Phase III Trial of Minocycline

A NINDS-supported Phase III 13-month clinical trial of minocycline began enrolling people living with ALS in November 2003. This multicenter, randomized, double blind trial will compare minocycline to placebo for efficacy, tolerability and safety. Researchers hope to demonstrate that minocycline slows disease progression in people living with ALS.

At study completion, the minocycline trial will be one of the largest investigator-initiated clinical trials conducted to date in ALS; it will provide data not only on the effectiveness of the treatment, but also on the course of ALS.

The Phase III trial will enroll 400 participants nationwide.

Each participant will undergo monthly outpatient evaluations for 13 months measuring any changes in functions, such as strength, pulmonary function, survival and quality of life.

Scientific Background:

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.

Preliminary Human Trials:

Minocycline has been tested in 2 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.

Inclusion Criteria:

• A clinical diagnosis of ALS
• 21 to 85 years of age, male or female
• FVC (forced vital capacity, a respiratory test) greater or equal to 75 percent of predicted
• Onset of weakness within three years prior to enrollment
• If on riluzole, on a stable dose for at least the past 30 days
• If a woman of childbearing age, not breast-feeding and surgically sterile or using an effective method of birth control; negative pregnancy test (note: adequate birth control includes use of intrauterine device or oral contraceptives plus a barrier method, such as a condom or diaphragm)
• Willing and able to give signed informed consent that has been approved by the local Institutional Review Board (IRB)

Exclusion Criteria:

• Allergy to tetracycline antibiotics
• Use of minocycline within 30 days of baseline visit
• Requirement for Tracheostomy ventilation (or noninvasive ventilation more than 23 hours per day)
• Diagnosis of other neurodegenerative diseases (Parkinson's disease, Alzheimer's disease, etc.)
• Forced vital capacity (FVC) less than 75 percent of predicted
• Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc.) over the last 30 days
• History of renal (kidney) disease
• History of liver disease
• History of hematologic disease
• History of systemic lupus erythematosus
• Using procainamide or hydralazine
• History of vestibular disease (excluding benign position vertigo)
• Pregnancy or breast-feeding
• Use of antiepileptic medications other than gabapentin
• Limited mental capacity rendering the subject unable to provide written informed consent or comply with evaluation procedures
• History of recent alcohol or drug abuse or nonadherence with treatment or other experimental protocols
• Use of any investigational drug within the past 30 days or drugs such as creatine, Vioxx, Celebrex, topiramate, or CoQ10.
• Familial ALS
• Potential to become pregnant; not practicing effective birth control

Main Sponsoring Location/Contact:

Paul H. Gordon, MD, Principal Investigator
Associate Medical Director
Eleanor and Lou Gehrig MDA /ALS Center
Neurological Institute
Columbia University Medical Center
710 West 168th Street
New York, NY 10032

For information about the trial, contact:

Carolyn Doorish, Project Coordinator
Eleanor and Lou Gehrig MDA /ALS Research Center
Columbia University Medical Center
Department of Neurology
710 West 168th Street, 9th Floor
New York, NY 10032
Phone: (212) 305-2027
Email: cd2141@columbia.edu

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