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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01892722




Registration number
NCT01892722
Ethics application status
Date submitted
8/05/2013
Date registered
4/07/2013

Titles & IDs
Public title
Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
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
Secondary ID [1] 0 0
2011-005677-23
Secondary ID [2] 0 0
CFTY720D2311
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Sclerosis 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of Relapses in Patients Treated for up to 24 Months
Timepoint [1] 0 0
24 months
Secondary outcome [1] 0 0
New/Newly Enlarged T2 Lesions
Timepoint [1] 0 0
24 months
Secondary outcome [2] 0 0
Time to First Relapse
Timepoint [2] 0 0
24 months
Secondary outcome [3] 0 0
Proportion of Patients Relapse-free
Timepoint [3] 0 0
24 months
Secondary outcome [4] 0 0
T1 Gd- Enhancing Lesions
Timepoint [4] 0 0
24 months
Secondary outcome [5] 0 0
Pharmacokinetics (Cavg) of Fingolimod-P
Timepoint [5] 0 0
24 months
Secondary outcome [6] 0 0
Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels
Timepoint [6] 0 0
24 months

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

Key
Minimum age
10 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/07/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
12/07/2029
Actual
Sample size
Target
245
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
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United States of America
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Alabama
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Edinburgh
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT01892722