Active, not recruitingNCT01892722Updated Aug 10, 2026Results posted

Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

A Phase 3 interventional study of Interferon beta-1a and Fingolimod in Multiple Sclerosis, sponsored by Novartis Pharmaceuticals. Active, not recruiting at 73 sites in 25 countries. Open to participants aged 10 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-08-10.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 240
Allocation: Randomized
Ages: 10 Years to 17 Years
Sex: All

Study summary

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

Read the detailed description

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

Conditions studied

Multiple Sclerosis

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Keywords

pediatric
multiple sclerosis
fingolimod
pre-pubertal
low weight
children
adolescent
MS
core phase
extension phase
younger cohort

In context

Multiple Sclerosis

3,264 studies on the registry are indexed under Multiple Sclerosis; 659 are open to participants now.

This study's enrollment of 240 is above the median of 50 across 2,201 interventional studies indexed under Multiple Sclerosis.

Browse Multiple Sclerosis studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.

Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.

Browse Novartis Pharmaceuticals studies →All Novartis studies →

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 10 Years to 17 Years
Sexes eligible: All
Accepts healthy volunteers: No

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 .
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.

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 ( 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.

Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

ECG (electrocardiogram): A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Washout period: A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Double (Participant, Investigator)
Enrollment: 240 participants (actual)

Study arms

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 · Drug: Placebo capsule
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 · Drug: Placebo i.m. injection
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

Interventions

DrugInterferon beta-1a
Administration once weekly via i.m. injections.
DrugFingolimod
Administrated orally once daily: 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
DrugPlacebo capsule
Matching placebo capsule required for double-dummy masking to blind formulations.
DrugPlacebo i.m. injection
Matching placebo i.m. injection required for double-dummy masking to blind formulations.

What researchers measure

Primary outcomes

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

Secondary outcomes

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

Results

Posted Sep 19, 2018

Participant flow

Participant flow — Overall Study
Milestone	Fingolimod	Interferon Beta-1a
Started	107	108
Full analysis set	107	107
Completed	100	88
Not completed	7	20
Withdrew: Protocol deviation	0	1
Withdrew: Administrative problems	0	1
Withdrew: Patient/guardian decision	0	2
Withdrew: Physician decision	1	2
Withdrew: Adverse event	3	2
Withdrew: Lack of efficacy	0	7
Withdrew: Withdrawal by subject	3	5

Outcome measures

PrimaryFrequency 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
Reported as:: Mean · Confirmed relapse per year

Frequency of Relapses in Patients Treated for up to 24 Months
Confirmed relapse per year	Fingolimod	Interferon Beta-1a
Frequency of Relapses in Patients Treated for up to 24 Months	0.122 (0.078 to 0.192)	0.675 (0.515 to 0.885)

Statistical analysis

Fingolimod vs Interferon Beta-1a · Negative binomial regression model · p = <0.001

SecondaryNew/Newly Enlarged T2 Lesions

Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24

Time frame:: 24 months