Trial Outcomes & Findings for Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis (NCT NCT01892722)

Last Updated: 2025-11-20

Results Overview

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).

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

240 participants

Primary outcome timeframe

24 months

Results posted on

2025-11-20

Participant Flow

This study is divided into a core phase and extension phase. In the core phase, patients were randomized to Fingolimod or Interferon beta-1a in a 1:1 ratio. The core phase disposition is reported for interim results disclosure. Upon completion of the extension phase, the extension disposition will be reported.

Participant milestones

Participant milestones
Measure	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.	Interferon Beta-1a An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Overall Study STARTED	107	108
Overall Study Full Analysis Set	107	107
Overall Study COMPLETED	100	88
Overall Study NOT COMPLETED	7	20

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	Interferon Beta-1a An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Overall Study Protocol deviation	0	1
Overall Study Administrative problems	0	1
Overall Study Patient/guardian decision	0	2
Overall Study Physician Decision	1	2
Overall Study Adverse Event	3	2
Overall Study Lack of Efficacy	0	7
Overall Study Withdrawal by Subject	3	5

Baseline Characteristics

Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fingolimod n=107 Participants 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.	Interferon Beta-1a n=108 Participants An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.	Total n=215 Participants Total of all reporting groups
Age, Continuous	15.2 Years STANDARD_DEVIATION 2.0	15.4 Years STANDARD_DEVIATION 1.60 • n=4 Participants	15.3 Years STANDARD_DEVIATION 1.81 • n=8 Participants
Sex: Female, Male Female	70 Participants	64 Participants n=4 Participants	134 Participants n=8 Participants
Sex: Female, Male Male	37 Participants	44 Participants n=4 Participants	81 Participants n=8 Participants
Race (NIH/OMB) American Indian or Alaska Native	3 Participants	2 Participants n=4 Participants	5 Participants n=8 Participants
Race (NIH/OMB) Asian	1 Participants	0 Participants n=4 Participants	1 Participants n=8 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Black or African American	1 Participants	4 Participants n=4 Participants	5 Participants n=8 Participants
Race (NIH/OMB) White	100 Participants	97 Participants n=4 Participants	197 Participants n=8 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants	5 Participants n=4 Participants	7 Participants n=8 Participants

PRIMARY outcome

Timeframe: 24 months

Population: Full analysis set (FAS): The FAS was comprised of all randomized patients with assigned treatments who received at least one dose of study medication.

Outcome measures

Outcome measures
Measure	Fingolimod n=107 Participants 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.	Interferon Beta-1a n=107 Participants An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Frequency of Relapses in Patients Treated for up to 24 Months	0.122 Confirmed relapse per year Interval 0.078 to 0.192	0.675 Confirmed relapse per year Interval 0.515 to 0.885

Adverse Events

Fingolimod

Serious events: 19 serious events

Other events: 82 other events

Deaths: 0 deaths

Interferon Beta-1a

Serious events: 10 serious events

Other events: 94 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER

Measure	Fingolimod n=107 participants at risk 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.	Interferon Beta-1a n=107 participants at risk An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Gastrointestinal disorders Vomiting	7.5% 8/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	6.5% 7/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
General disorders Chills	0.93% 1/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	10.3% 11/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
General disorders Fatigue	9.3% 10/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
General disorders Influenza like illness	4.7% 5/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	37.4% 40/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
General disorders Pyrexia	7.5% 8/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	19.6% 21/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Infections and infestations Influenza	11.2% 12/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	3.7% 4/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Infections and infestations Nasopharyngitis	7.5% 8/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	4.7% 5/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Blood and lymphatic system disorders Leukopenia	13.1% 14/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	2.8% 3/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Gastrointestinal disorders Abdominal pain	8.4% 9/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	8.4% 9/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Gastrointestinal disorders Diarrhoea	7.5% 8/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	9.3% 10/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Gastrointestinal disorders Nausea	8.4% 9/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	4.7% 5/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Gastrointestinal disorders Toothache	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	1.9% 2/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Infections and infestations Rhinitis	9.3% 10/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	8.4% 9/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Infections and infestations Upper respiratory tract infection	15.9% 17/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	4.7% 5/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Infections and infestations Viral upper respiratory tract infection	21.5% 23/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	24.3% 26/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Investigations Glomerular filtration rate decreased	0.93% 1/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Investigations White blood cell count decreased	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	0.00% 0/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Musculoskeletal and connective tissue disorders Back pain	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	5.6% 6/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Nervous system disorders Headache	31.8% 34/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	29.0% 31/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Psychiatric disorders Anxiety	6.5% 7/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	1.9% 2/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Respiratory, thoracic and mediastinal disorders Cough	9.3% 10/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	11.2% 12/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	8.4% 9/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.	4.7% 5/107 • Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.