Multiple SclerosisPhase 3NCT01892722
Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
A Phase 3 study testing Interferon beta-1a and Fingolimod in people with Multiple Sclerosis, run by Novartis Pharmaceuticals at 73 sites in 25 countries.
Trial summary
To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS) Read more about this study
- Who is it for?
- Ages 10 to 17 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- Interferon beta-1a and Fingolimod
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 73 sitesin 25 countries
- Study dates
- 26 Jul 2013to 18 Feb 2030
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
- 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.
Who cannot join
- 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 who completed the Core Phase, but prematurely discontinued study drug.
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- 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. 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.
Trial locations and contacts
73 sites in 25 countries.
73 sites in 25 countries
United States10 sites
UAB Childrens Hospital Harbor Center Neurology Dept
Birmingham, Alabama, 35294
Massachusetts General Hospital
Boston, Massachusetts, 02114
Wayne State University
Detroit, Michigan, 48201
Childrens Hospital Los Angeles
Los Angeles, California, 90027
University of Miami
Miami, Florida, 33136
Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
University of Utah Clinical Trials Office
Salt Lake City, Utah, 84108
UCSF
San Francisco, California, 94115
AMO Corporation
Tallahassee, Florida, 32312
Italy8 sites
Novartis Investigative Site
Bari, BA, 70124
Novartis Investigative Site
Catania, CT, 95123
Novartis Investigative Site
Cefalù, PA, 90015
Novartis Investigative Site
Gallarate, VA, 21013
Novartis Investigative Site
Milan, MI, 20132
Novartis Investigative Site
Montichiari, BS, 25018
Novartis Investigative Site
Roma, RM, 00133
Novartis Investigative Site
Roma, RM, 00189
Spain6 sites
Novartis Investigative Site
Barakaldo, Vizcaya, 48903
Novartis Investigative Site
Madrid, 28006
Novartis Investigative Site
Málaga, 29010
Novartis Investigative Site
Seville, 41009
Novartis Investigative Site
Valencia, 46010
Novartis Investigative Site
Vigo, Pontevedra, 36212
France5 sites
Novartis Investigative Site
Bordeaux, 33076
Novartis Investigative Site
Le Kremlin-Bicêtre, 94275
Novartis Investigative Site
Marseille, 13885
Novartis Investigative Site
Montpellier, 34090
Novartis Investigative Site
Toulouse, 31059
Germany5 sites
Novartis Investigative Site
Bochum, 44791
Novartis Investigative Site
Dresden, Saxony, 01307
Novartis Investigative Site
Erlangen, 91054
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Novartis Investigative Site
Göttingen, Lower Saxony, 37075
Russia5 sites
Novartis Investigative Site
Kazan', 420043
Novartis Investigative Site
Moscow, 119602
Novartis Investigative Site
Moscow, 119991
Novartis Investigative Site
Novosibirsk, 630087
Novartis Investigative Site
Saint Petersburg, 197110
Turkey (Türkiye)5 sites
Novartis Investigative Site
Ankara, Sihhiye-Altindag, 06230
Novartis Investigative Site
Ankara, Yenimahalle, 06500
Novartis Investigative Site
Istanbul, Fatih, 34098
Novartis Investigative Site
Izmir, Balcova, 35340
Novartis Investigative Site
Samsun, Atakum, 55200
Brazil4 sites
Novartis Investigative Site
Belo Horizonte, Minas Gerais, 30150-221
Novartis Investigative Site
Goiânia, 74605-020
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, 20270-004
Novartis Investigative Site
São Paulo, São Paulo, 05403-000
Poland4 sites
Novartis Investigative Site
Lodz, 93-338
Novartis Investigative Site
Lublin, 20-093
Novartis Investigative Site
Poznan, 60-355
Novartis Investigative Site
Wroclaw, 54-049
United Kingdom4 sites
Novartis Investigative Site
Edinburgh, EH9 1LF
Novartis Investigative Site
London, NW1 2BU
Novartis Investigative Site
London, WC1N 3JH
Novartis Investigative Site
West Midlands, Birmingham, B4 6NH
Canada2 sites
Novartis Investigative Site
Calgary, Alberta, T3B 6A8
Novartis Investigative Site
Ottawa, Ontario, K1H 8L6
Mexico2 sites
Novartis Investigative Site
Distrito Federal, 03310
Novartis Investigative Site
Mexico City, D F, 06700
Netherlands1 site
Novartis Investigative Site
Rotterdam, South Holland, 3015 CN
About this study
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
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
- Multiple Sclerosis
- Being tested
- Interferon beta-1a, Fingolimod, Placebo capsule, Placebo i.m. injection
- Keywords
- pediatric, multiple sclerosis, fingolimod, pre-pubertal, low weight, children, adolescent, MS, core phase, extension phase, younger cohort
- Medical subject headings
- Multiple Sclerosis, Thinness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Interferon beta-1aDrug
Administration once weekly via i.m. injections.
FingolimodDrug
Administrated orally once daily:
0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
Placebo capsuleDrug
Matching placebo capsule required for double-dummy masking to blind formulations.
Placebo i.m. injectionDrug
Matching placebo i.m. injection required for double-dummy masking to blind formulations.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: 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) Receives: Drug: Fingolimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 24 months
Secondary outcomes (6)
New/Newly Enlarged T2 Lesions
Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24
Measured: 24 months
Time to First Relapse
Time to first relapse was determined.
Measured: 24 months
Proportion of Patients Relapse-free
Proportion of patients relapse-free was determined
Measured: 24 months
T1 Gd- Enhancing Lesions
Number of T1 Gd-enhancing lesions per scan up to Month 24
Measured: 24 months
Pharmacokinetics (Cavg) of Fingolimod-P
Cavg (average drug concentration over the dose interval) will be evaluated.
Measured: 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.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2013 (estimated)
- Study start26 Jul 2013
- Primary completion14 Jul 2017
- Study completion18 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 240 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01892722
- Sponsor's protocol ID
- CFTY720D2311
- Other IDs
- 2011-005677-23
- Status last verified
- Aug 2026
- First submitted
- 8 May 2013
- Last updated
- 10 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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 www.clinicalstudydatarequest.com
Publications
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.
PubMed 31467033Chitnis 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.
PubMed 30207920