centers. The drug failed to slow the decline in functional outcome measures and in some patients worsened measurable outcomes.
People who are taking minocycline should contact their physicians to discuss discontinuing it.
PURPOSE AND RATIONALE:
The purpose of this study is to evaluate the safety and effectiveness of minocycline treatment of amyotrophic lateral sclerosis (ALS). The trial enrolled people living with ALS at 29 sites across the United States.
Minocycline, an antibiotic in the tetracycline family, has anti-inflammatory and neuroprotective properties and is known to readily penetrate the central nervous system (brain and spinal cord). Minocycline has been tested and been shown to protect nerve cells in many scientific experiments. It reduces cell death and prolongs survival in animal models of ALS, stroke, trauma, Huntington’s disease, and Parkinson’s disease. It has been shown to be beneficial in multiple different animal experiments of ALS, conducted in Europe, Canada and the United States.
Minocycline has been tested in two preliminary human trials and has been shown to be safe in patients with ALS. It has been well tolerated in conjunction with riluzole (Rilutek), the only currently FDA approved medication for ALS.
STUDY DETAILS:
In this multicenter trial, participants were randomly chosen to be treated with the study drug or a placebo (inert substance). The trial investigators measured changes in function, using the ALS Functional Rating Scale. The scale includes measures of strength, pulmonary function, survival and quality of life.
Each participant underwent monthly outpatient evaluations and analysis of laboratory and adverse events for 13 months.
The coordinating center for this trial is the Eleanor and Lou Gehrig MDA/ALS Center, Columbia University Medical Center, in New York, Paul H. Gordon, M.D., principal investigator. For information about the trial contact Carolyn Doorish, Project Coordinator,(212) 305-2027, cd2141@columbia.edu.
OPENING DATES:
Nov 15 2003
CLOSING DATES:
Dec 31 2005
TARGET NUMBER OF PARTICIPANTS:
400
RECRUITMENT STATUS:
Closed
PUBLICATIONS:
Gordon PH, Moore DH, Miller RG, Florence JM, Verheijde JL, Doorish C, Hilton JF, Spitalny GM, Macarthur RB, Mitsumoto H, Neville HE, Boylan K, Mozaffar T, Belsh JM, Ravits J, Bedlack RS, Graves MC, McCluskey LF, Barohn RJ, Tandan R; for the Western ALS Study Group. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial. Lancet Neurol. 2007 Dec;6(12):1045-53. Epub 2007 Nov 5. PubMed summary
