Trial Outcomes & Findings for A 12 -Month, Open-label, Multi-center Study to Explore the Health Outcomes of FTY720 (NCT NCT01578330)

NCT ID: NCT01578330

Last Updated: 2016-06-07

Results Overview

The Treatment Satisfaction Questionnaire for Medication (TSQM-9) is a psychometric measure of a patient's satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The scores were computed by adding items for each domain, i.e. 1 to 3 for effectiveness, 4 - 6 for convenience and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) X 3 items = 18 for the effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. A positive change from baseline indicates improvement.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 42 participants
• Primary outcome timeframe: Baseline and month 12
• Results posted on: 2016-06-07

Participant Flow

Participant milestones

Measure	Fingolimod, FTY720 Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Overall Study STARTED	42
Overall Study Per Protocol Population	35
Overall Study COMPLETED	35
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	Fingolimod, FTY720 Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Overall Study Adverse Event	2
Overall Study Protocol Violation	5

Baseline Characteristics

A 12 -Month, Open-label, Multi-center Study to Explore the Health Outcomes of FTY720

Baseline characteristics by cohort

Measure	Fingolimod, FTY720 n=42 Participants Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Age, Continuous	35.7 Years STANDARD_DEVIATION 9.4 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and month 12

Population: Per Protocol Population: included all patients who had evaluable data for the primary variable from first to last visit

The Treatment Satisfaction Questionnaire for Medication (TSQM-9) is a psychometric measure of a patient's satisfaction with medication. It consists of 3 subscales: effectiveness, convenience and global satisfaction. The scores were computed by adding items for each domain, i.e. 1 to 3 for effectiveness, 4 - 6 for convenience and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) X 3 items = 18 for the effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. A positive change from baseline indicates improvement.

Outcome measures

Measure	Fingolimod, FTY720 n=35 Participants Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Mean Patient-Reported Treatment Satisfaction Questionnaire for Medication Scores (TSQM-9) Baseline (n=34)	32.0 Scores on a scale Standard Deviation 9.9
Mean Patient-Reported Treatment Satisfaction Questionnaire for Medication Scores (TSQM-9) Month 12 (n=32)	44.7 Scores on a scale Standard Deviation 9.9

SECONDARY outcome

Timeframe: Month 1

Population: Per Protocol Population: included all patients who had evaluable data for the primary variable from first to last visit

The SF-36v2 is a validated health-related quality of life instrument used in numerous disease states, including MS. It is a self-administered survey that measures 8 domains of health including: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems and general mental health. Additionally, two summary scale scores can be calculated: the Physical Component Summary (PCS) and the Mental Component Summary (MCS). If half or more questions within a domain were answered, then a score was calculated for that domain. Otherwise, the patient score for that domain was set to missing. If the patient was missing any 1 of the 8 scale scores, then the physical and mental component scores were set to missing. An algorithm was used to create a score from 0 to 100 for each domain score and component score. A positive change from baseline indicates improvement.

Outcome measures

Measure	Fingolimod, FTY720 n=35 Participants Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Function	59.7 score on scale Standard Deviation 30.9
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Role	53.6 score on scale Standard Deviation 43.8
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Pain	61.7 score on scale Standard Deviation 23.7
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). General Health	47.8 score on scale Standard Deviation 20.5
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Vitality	28.9 score on scale Standard Deviation 23.1
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Social Function	61.8 score on scale Standard Deviation 26.4
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Emotional Role	48.4 score on scale Standard Deviation 43.0
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Mental Health	31.5 score on scale Standard Deviation 17.0

SECONDARY outcome

Timeframe: Month 6

Population: Per Protocol Population: included all patients who had evaluable data for the primary variable from first to last visit

The SF-36v2 is a validated health-related quality of life instrument used in numerous disease states, including MS. It is a self-administered survey that measures 8 domains of health including: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems and general mental health. Additionally, two summary scale scores can be calculated: the Physical Component Summary (PCS) and the Mental Component Summary (MCS). If half or more questions within a domain were answered, then a score was calculated for that domain. Otherwise, the patient score for that domain was set to missing. If the patient was missing any 1 of the 8 scale scores, then the physical and mental component scores were set to missing. An algorithm was used to create a score from 0 to 100 for each domain score and component score. A positive change from baseline indicates improvement.

