Summary: Investigators across the U.S. and in other countries are recruiting 190 children and adolescents with MS (ages 10-17) for a clinical trial comparing the effectiveness of oral fingolimod (Gilenya®, Novartis Pharmaceuticals AG) with interferon beta-1a (Avonex®, Biogen Idec) injected into the muscle. The study is funded by Novartis. The U.S. arm of the study has been endorsed by the Network of Pediatric MS Centers, which was inaugurated and is supported by the National MS Society.
Rationale: Although MS occurs most commonly in adults, it is also diagnosed in children and adolescents. However, clinical trials have not formally evaluated the safety or effectiveness of these therapies in children with MS.
Gilenya was approved by the U.S. Food and Drug Administration in 2010 for adults with relapsing forms of MS for its ability to reduce the frequency of clinical relapses and to delay the accumulation of physical disability. Gilenya is a new class of medication called a sphingosine 1-phosphate receptor modulator, which is thought to act by retaining certain white blood cells in the lymph nodes, thereby preventing those cells from crossing the blood-brain barrier into the central nervous system. Preventing the entry of these cells reduces inflammatory damage to nerve cells.
This study is evaluating the safety and effectiveness of Gilenya in children/adolescents with MS. The study will compare Gilenya to another therapy, Avonex, so that all participants will be on active therapy during the trial.
Eligibility and Details: Participants are ages 10 to 17, with a diagnosis of MS, who have had at least one MS relapse during the previous year or two MS relapses in the previous two years. Participants with significant heart abnormalities upon testing, or severe kidney dysfunction, are excluded. Further enrollment criteria are available from the contact below.
Participants are being randomly assigned to receive either Gilenya (administered orally daily at a dose of either 0.5 mg or 0.25 mg, depending on body weight) and a placebo injection once weekly intramuscularly; or Avonex injected once weekly intramuscularly, and daily oral placebo. Participants are being treated for 24 months, and receive extensive safety evaluations prior to drug initiation and subsequently during the trial.
The primary outcome being measured is the frequency of relapses. Secondary outcomes include disease activity observed on MRI scans, safety, and how the drug is absorbed by the body.
Contact: To learn more about the enrollment criteria for this study, and to find out if you are eligible to participate, please contact Shazia Ahmad at UBC via phone at 703-663-2940 or e-mail PARADIGMS@ubc.com.
Sites will be enrolling in the following cities; please check with Ms. Ahmad to find out whether the site nearest you is recruiting participants yet:
Buffalo, New York
Chapel Hill, North Carolina
Durham, North Carolina
Fort Worth, Texas
Greenville, South Carolina
Los Angeles, California
North Haven, Connecticut
Rochester, New York
Salt Lake City, Utah
San Antonio, Texas
San Francisco, California
Sioux Falls, South Dakota
Stony Brook, New York
Syracuse, New York
Washington, District of Columbia
Download a brochure that discusses issues to think about when considering enrolling in an MS clinical trial (PDF).