Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
- Conditions
- Multiple Sclerosis
- Interventions
- Registration Number
- NCT01892722
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS)
- Detailed Description
The study is divided into a Core Phase, which includes the Double-Blind Treatment Period, and an Extension Phase in which all patients will be treated with fingolimod. The Core Phase is a 24-month, double-blind, randomized, active-controlled, parallel-group multicenter study phase to evaluate the efficacy and safety of fingolimod compared to IFN β-1a in children/adolescent patients aged 10-17 years old with MS. The Extension Phase is a 60-month (5 year) study phase for patients who complete the Core Phase of the study and meet all inclusion/exclusion criteria and for patients who will be recruited in the younger cohort to participate in the Extension Phase. The 'younger cohort' refers to the population of pediatric patients fulfilling 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). The recruitment of the younger cohort (up to 25 patients) was requested as a post- approval health authority commitment
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 240
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Fingolimod 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 Fingolimod Placebo capsule 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 Interferon beta-1a 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 Interferon beta-1a Placebo i.m. injection 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 Fingolimod-Younger Cohort Fingolimod 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)
- Primary Outcome Measures
Name Time Method Frequency of Relapses in Patients Treated for up to 24 Months 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
Name Time Method Time to First Relapse 24 months Time to first relapse was determined.
T1 Gd- Enhancing Lesions 24 months Number of T1 Gd-enhancing lesions per scan up to Month 24
Proportion of Patients Relapse-free 24 months Proportion of patients relapse-free was determined
Pharmacokinetics (Cavg) of Fingolimod-P 24 months Cavg (average drug concentration over the dose interval) will be evaluated.
New/Newly Enlarged T2 Lesions 24 months Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24
Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels 24 months Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.
Trial Locations
- Locations (12)
AMO Corporation
🇺🇸Tallahassee, Florida, United States
UAB Childrens Hospital Harbor Center Neurology Dept
🇺🇸Birmingham, Alabama, United States
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
UCSF
🇺🇸San Francisco, California, United States
University of Miami
🇺🇸Miami, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Wayne State University
🇺🇸Detroit, Michigan, United States
Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
Childrens Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Utah Clinical Trials Office
🇺🇸Salt Lake City, Utah, United States
Novartis Investigative Site
🇬🇧London, United Kingdom
San Jorge Childrens Hospital
🇵🇷Santurce, Puerto Rico