Outcome measures

Measure	Fingolimod, FTY720 n=35 Participants Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Function (n=33)	57.9 score on scale Standard Deviation 31.1
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Role (n=34)	47.8 score on scale Standard Deviation 46.0
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Pain (n=34)	62.6 score on scale Standard Deviation 24.8
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). General Health (n=33)	48.5 score on scale Standard Deviation 20.3
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Vitality (n=34)	35.2 score on scale Standard Deviation 22.9
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Social Function (n=34)	60.6 score on scale Standard Deviation 29.9
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Emotional Role (n=34)	54.8 score on scale Standard Deviation 43.3
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Mental Health (n=34)	36.5 score on scale Standard Deviation 20.3

SECONDARY outcome

Timeframe: Month 12

Population: Per Protocol Population: included all patients who had evaluable data for the primary variable from first to last visit

The SF-36v2 is a validated health-related quality of life instrument used in numerous disease states, including MS. It is a self-administered survey that measures 8 domains of health including: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems and general mental health. Additionally, two summary scale scores can be calculated: the Physical Component Summary (PCS) and the Mental Component Summary (MCS). If half or more questions within a domain were answered, then a score was calculated for that domain. Otherwise, the patient score for that domain was set to missing. If the patient was missing any 1 of the 8 scale scores, then the physical and mental component scores were set to missing. An algorithm was used to create a score from 0 to 100 for each domain score and component score. A positive change from baseline indicates improvement.

Outcome measures

Measure	Fingolimod, FTY720 n=35 Participants Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Function (n=31)	59.5 score on scale Standard Deviation 34.3
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Physical Role (n=32)	46.9 score on scale Standard Deviation 44.7
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Pain (n=32)	65.9 score on scale Standard Deviation 23.3
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). General Health (n=31)	50.8 score on scale Standard Deviation 21.9
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Vitality (n=32)	33.1 score on scale Standard Deviation 20.0
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Social Function (n=32)	65.8 score on scale Standard Deviation 29.1
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Emotional Role (n=31)	52.7 score on scale Standard Deviation 46.2
Mean Patient-reported Health-related Quality-of-life With Fingolimod (Short Form Health Survey: SF-36). Mental Health (n=32)	36.3 score on scale Standard Deviation 20.3

Adverse Events

Fingolimod, FTY720

Serious events: 4 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Measure	Fingolimod, FTY720 n=42 participants at risk Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Immune system disorders Multiple Sclerosis aggravated	2.4% 1/42
Injury, poisoning and procedural complications Fracture	2.4% 1/42
Injury, poisoning and procedural complications Medication taken before pregnancy	2.4% 1/42
Surgical and medical procedures Middle ear infection surgery	2.4% 1/42

Other adverse events

Measure	Fingolimod, FTY720 n=42 participants at risk Patients received fingolimod 0.5 mg oral capsules daily with or without food.
Renal and urinary disorders Urinary infection	21.4% 9/42
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	19.0% 8/42
Hepatobiliary disorders Increase of hepatic enzymes	11.9% 5/42
Blood and lymphatic system disorders Lymphopenia	16.7% 7/42
Immune system disorders Multiple Sclerosis Aggravated	9.5% 4/42
Nervous system disorders Headache	9.5% 4/42
Metabolism and nutrition disorders Hyperlipidemia	9.5% 4/42
Infections and infestations Tooth abscess	7.1% 3/42
Gastrointestinal disorders Nausea	7.1% 3/42
Renal and urinary disorders Incontinence	7.1% 3/42

Additional Information

Study Director

Novartis

Phone: 862-778-8300

Email: trialandresults.registries@novartis.com

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 or disclosure of trial results in their entirety
• Publication restrictions are in place

Restriction type: OTHER