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Trial record 1 of 1 for:    NCT01892722
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Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01892722
Recruitment Status : Active, not recruiting
First Posted : July 4, 2013
Results First Posted : September 19, 2018
Last Update Posted : February 26, 2019
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Drug: Interferon beta-1a i.m. injections Drug: Fingolimod oral capsules Drug: Placebo oral capsule Drug: Placebo i.m. injection Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 215 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A 2 Year, Double-blind, Randomized, Multicenter, Active-controlled Core Phase to Evaluate Safety & Efficacy of Daily Fingolimod vs Weekly Interferon β-1a im in Pediatric Patients With Multiple Sclerosis and 5 Year Fingolimod Extension Phase
Actual Study Start Date : July 26, 2013
Actual Primary Completion Date : July 14, 2017
Estimated Study Completion Date : March 15, 2023

Arm Intervention/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.
Drug: Fingolimod oral capsules
Administration orally once daily.

Drug: Placebo i.m. injection
Placebo i.m. injection required for double-dummy masking to blind formulations.

Active Comparator: Interferon beta-1a
An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Drug: Interferon beta-1a i.m. injections
Administration once weekly via i.m. injections.

Drug: Placebo oral capsule
Placebo capsule required for double-dummy masking to blind formulations.

Primary Outcome Measures :
  1. Frequency of Relapses in Patients Treated for up to 24 Months [ Time Frame: 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).

Secondary Outcome Measures :
  1. New/Newly Enlarged T2 Lesions [ Time Frame: 24 months ]
    Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24

  2. Time to First Relapse [ Time Frame: 24 months ]
    Time to first relapse was determined.

  3. Proportion of Patients Relapse-free [ Time Frame: 24 months ]
    Proportion of patients relapse-free was determined

  4. T1 Gd- Enhancing Lesions [ Time Frame: 24 months ]
    Number of T1 Gd-enhancing lesions per scan up to Month 24

  5. Pharmacokinetics (Cavg) of Fingolimod-P [ Time Frame: 24 months ]
    Cavg (average drug concentration over the dose interval) will be evaluated.

  6. Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels [ Time Frame: 24 months ]
    Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key 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

Key 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01892722

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Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Novartis ( Novartis Pharmaceuticals ):
Statistical Analysis Plan  [PDF] August 4, 2017
Study Protocol  [PDF] November 16, 2016

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01892722    
Other Study ID Numbers: CFTY720D2311
2011-005677-23 ( EudraCT Number )
First Posted: July 4, 2013    Key Record Dates
Results First Posted: September 19, 2018
Last Update Posted: February 26, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
multiple sclerosis
Additional relevant MeSH terms:
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Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Interferon beta-1a
Fingolimod Hydrochloride
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic
Immunosuppressive Agents