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Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

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ClinicalTrials.gov Identifier: NCT01892722
Recruitment Status : Active, not recruiting
First Posted : July 4, 2013
Last Update Posted : April 17, 2018
Sponsor:
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

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
Active Comparator: Interferon beta-1a i.m.
Interferon beta-1a once-weekly i.m.
Drug: Interferon beta-1a i.m. injections
Administration once weekly via i.m. injections. Double-dummy masking is required to blind formulations: Patients in the interferon beta-1a i.m. arm will also take daily placebo capsules matched in appearance to the fingolimod active capsules.
Drug: Placebo oral capsule
Placebo capsule required for double-dummy masking to blind formulations: Patients in interferon beta-1a i.m. arm will also receive daily oral placebo capsules matched in appearance to the fingolimod active capsules.
Experimental: Fingolimod
Fingolimod will be 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. Double-dummy masking is required to blind formulations: Patients in the fingolimod arm will also take weekly placebo i.m. injections (syringes matched in appearance to the active interferon beta-1a i.m. syringes).
Drug: Placebo i.m. injection
Placebo i.m. injection required for double-dummy masking to blind formulations: Patients in the fingolimod arm will also take weekly placebo i.m. injections (syringes matched in appearance to the active interferon beta-1a i.m. syringes).



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.



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

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

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 Other protocol-defined inclusion/exclusion criteria may apply

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 ClinicalTrials.gov identifier (NCT number): NCT01892722


  Show 86 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01892722     History of Changes
Other Study ID Numbers: CFTY720D2311
2011-005677-23 ( EudraCT Number )
First Posted: July 4, 2013    Key Record Dates
Last Update Posted: April 17, 2018
Last Verified: April 2018

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
pediatric
multiple sclerosis

Additional relevant MeSH terms:
Multiple Sclerosis
Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Interferons
Interferon-beta
Interferon beta-1a
Fingolimod Hydrochloride
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunologic Factors
Physiological Effects of Drugs
Immunosuppressive Agents
Adjuvants, Immunologic