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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 : Recruiting
First Posted : July 4, 2013
Results First Posted : September 19, 2018
Last Update Posted : August 14, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Multiple Sclerosis
Interventions Drug: Interferon beta-1a
Drug: Fingolimod
Drug: Placebo capsule
Drug: Placebo i.m. injection
Enrollment 245
Recruitment Details  
Pre-assignment Details This study is divided into a core phase and extension phase. In the core phase, patients were randomized to Fingolimod or Interferon beta-1a in a 1:1 ratio. The core phase disposition is reported for interim results disclosure. Upon completion of the extension phase, the extension disposition will be reported.
Arm/Group Title Fingolimod Interferon Beta-1a
Hide Arm/Group Description 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. An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Period Title: Overall Study
Started 107 108
Full Analysis Set 107 107
Completed 100 88
Not Completed 7 20
Reason Not Completed
Protocol deviation             0             1
Administrative problems             0             1
Patient/guardian decision             0             2
Physician Decision             1             2
Adverse Event             3             2
Lack of Efficacy             0             7
Withdrawal by Subject             3             5
Arm/Group Title Fingolimod Interferon Beta-1a Total
Hide Arm/Group Description 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. An intramuscular (IM) injection of Interferon beta-1a was administered once weekly. Total of all reporting groups
Overall Number of Baseline Participants 107 108 215
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 107 participants 108 participants 215 participants
15.2  (2.0) 15.4  (1.60) 15.3  (1.81)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 107 participants 108 participants 215 participants
Female
70
  65.4%
64
  59.3%
134
  62.3%
Male
37
  34.6%
44
  40.7%
81
  37.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 107 participants 108 participants 215 participants
American Indian or Alaska Native
3
   2.8%
2
   1.9%
5
   2.3%
Asian
1
   0.9%
0
   0.0%
1
   0.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   0.9%
4
   3.7%
5
   2.3%
White
100
  93.5%
97
  89.8%
197
  91.6%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
2
   1.9%
5
   4.6%
7
   3.3%
1.Primary Outcome
Title Frequency of Relapses in Patients Treated for up to 24 Months
Hide Description 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).
Time Frame 24 months
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set (FAS): The FAS was comprised of all randomized patients with assigned treatments who received at least one dose of study medication.
Arm/Group Title Fingolimod Interferon Beta-1a
Hide Arm/Group Description:
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.
An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
Overall Number of Participants Analyzed 107 107
Mean (95% Confidence Interval)
Unit of Measure: Confirmed relapse per year
0.122
(0.078 to 0.192)
0.675
(0.515 to 0.885)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Fingolimod, Interferon Beta-1a
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Negative binomial regression model
Comments [Not Specified]
2.Secondary Outcome
Title New/Newly Enlarged T2 Lesions
Hide Description Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24
Time Frame 24 months
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Time to First Relapse
Hide Description Time to first relapse was determined.
Time Frame 24 months
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Proportion of Patients Relapse-free
Hide Description Proportion of patients relapse-free was determined
Time Frame 24 months
Outcome Measure Data Not Reported
5.Secondary Outcome
Title T1 Gd- Enhancing Lesions
Hide Description Number of T1 Gd-enhancing lesions per scan up to Month 24
Time Frame 24 months
Outcome Measure Data Not Reported
6.Secondary Outcome
Title Pharmacokinetics (Cavg) of Fingolimod-P
Hide Description Cavg (average drug concentration over the dose interval) will be evaluated.
Time Frame 24 months
Outcome Measure Data Not Reported
7.Secondary Outcome
Title Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels
Hide Description Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.
Time Frame 24 months
Outcome Measure Data Not Reported
Time Frame Time Frame Adverse Events (AEs) are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) for the primary endpoint. All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit for the primary endpoint, up to approximately 4 years. The trial is ongoing.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Fingolimod Interferon Beta-1a
Hide Arm/Group Description 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. An intramuscular (IM) injection of Interferon beta-1a was administered once weekly.
