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Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

PHASE3RECRUITING

To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i. m.

in pediatric patients with multiple sclerosis (MS).

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  • 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
  • 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
  • Exclusion criteria

  • 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
  • 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
  • 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
  • All newly recruited patients in the younger cohort that enroll directly into the Extension Phase must fulfill the exclusion criteria for the core phase
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    Eligibility

    Age eligible for study : 10 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2013-07-26

    Primary completion: 2017-07-14

    Study completion finish: 2029-07-12

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE3

    trial

    Trial ID

    NCT01892722

    Intervention or treatment

    DRUG: Interferon beta-1a

    DRUG: Fingolimod

    DRUG: Placebo capsule

    DRUG: Placebo i.m. injection

    Conditions

    • Multiple Sclerosis

    Find a site

    Closest Location:

    Novartis Investigative Site

    Research sites nearby

    Select from list below to view details:

    • Novartis Investigative Site

      Parkville, Victoria, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    EXPERIMENTAL: Fingolimod
    • Fingolimod was administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight) with the aim to achieve systemic exposure in range of that in adults at the licensed 0.5 mg dose. Participants in this arm during core continued into extension and received open-label treatment
    DRUG: Fingolimod
    • Administrated orally once daily:
    • 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
    ACTIVE_COMPARATOR: Interferon beta-1a
    • An intramuscular (IM) injection of Interferon beta-1a was administered once weekly during core phase. Participants switched to receive open-label fingolimod in extension phase
    DRUG: Interferon beta-1a
    • Administration once weekly via i.m. injections.
    EXPERIMENTAL: Fingolimod-Younger Cohort
    • The 'younger cohort' refers to the new pediatric patients to be recruited in the extension phase who fulfill 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)
    DRUG: Fingolimod
    • Administrated orally once daily:
    • 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    Frequency of Relapses in Patients Treated for up to 24 MonthsFrequency 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).24 months

    Secondary outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    New/Newly Enlarged T2 LesionsAnnualized rate of the number of new/newly enlarged T2 lesions up to Month 2424 months
    Time to First RelapseTime to first relapse was determined.24 months
    Proportion of Patients Relapse-freeProportion of patients relapse-free was determined24 months
    T1 Gd- Enhancing LesionsNumber of T1 Gd-enhancing lesions per scan up to Month 2424 months
    Pharmacokinetics (Cavg) of Fingolimod-PCavg (average drug concentration over the dose interval) will be evaluated.24 months
    Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte LevelsPopulation PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.24 months

    Frequently Asked Questions

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    References

    Clinical Trials Gov: Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

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