Trial Outcomes & Findings for Efficacy and Safety of FTY720 in Patients With Relapsing Multiple Sclerosis (MS) (NCT NCT00537082)
NCT ID: NCT00537082
Last Updated: 2011-04-21
Results Overview
Brain Magnetic Resonance Imaging (MRI) was performed at Month 3 and Month 6. Gadolinium-enhancing lesions (active lesions) represent acute inflammatory activity only and dissipate within 2-8 weeks of appearance. MRI scans were analyzed by blinded readers at the central MRI Evaluation Center to ensure consistency. Any Gd-enhanced T1 weighted MRI data obtained less than 14 days after the steroid used to treat MS relapses is invalid and excluded.
COMPLETED
PHASE2
171 participants
Month 3 and Month 6
2011-04-21
Participant Flow
Participant milestones
| Measure |
FTY720 1.25 mg
Administered orally once daily for 6 months
|
FTY720 0.5 mg
Administered orally once daily for 6 months
|
Placebo
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Overall Study
STARTED
|
57
|
57
|
57
|
|
Overall Study
COMPLETED
|
48
|
48
|
51
|
|
Overall Study
NOT COMPLETED
|
9
|
9
|
6
|
Reasons for withdrawal
| Measure |
FTY720 1.25 mg
Administered orally once daily for 6 months
|
FTY720 0.5 mg
Administered orally once daily for 6 months
|
Placebo
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
6
|
6
|
3
|
|
Overall Study
Protocol Violation
|
0
|
2
|
1
|
|
Overall Study
Lack of Efficacy
|
0
|
0
|
2
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
0
|
|
Overall Study
Discontinued before initiating treatment
|
3
|
0
|
0
|
Baseline Characteristics
Efficacy and Safety of FTY720 in Patients With Relapsing Multiple Sclerosis (MS)
Baseline characteristics by cohort
| Measure |
FTY720 1.25 mg
n=57 Participants
Administered orally once daily for 6 months
|
FTY720 0.5 mg
n=57 Participants
Administered orally once daily for 6 months
|
Placebo
n=57 Participants
Administered orally once daily for 6 months
|
Total
n=171 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age Continuous
|
36.0 years
STANDARD_DEVIATION 9.31 • n=5 Participants
|
35.0 years
STANDARD_DEVIATION 9.01 • n=7 Participants
|
35.0 years
STANDARD_DEVIATION 8.93 • n=5 Participants
|
35.3 years
STANDARD_DEVIATION 9.04 • n=4 Participants
|
|
Sex: Female, Male
Female
|
39 Participants
n=5 Participants
|
40 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
118 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
53 Participants
n=4 Participants
|
|
Region of Enrollment
Japan
|
57 participants
n=5 Participants
|
57 participants
n=7 Participants
|
57 participants
n=5 Participants
|
171 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Month 3 and Month 6Population: Modified Full Analysis Set (Modified FAS) included all patients who were randomized and received at least one dose of study drug and had at least one valid post-randomized MRI scan at Month 3 or longer.
Brain Magnetic Resonance Imaging (MRI) was performed at Month 3 and Month 6. Gadolinium-enhancing lesions (active lesions) represent acute inflammatory activity only and dissipate within 2-8 weeks of appearance. MRI scans were analyzed by blinded readers at the central MRI Evaluation Center to ensure consistency. Any Gd-enhanced T1 weighted MRI data obtained less than 14 days after the steroid used to treat MS relapses is invalid and excluded.
Outcome measures
| Measure |
FTY720 1.25 mg
n=50 Participants
Administered orally once daily for 6 months
|
FTY720 0.5 mg
n=50 Participants
Administered orally once daily for 6 months
|
Placebo
n=52 Participants
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Number of Patients Free of Gadolinium-enhanced T1-Weighted Magnetic Resonance Imaging (MRI) Lesions at Both Month 3 and Month 6
|
43 Participants
|
35 Participants
|
21 Participants
|
SECONDARY outcome
Timeframe: up to Month 6Population: Full Analysis Set (FAS) consisted of all patients who were randomized and received at least one dose of study drug. This population was only used for the analyses of relapse and EDSS.
A relapse must have been confirmed by a neurologist and was confirmed when it was accompanied by an increase of at least half a step (0.5) on the Expanded Disability Status Scale (EDSS) or an increase of 1 point on two different Functional Systems (FS) of the EDSS or 2 points on one of the FS (excluding Bowel/Bladder or Cerebral FS).
Outcome measures
| Measure |
FTY720 1.25 mg
n=54 Participants
Administered orally once daily for 6 months
|
FTY720 0.5 mg
n=57 Participants
Administered orally once daily for 6 months
|
Placebo
n=57 Participants
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Number of Patients Free of MS Relapse up to Month 6 (Confirmed Relapse Only)
|
45 Participants
|
45 Participants
|
37 Participants
|
SECONDARY outcome
Timeframe: up to Month 3 and up to Month 6Population: Modified Full Analysis Set (Modified FAS) included all patients who were randomized and received at least one dose of study drug and had at least one valid post-randomized MRI scan at Month 3 or longer.
The number of T2 lesions were obtained from MRI scans at Screening visit. The numbers of new/newly enlarging T2 lesions were obtained from MRI scans at Month 3 or more. New lesions were identified by comparing each lesion already seen in previous examinations. Lesions expanding throughout several slices were counted as only one lesion.
