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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01892722
Registration number
NCT01892722
Ethics application status
Date submitted
8/05/2013
Date registered
4/07/2013
Date last updated
23/04/2024
Titles & IDs
Public title
Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
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Scientific title
A 2 Year, Double-blind, Randomized, Multicenter, Active-controlled Core Phase to Evaluate Safety & Efficacy of Daily Fingolimod vs Weekly Interferon ß-1a im in Pediatric Patients With Multiple Sclerosis and 5 Year Fingolimod Extension Phase
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Secondary ID [1]
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2011-005677-23
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Secondary ID [2]
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CFTY720D2311
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Multiple Sclerosis
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Interferon beta-1a
Treatment: Drugs - Fingolimod
Treatment: Drugs - Placebo capsule
Treatment: Drugs - Placebo i.m. injection
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
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
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)
Treatment: Drugs: Interferon beta-1a
Administration once weekly via i.m. injections.
Treatment: Drugs: Fingolimod
Administrated orally once daily:
0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.
Treatment: Drugs: Placebo capsule
Matching placebo capsule required for double-dummy masking to blind formulations.
Treatment: Drugs: Placebo i.m. injection
Matching placebo i.m. injection required for double-dummy masking to blind formulations.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Frequency of Relapses in Patients Treated for up to 24 Months
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Assessment method [1]
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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).
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Timepoint [1]
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24 months
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Secondary outcome [1]
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New/Newly Enlarged T2 Lesions
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Assessment method [1]
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Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24
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Timepoint [1]
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24 months
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Secondary outcome [2]
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Time to First Relapse
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Assessment method [2]
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Time to first relapse was determined.
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Timepoint [2]
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24 months
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Secondary outcome [3]
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Proportion of Patients Relapse-free
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Assessment method [3]
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Proportion of patients relapse-free was determined
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Timepoint [3]
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24 months
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Secondary outcome [4]
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T1 Gd- Enhancing Lesions
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Assessment method [4]
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Number of T1 Gd-enhancing lesions per scan up to Month 24
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Timepoint [4]
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24 months
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Secondary outcome [5]
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Pharmacokinetics (Cavg) of Fingolimod-P
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Assessment method [5]
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Cavg (average drug concentration over the dose interval) will be evaluated.
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Timepoint [5]
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24 months
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Secondary outcome [6]
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Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels
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Assessment method [6]
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Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.
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Timepoint [6]
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24 months
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Eligibility
Key inclusion 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
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Minimum age
10
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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 ECG.
- 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.
1. 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
2. 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/07/2013
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
12/07/2029
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Actual
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Sample size
Target
235
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
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Name
Novartis Pharmaceuticals
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Address
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Ethics approval
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Summary
Brief summary
To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric
patients with multiple sclerosis (MS)
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01892722
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Novartis Pharmaceuticals
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Novartis Pharmaceuticals
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Novartis Pharmaceuticals
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1-888-669-6682
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01892722
Download to PDF