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Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2016 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01892722
First received: May 8, 2013
Last updated: November 10, 2016
Last verified: November 2016
  Purpose
To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pedaitric patients with multiple sclerosis

Condition Intervention 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
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Two-year, Double-blind, Randomzied, Multicenter, Active-controlled Study to Evaluate Safety and Efficacy of Oral Fingolimod Versus Interferon Beta-1a i.m. in Pediatric Patients With Multiple Sclerosis

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Frequency of relapses" in patients treated for up to 24 months [ Time Frame: 24 months ] [ Designated as safety issue: No ]
    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:
  • Number of new/newly enlarged T2 (n/neT2) lesions [ Time Frame: 24 months ] [ Designated as safety issue: No ]
    Cumulative number of new/newly enlarged T2 lesions (n/neT2) over 24 months.

  • Frequency and nature of adverse events as a measure of Safety and Tolerability [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
    Based on frequency adverse events (the number and percentage of patients having any AE by primary system organ class and preferred term) and on the incidence of clinically notable laboratory abnormalities.

  • Pharmacokinetics (Cavg)of fingolimod and fingolimod-P [ Time Frame: 24 months ] [ Designated as safety issue: No ]
    Characterization of pharmacokinetics (PK): Cavg (average drug concentration over the dose interval) will be evaluated. Determine if population covariates affect PK in the pediatric MS population.


Estimated Enrollment: 190
Study Start Date: July 2013
Estimated Study Completion Date: March 2023
Estimated Primary Completion Date: March 2023 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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).

  Eligibility

Ages Eligible for Study:   10 Years to 17 Years   (Child)
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01892722

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals +41613241111

  Show 104 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01892722     History of Changes
Other Study ID Numbers: CFTY720D2311  2011-005677-23 
Study First Received: May 8, 2013
Last Updated: November 10, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
pediatric
multiple sclerosis

Additional relevant MeSH terms:
Sclerosis
Multiple 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

ClinicalTrials.gov processed this record on December 06, 2016