All-Cause Mortality
Fingolimod Interferon Beta-1a
Affected / at Risk (%) Affected / at Risk (%)
Total   0/107 (0.00%)   0/107 (0.00%) 
Hide Serious Adverse Events
Fingolimod Interferon Beta-1a
Affected / at Risk (%) Affected / at Risk (%)
Total   19/107 (17.76%)   10/107 (9.35%) 
Blood and lymphatic system disorders     
Agranulocytosis  1  1/107 (0.93%)  0/107 (0.00%) 
Leukopenia  1  2/107 (1.87%)  0/107 (0.00%) 
Cardiac disorders     
Atrioventricular block second degree  1  1/107 (0.93%)  0/107 (0.00%) 
Supraventricular tachycardia  1  0/107 (0.00%)  1/107 (0.93%) 
Eye disorders     
Autoimmune uveitis  1  1/107 (0.93%)  0/107 (0.00%) 
Uveitis  1  0/107 (0.00%)  1/107 (0.93%) 
Gastrointestinal disorders     
Dyspepsia  1  1/107 (0.93%)  0/107 (0.00%) 
Gastrointestinal necrosis  1  1/107 (0.93%)  0/107 (0.00%) 
Gastrooesophageal reflux disease  1  0/107 (0.00%)  1/107 (0.93%) 
Rectal tenesmus  1  1/107 (0.93%)  0/107 (0.00%) 
Small intestinal obstruction  1  1/107 (0.93%)  0/107 (0.00%) 
General disorders     
Fatigue  1  0/107 (0.00%)  1/107 (0.93%) 
Pyrexia  1  0/107 (0.00%)  1/107 (0.93%) 
Infections and infestations     
Abscess oral  1  1/107 (0.93%)  0/107 (0.00%) 
Appendicitis  1  1/107 (0.93%)  0/107 (0.00%) 
Cellulitis  1  1/107 (0.93%)  0/107 (0.00%) 
Gastritis viral  1  0/107 (0.00%)  1/107 (0.93%) 
Gastrointestinal infection  1  1/107 (0.93%)  0/107 (0.00%) 
Paronychia  1  0/107 (0.00%)  1/107 (0.93%) 
Viral infection  1  1/107 (0.93%)  0/107 (0.00%) 
Viral pharyngitis  1  1/107 (0.93%)  0/107 (0.00%) 
Injury, poisoning and procedural complications     
Head injury  1  1/107 (0.93%)  0/107 (0.00%) 
Humerus fracture  1  1/107 (0.93%)  0/107 (0.00%) 
Investigations     
Alanine aminotransferase increased  1  1/107 (0.93%)  0/107 (0.00%) 
Body temperature increased  1  0/107 (0.00%)  1/107 (0.93%) 
Gamma-glutamyltransferase increased  1  1/107 (0.93%)  0/107 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  1/107 (0.93%)  0/107 (0.00%) 
Muscular weakness  1  1/107 (0.93%)  0/107 (0.00%) 
Nervous system disorders     
Dizziness  1  0/107 (0.00%)  1/107 (0.93%) 
Epilepsy  1  1/107 (0.93%)  0/107 (0.00%) 
Generalised tonic-clonic seizure  1  1/107 (0.93%)  0/107 (0.00%) 
Headache  1  0/107 (0.00%)  1/107 (0.93%) 
Migraine  1  1/107 (0.93%)  0/107 (0.00%) 
Migraine without aura  1  1/107 (0.93%)  0/107 (0.00%) 
Multiple sclerosis plaque  1  1/107 (0.93%)  0/107 (0.00%) 
Multiple sclerosis relapse  1  1/107 (0.93%)  3/107 (2.80%) 
Optic neuritis  1  0/107 (0.00%)  1/107 (0.93%) 
Seizure  1  2/107 (1.87%)  0/107 (0.00%) 
Sensory loss  1  0/107 (0.00%)  1/107 (0.93%) 
Renal and urinary disorders     
Bladder spasm  1  1/107 (0.93%)  0/107 (0.00%) 
Dysuria  1  1/107 (0.93%)  0/107 (0.00%) 
Skin and subcutaneous tissue disorders     
Hypersensitivity vasculitis  1  1/107 (0.93%)  0/107 (0.00%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Fingolimod Interferon Beta-1a
Affected / at Risk (%) Affected / at Risk (%)
Total   82/107 (76.64%)   94/107 (87.85%) 
Blood and lymphatic system disorders     
Leukopenia  1  14/107 (13.08%)  3/107 (2.80%) 
Gastrointestinal disorders     
Abdominal pain  1  9/107 (8.41%)  9/107 (8.41%) 
Diarrhoea  1  8/107 (7.48%)  10/107 (9.35%) 
Nausea  1  9/107 (8.41%)  5/107 (4.67%) 
Toothache  1  6/107 (5.61%)  2/107 (1.87%) 
Vomiting  1  8/107 (7.48%)  7/107 (6.54%) 
General disorders     
Chills  1  1/107 (0.93%)  11/107 (10.28%) 
Fatigue  1  10/107 (9.35%)  6/107 (5.61%) 
Influenza like illness  1  5/107 (4.67%)  40/107 (37.38%) 
Pyrexia  1  8/107 (7.48%)  21/107 (19.63%) 
Infections and infestations     
Influenza  1  12/107 (11.21%)  4/107 (3.74%) 
Nasopharyngitis  1  8/107 (7.48%)  5/107 (4.67%) 
Rhinitis  1  10/107 (9.35%)  9/107 (8.41%) 
Upper respiratory tract infection  1  17/107 (15.89%)  5/107 (4.67%) 
Viral upper respiratory tract infection  1  23/107 (21.50%)  26/107 (24.30%) 
Investigations     
Glomerular filtration rate decreased  1  1/107 (0.93%)  6/107 (5.61%) 
White blood cell count decreased  1  6/107 (5.61%)  0/107 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  6/107 (5.61%)  6/107 (5.61%) 
Nervous system disorders     
Headache  1  34/107 (31.78%)  31/107 (28.97%) 
Psychiatric disorders     
Anxiety  1  7/107 (6.54%)  2/107 (1.87%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  10/107 (9.35%)  12/107 (11.21%) 
Oropharyngeal pain  1  9/107 (8.41%)  5/107 (4.67%) 
1
Term from vocabulary, MedDRA (20.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
EMail: novartis.email@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01892722    
Other Study ID Numbers: CFTY720D2311
2011-005677-23 ( EudraCT Number )
First Submitted: May 8, 2013
First Posted: July 4, 2013
Results First Submitted: June 8, 2018
Results First Posted: September 19, 2018
Last Update Posted: August 14, 2020