Phase 2 Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen in Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a rare, neurodegenerative disorder that results in
progressive wasting and paralysis of voluntary muscles. It is known that nerve cells called
motor neurons die in the brains and spinal cords of people with amyotrophic lateral
sclerosis (ALS). However, the cause of this cell death is unknown.
In this double blind, randomized, selection design trial, researchers will evaluate the
safety and effectiveness of creatine and tamoxifen in volunteers with ALS. There are a
large number of potential drugs that may improve the survival or slow down the disease
progression in people with ALS. The current strategy is to test one drug at a time against
placebo. "Selection Design" is a different type of study design. A Selection Design study
uses multiple drugs to screen against each other and picks the winner to take to a larger
study. This design can speed the search for effective drugs to treat ALS. In this
Selection Design study, each volunteer will take one active study drug (creatine 30gm,
tamoxifen 40mg, or tamoxifen 80mg) and one placebo.
Approximately 60 eligible volunteers with ALS will be recruited from multiple centers in the
US that belong to the Northeast ALS Consortium (NEALS). Volunteers will be randomly
assigned equally to the three treatment arms: creatine 30gm/day, tamoxifen 40mg/day and
tamoxifen 80mg/day. Volunteers will take study treatment for 38 weeks. After screening and
randomization, volunteers will be followed at weeks 4, 10, 18, 28 and week 38. A final
telephone interview will occur at week 42 (off study drug).
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Decline in ALSFRS-R
Primary efficacy will be assessed by analyzing the mean rate of decline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score over nine months.
38 weeks of treatment followed by a telephone interview at 42 weeks.
Nazem Atassi, MD, MMSc
Masaschusetts General Hospital, Boston, MA
United States: Food and Drug Administration
|University of Washington Medical Center||Seattle, Washington 98195-6043|
|Medical College of Wisconsin||Milwaukee, Wisconsin 53226|
|University of Kansas Medical Center||Kansas City, Kansas 66160-7353|
|Carolinas Medical Center||Charlotte, North Carolina 28232-2861|
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|
|SUNY Upstate Medical University||Syracuse, New York 13210|
|University of Massachusetts Medical Center||Worcester, Massachusetts 01605|
|Washington University at St. Louis||Saint Louis, Missouri 63110|
|Pennsylvania State University, Hershey Medical Center||Hershey, Pennsylvania 17033|