Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

NCT01892722 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 2 other identifiers: CFTY720D23112011-005677-23
• Study Type: interventional
• Target: 240
• Locations: 23 countries
• Sites: 71

Brief Summary

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

Conditions

Multiple Sclerosis

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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).

  24 months

Secondary Outcomes (6)

• New/Newly Enlarged T2 Lesions

  24 months

• Time to First Relapse

  24 months

• Proportion of Patients Relapse-free

  24 months

• T1 Gd- Enhancing Lesions

  24 months

• Pharmacokinetics (Cavg) of Fingolimod-P

  24 months

Study Arms (3)

Fingolimod

EXPERIMENTAL

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

Interferon beta-1a

ACTIVE COMPARATOR

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

Fingolimod-Younger Cohort

EXPERIMENTAL

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

Interferon beta-1a DRUG

Administration once weekly via i.m. injections.

Interferon beta-1a

Fingolimod DRUG

Administrated orally once daily: 0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.

Fingolimod; Fingolimod-Younger Cohort

Placebo capsule DRUG

Matching placebo capsule required for double-dummy masking to blind formulations.

Fingolimod

Placebo i.m. injection DRUG

Matching placebo i.m. injection required for double-dummy masking to blind formulations.

Interferon beta-1a

Eligibility Criteria

• Age: 10 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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
• Applies to patients newly recruited to participate in the Extension Phase.
• Central review (including initial MRI report) of the diagnosis of pediatric MS will be required for all newly recruited patients.
• 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
• Applies to patients newly recruited in the younger cohort to participate in the Extension Phase.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (73)

UAB Childrens Hospital Harbor Center Neurology Dept

Birmingham, Alabama, 35294, United States

Location

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

UCSF

San Francisco, California, 94115, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

AMO Corporation

Tallahassee, Florida, 32312, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Wayne State University

