To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS)
The study is divided into a Core Phase, which includes the Double-Blind Treatment Period, and an Extension Phase in which all patients will be treated with fingolimod. The Core Phase is a 24-month, double-blind, randomized, active-controlled, parallel-group multicenter study phase to evaluate the efficacy and safety of fingolimod compared to IFN β-1a in children/adolescent patients aged 10-17 years old with MS. The Extension Phase is a 60-month (5 year) study phase for patients who complete the Core Phase of the study and meet all inclusion/exclusion criteria and for patients who will be recruited in the younger cohort to participate in the Extension Phase. The 'younger cohort' refers to the population of pediatric patients fulfilling any single one or a combination of the following criteria: being ≤12 years of age, or weighing ≤40 kg, or being prepubertal (i.e. pubertal status of Tanner stage \<2). The recruitment of the younger cohort (up to 25 patients) was requested as a post- approval health authority commitment
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
240
Administration once weekly via i.m. injections.
Administrated orally once daily: 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
Matching placebo capsule required for double-dummy masking to blind formulations.
Frequency of Relapses in Patients Treated for up to 24 Months
Frequency of relapses assessed by the annualized relapse rate (ARR). The ARR is defined as the average number of confirmed relapses per year (total number of confirmed relapses divided by the total days in the study multiplied by 365.25).
Time frame: 24 months
New/Newly Enlarged T2 Lesions
Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24
Time frame: 24 months
Time to First Relapse
Time to first relapse was determined.
Time frame: 24 months
Proportion of Patients Relapse-free
Proportion of patients relapse-free was determined
Time frame: 24 months
T1 Gd- Enhancing Lesions
Number of T1 Gd-enhancing lesions per scan up to Month 24
Time frame: 24 months
Pharmacokinetics (Cavg) of Fingolimod-P
Cavg (average drug concentration over the dose interval) will be evaluated.
Time frame: 24 months
Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels
Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.
Time frame: 24 months
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Matching placebo i.m. injection required for double-dummy masking to blind formulations.
UAB Childrens Hospital Harbor Center Neurology Dept
Birmingham, Alabama, United States
Childrens Hospital Los Angeles
Los Angeles, California, United States
UCSF
San Francisco, California, United States
University of Miami
Miami, Florida, United States
AMO Corporation
Tallahassee, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Wayne State University
Detroit, Michigan, United States
Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Utah Clinical Trials Office
Salt Lake City, Utah, United States
...and 63 more locations