Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

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

ClinicalTrials.gov Identifier: NCT01892722

Novartis Reference Number: CFTY720D2311

Last Update: Jan 17, 2023

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS)

Condition

Multiple Sclerosis

Phase

Phase 3

Overall status

Recruiting

Start date

Jul 26, 2013

Completion date

Nov 02, 2028

Gender

All

Age(s)

10 Years - 17 Years (Child)

Interventions

Drug

Interferon beta-1a

Administration once weekly via i.m. injections.

Drug

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.

Drug

Placebo capsule

Matching placebo capsule required for double-dummy masking to blind formulations.

Drug

Placebo i.m. injection

Matching placebo i.m. injection required for double-dummy masking to blind formulations.

Eligibility 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 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.

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.