Detroit, Michigan, 48201, United States

Location

Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104 4399, United States

Location

University of Utah Clinical Trials Office

Salt Lake City, Utah, 84108, United States

Location

Novartis Investigative Site

Parkville, Victoria, 3052, Australia

Location

Novartis Investigative Site

Vienna, 1090, Austria

Location

Novartis Investigative Site

Belo Horizonte, Minas Gerais, 30150 221, Brazil

Location

Novartis Investigative Site

Rio de Janeiro, Rio de Janeiro, 20270-004, Brazil

Location

Novartis Investigative Site

São Paulo, São Paulo, 05403-000, Brazil

Location

Novartis Investigative Site

Goiânia, 74605 020, Brazil

Location

Novartis Investigative Site

Sofia, 1113, Bulgaria

Location

Novartis Investigative Site

Calgary, Alberta, T3B 6A8, Canada

Location

Novartis Investigative Site

Ottawa, Ontario, K1H 8L6, Canada

Location

Novartis Investigative Site

Osijek, 31000, Croatia

Location

Novartis Investigative Site

Tallinn, 10617, Estonia

Location

Novartis Investigative Site

Bordeaux, 33076, France

Location

Novartis Investigative Site

Le Kremlin-Bicêtre, 94275, France

Location

Novartis Investigative Site

Marseille, 13885, France

Location

Novartis Investigative Site

Montpellier, 34090, France

Location

Novartis Investigative Site

Toulouse, 31059, France

Location

Novartis Investigative Site

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Novartis Investigative Site

Göttingen, Lower Saxony, 37075, Germany

Location

Novartis Investigative Site

Dresden, Saxony, 01307, Germany

Location

Novartis Investigative Site

Bochum, 44791, Germany

Location

Novartis Investigative Site

Erlangen, 91054, Germany

Location

Novartis Investigative Site

Bari, BA, 70124, Italy

Location

Novartis Investigative Site

Montichiari, BS, 25018, Italy

Location

Novartis Investigative Site

Catania, CT, 95123, Italy

Location

Novartis Investigative Site

Milan, MI, 20132, Italy

Location

Novartis Investigative Site

Cefalù, PA, 90015, Italy

Location

Novartis Investigative Site

Roma, RM, 00133, Italy

Location

Novartis Investigative Site

Roma, RM, 00189, Italy

Location

Novartis Investigative Site

Gallarate, VA, 21013, Italy

Location

Novartis Investigative Site

Riga, LV-1004, Latvia

Location

Novartis Investigative Site

Kaunas, LTU, LT 50161, Lithuania

Location

Novartis Investigative Site

Mexico City, D F, 06700, Mexico

Location

Novartis Investigative Site

Distrito Federal, 03310, Mexico

Location

Novartis Investigative Site

Rotterdam, South Holland, 3015 CN, Netherlands

Location

Novartis Investigative Site

Lodz, 93-338, Poland

Location

Novartis Investigative Site

Lublin, 20-093, Poland

Location

Novartis Investigative Site

Poznan, 60-355, Poland

Location

Novartis Investigative Site

Wroclaw, 54-049, Poland

Location

San Jorge Childrens Hospital

Santurce, 00912, Puerto Rico

Location

Novartis Investigative Site

Bucharest, 041914, Romania

Location

Novartis Investigative Site

Kazan', 420043, Russia

Location

Novartis Investigative Site

Moscow, 119602, Russia

Location

Novartis Investigative Site

Moscow, 119991, Russia

Location

Novartis Investigative Site

Novosibirsk, 630087, Russia

Location

Novartis Investigative Site

Saint Petersburg, 197110, Russia

Location

Novartis Investigative Site

Belgrade, 11000, Serbia

Location

Novartis Investigative Site

Bratislava, 833 40, Slovakia

Location

Novartis Investigative Site

Seville, Andalusia, 41009, Spain

Location

Novartis Investigative Site

Vigo, Pontevedra, 36212, Spain

Location

Novartis Investigative Site

Barakaldo, Vizcaya, 48903, Spain

Location

Novartis Investigative Site

Madrid, 28006, Spain

Location

Novartis Investigative Site

Málaga, 29010, Spain

Location

Novartis Investigative Site

Valencia, 46010, Spain

Location

Novartis Investigative Site

Samsun, Atakum, 55200, Turkey (Türkiye)

Location

Novartis Investigative Site

Izmir, Balcova, 35340, Turkey (Türkiye)

Location

Novartis Investigative Site

Istanbul, Fatih, 34098, Turkey (Türkiye)

Location

Novartis Investigative Site

Ankara, Sihhiye-Altindag, 06230, Turkey (Türkiye)

Location

Novartis Investigative Site

Ankara, Yenimahalle, 06500, Turkey (Türkiye)

Location

Novartis Investigative Site

Kharkiv, 61068, Ukraine

Location

Novartis Investigative Site

West Midlands, Birmingham, B4 6NH, United Kingdom

Location

Novartis Investigative Site

Edinburgh, EH9 1LF, United Kingdom

Location

Novartis Investigative Site

London, NW1 2BU, United Kingdom

Location

Novartis Investigative Site

London, WC1N 3JH, United Kingdom

Location

Related Publications (2)

• Deiva K, Huppke P, Banwell B, Chitnis T, Gartner J, Krupp L, Waubant E, Stites T, Pearce GL, Merschhemke M. Consistent control of disease activity with fingolimod versus IFN beta-1a in paediatric-onset multiple sclerosis: further insights from PARADIGMS. J Neurol Neurosurg Psychiatry. 2020 Jan;91(1):58-66. doi: 10.1136/jnnp-2019-321124. Epub 2019 Aug 29.

  PMID: 31467033 DERIVED

• Chitnis T, Arnold DL, Banwell B, Bruck W, Ghezzi A, Giovannoni G, Greenberg B, Krupp L, Rostasy K, Tardieu M, Waubant E, Wolinsky JS, Bar-Or A, Stites T, Chen Y, Putzki N, Merschhemke M, Gartner J; PARADIGMS Study Group. Trial of Fingolimod versus Interferon Beta-1a in Pediatric Multiple Sclerosis. N Engl J Med. 2018 Sep 13;379(11):1017-1027. doi: 10.1056/NEJMoa1800149.

  PMID: 30207920 DERIVED

Related Links

• Recruitment website

MeSH Terms

Conditions

Multiple Sclerosis; Thinness

Interventions

Interferon beta-1a; Fingolimod Hydrochloride

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Interferon-beta; Interferon Type I; Interferons; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Sphingosine; Amino Alcohols; Alcohols; Organic Chemicals; Propylene Glycols; Glycols; Amines

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 8, 2013
• First Posted: July 4, 2013
• Study Start: July 26, 2013
• Primary Completion: July 14, 2017
• Study Completion (Estimated): February 18, 2030
• Last Updated: November 20, 2025
• Results First Posted: September 19, 2018

Record last verified: 2025-11

Locations

SE (1); ES (6); RU (5); TU (5); UA (1); FR (5); RO (1); PL (4); DE (5); GB (4); SL (1); CR (1); AU (1); LI (1); LA (1); ME (2); BU (1); NL (1); PR (1); CA (2); US (10); BR (4); IT (8)