Outcome measures
| Measure |
FTY720 1.25 mg
n=50 Participants
Administered orally once daily for 6 months
|
FTY720 0.5 mg
n=50 Participants
Administered orally once daily for 6 months
|
Placebo
n=52 Participants
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Number of Patients Free of New or Newly Enlarged T2 Lesions
Month 3 (n = 50, 49, 51)
|
30 Participants
|
34 Participants
|
23 Participants
|
|
Number of Patients Free of New or Newly Enlarged T2 Lesions
Month 6 (n = 48, 48, 50)
|
28 Participants
|
31 Participants
|
18 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsPopulation: Full Analysis Set (FAS) consisted of all patients who were randomized and received at least one dose of study drug. This population was only used for the analyses of relapse and EDSS.
Annualized relapse rate (ARR) of the treatment group is calculated by taking the total number of confirmed relapses for all the patients in the treatment group divided by the total number of days on study for all patients in the group and multiplied by 365.25 to obtain the annual rate.
Outcome measures
| Measure |
FTY720 1.25 mg
n=54 Participants
Administered orally once daily for 6 months
|
FTY720 0.5 mg
n=57 Participants
Administered orally once daily for 6 months
|
Placebo
n=57 Participants
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Annualized Relapse Rate (ARR) at 6 Months
|
0.407 Relapses per year
|
0.512 Relapses per year
|
1.131 Relapses per year
|
Adverse Events
FTY720 1.25mg
FTY720 0.5mg
Placebo
Serious adverse events
| Measure |
FTY720 1.25mg
n=54 participants at risk
Administered orally once daily for 6 months
|
FTY720 0.5mg
n=57 participants at risk
Administered orally once daily for 6 months
|
Placebo
n=57 participants at risk
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Cardiac disorders
Atrioventricular block first degree
|
0.00%
0/54
|
1.8%
1/57
|
0.00%
0/57
|
|
Cardiac disorders
Atrioventricular block second degree
|
3.7%
2/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Cardiac disorders
Bradycardia
|
14.8%
8/54
|
5.3%
3/57
|
0.00%
0/57
|
|
Cardiac disorders
Sinus bradycardia
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Cardiac disorders
Stress cardiomyopathy
|
0.00%
0/54
|
0.00%
0/57
|
1.8%
1/57
|
|
Gastrointestinal disorders
Enterocolitis
|
0.00%
0/54
|
0.00%
0/57
|
1.8%
1/57
|
|
General disorders
Chest discomfort
|
0.00%
0/54
|
1.8%
1/57
|
0.00%
0/57
|
|
General disorders
Malaise
|
0.00%
0/54
|
1.8%
1/57
|
0.00%
0/57
|
|
Investigations
Heart rate decreased
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Investigations
Liver function test abnormal
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Nervous system disorders
Facial palsy
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Nervous system disorders
Multiple sclerosis relapse
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
|
Surgical and medical procedures
Abortion induced
|
0.00%
0/54
|
0.00%
0/57
|
1.8%
1/57
|
|
Vascular disorders
Hypotension
|
1.9%
1/54
|
0.00%
0/57
|
0.00%
0/57
|
Other adverse events
| Measure |
FTY720 1.25mg
n=54 participants at risk
Administered orally once daily for 6 months
|
FTY720 0.5mg
n=57 participants at risk
Administered orally once daily for 6 months
|
Placebo
n=57 participants at risk
Administered orally once daily for 6 months
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
5.6%
3/54
|
1.8%
1/57
|
0.00%
0/57
|
|
Blood and lymphatic system disorders
Lymphopenia
|
5.6%
3/54
|
1.8%
1/57
|
0.00%
0/57
|
|
Gastrointestinal disorders
Constipation
|
3.7%
2/54
|
7.0%
4/57
|
1.8%
1/57
|
|
Gastrointestinal disorders
Dental caries
|
5.6%
3/54
|
5.3%
3/57
|
1.8%
1/57
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
6/54
|
5.3%
3/57
|
5.3%
3/57
|
|
Gastrointestinal disorders
Gastritis
|
5.6%
3/54
|
3.5%
2/57
|
0.00%
0/57
|
|
Gastrointestinal disorders
Nausea
|
9.3%
5/54
|
7.0%
4/57
|
3.5%
2/57
|
|
General disorders
Pyrexia
|
3.7%
2/54
|
3.5%
2/57
|
7.0%
4/57
|
|
Infections and infestations
Bronchitis
|
5.6%
3/54
|
1.8%
1/57
|
0.00%
0/57
|
|
Infections and infestations
Nasopharyngitis
|
38.9%
21/54
|
42.1%
24/57
|
33.3%
19/57
|
|
Infections and infestations
Pharyngitis
|
5.6%
3/54
|
5.3%
3/57
|
5.3%
3/57
|
|
Infections and infestations
Tinea pedis
|
1.9%
1/54
|
5.3%
3/57
|
3.5%
2/57
|
|
Investigations
Liver function test abnormal
|
33.3%
18/54
|
21.1%
12/57
|
5.3%
3/57
|
|
Nervous system disorders
Dizziness
|
5.6%
3/54
|
7.0%
4/57
|
1.8%
1/57
|
|
Nervous system disorders
Headache
|
9.3%
5/54
|
8.8%
5/57
|
7.0%
4/57
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.7%
2/54
|
3.5%
2/57
|
5.3%
3/57
|
Additional Information
Study Director
Novartis Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER