Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis
Intervention: Interferon beta-1a; Fingolimod; Placebo capsule; Placebo i.m. injection
Intervention description: 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.; Matching placebo i.m. injection required for double-dummy masking to blind formulations.
Intervention MeSH terms: Interferons; Interferon-beta; Interferon beta-1a; Fingolimod Hydrochloride
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: There are 2 arms in the core phase. In the extension phase all participants are receiving open-label.study drug. A third arm has been added to enroll up to 25 new younger patients per HA post approval commitment.
Masking: Double (Participant, Investigator)
Enrollment: 220.0
Sex: All
Minimum age: 10 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: pediatric; multiple sclerosis; fingolimod; pre-pubertal; low weight; children; adolescent; MS; core phase; extension phase; younger cohort

Summary:

      To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric
      patients with multiple sclerosis (MS)
    

Eligibility criteria:

        Key Inclusion Criteria Core Phase:

          -  diagnosis of multiple sclerosis

          -  at least one MS relapse during the previous year or two MS relapses in the previous 2
             years or evidence of Gd enhancing lesions on MRI within 6 months EDSS score of 0 to
             5.5, inclusive

        Key Exclusion Criteria Core Phase:

          -  patients with progressive MS

          -  patients with an active, chronic disease of the immune system other than MS

          -  patients meeting the definition of ADEM

          -  patients with severe cardiac disease or significant findings on the screening ECG.

          -  patients with severe renal insufficiency

        Key Inclusion Criteria Extension Phase:

        Applies to all patients participating in the Core Phase and then entering the Extension
        Phase. 1. Patients that originally met Core Phase Inclusion criteria and completed the Core
        phase on or off of study drug.

        Applies to patients newly recruited to participate in the Extension Phase.

          -  All newly recruited patients' that enroll directly into the Extension Phase must
             fulfill the local country health authority product label approved for pediatric age
             group for inclusion criteria.

          -  Central review (including initial MRI report) of the diagnosis of pediatric MS will be
             required for all newly recruited patients.

        Key Exclusion Criteria Extension Phase:

        Applies to patients who completed the Core Phase, but prematurely discontinued study drug.

          1. Premature discontinuation of the study drug during the Core Phase due to:

               -  an adverse event,

               -  serious adverse event,

               -  laboratory abnormality

               -  other conditions leading to permanent study drug discontinuation due to safety
                  reasons

          2. Patients with known new events or concomitant medications (washout periods required
             prior to Visit 15) that would exclude them from the Core Phase exclusion criteria.
             Serological or other additional tests will not be required.

        Applies to patients newly recruited in the younger cohort to participate in the Extension
        Phase.

        1. All newly recruited patients in the younger cohort that enroll directly into the
        Extension Phase must fulfill the exclusion criteria for the core phase.