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AzTrials

Primary Progressive Multiple SclerosisPhase 3INFORMSNCT00731692

This Was an Open-label, Single-arm Extension Study (CFTY720D2306E1) to a Double-blind, Randomized Multicenter, Placebo-controlled, Parallel-group Core Study (CFTY720D2306) in PPMS.

A Phase 3 study testing FTY720 in people with Primary Progressive Multiple Sclerosis, run by Novartis Pharmaceuticals at 157 sites in 18 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this study is to evaluate whether FTY720 is effective in delaying MS disability progression compared to placebo in patients with PPMS. This was an open-label, single-arm extension study to a double-blind, randomized multicenter, placebo-controlled, parallel-group core study. Read more about this study

Who is it for?
Ages 25 to 65 years
Conditions the study is for
Primary Progressive Multiple Sclerosis
What is being tested?
FTY720
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
970 participants
Where
157 sitesin 18 countries
Study dates
28 Jul 2008to 22 Jun 2015
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 25 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • General
  • sign written informed consent prior to participating in the study
  • 25 through 65 years of age inclusive
  • females of childbearing potential must:
  • have a negative pregnancy test at Baseline (prior to randomization) and
  • use simultaneously two forms of effective contraception during the treatment and 3-months after discontinuation of study medication
Show all 17Show fewer
  • Primary Progressive Multiple sclerosis.
  • diagnosis of primary progressive multiple sclerosis (according to the 2005 Revised McDonald criteria):
  • time since first reported symptoms between 2 and 10 years
  • evidence of clinical disability progression in the 2 years prior to Screening
  • disability status at Screening
  • EDSS score of 3.5-6.0 inclusive
  • pyramidal functional system score of 2 or more
  • 25'TWT less than 30 seconds
  • Extension study Inclusion criteria
  • Patients initially randomized to fingolimod 1.25 mg or placebo as part of the first study cohort, were to have completed at least 3 years on study drug treatment at the time of extension study initiation.
  • Patients initially randomized to fingolimod 0.5 mg or placebo as part of the second study cohort, were to have continued on study drug treatment until such time as the last ongoing patient enrolled in the study had reached 3 years in study

Who cannot join

  • PPMS specific:
  • History of relapses/attacks
  • Progressive neurological disorder other than PPMS
  • Pure cerebellar syndrome or pure visual progressive syndrome or pure
  • cognitive progressive syndrome
  • Presence of spinal cord compression at screening MRI
Show all 57Show fewer
  • Relevant history of vitamin B12 deficit
  • Evidence of syphilis or borreliosis at Screening
  • Cardiovascular conditions:
  • Myocardial infarction within the past 6 months or current unstable ischemic heart disease
  • History of angina pectoris due to coronary spasm or history of Raynaud's phenomenon
  • Severe cardiac failure or cardiac arrest
  • History of symptomatic bradycardia
  • Resting pulse <55 bpm pre-dose
  • History of sick sinus syndrome or sino-atrial heart block
  • History or presence of second and third degree AV block or an increase QT interval (QTc>440 ms)
  • Arrythmia requiring treatment with class III antiarrythmic drugs
  • History of positive tilt test from workout of vasovagal syncope
  • Hypertension, not controlled with medication
  • Pulmonary:
  • Severe respiratory disease or pulmonary fibrosis
  • TB
  • Abnormal X-ray, suggestive of active pulmonary disease
  • Abnormal PFT: <70% of predicted for FEV1 and FVC; <60% for DLCO
  • Patients receiving chronic (daily) therapies for asthma
  • Hepatic:
  • Known history of alcohol abuse, chronic liver or biliary disease
  • Total or conjugated Brb >ULN, unless in context of Gilbert's syndrome
  • AP >1.5xULN; ALT/AST >2xULN; GGT>3xULN
  • Other:
  • History of chronic disease of the immune system other than MS
  • Malignancy (other than successfully treated SCC or BCC)
  • Diabetes Mellitus
  • Macular Edema present at screening
  • HIV, Hepatitis C or B, other active infection
  • History of total lymphoid irradiation or bone marrow transplantation
  • Serum creatinine >1.7 mg/dl
  • WBC <3500 cells/mm3
  • Lymphocyte count <800 cells/mm3
  • History of substance abuse or any other factor that may interfere with subject ability to cooperate and comply with the study procedures
  • Unable to undergo MRI scans
  • Participation in any therapeutical clinical research study in the 6 months prior to randomization
  • Pregnant or lactating women
  • Drugs requiring wash-out period:
  • 3 months:
  • Systemic corticosteroids or ACTH
  • INF-beta
  • 6 months:
  • Immunosuppressive medication
  • Immunoglobulins
  • Monoclonal antibodies
  • Drugs that exclude participation in the study:
  • Cladribine
  • Cyclophosphamide
  • Mitoxantrone (except: patients who received a cumulative dose of no more than 60mg/m2 more than 5 years ago could enter the study)
  • Extension study Exclusion criteria
  • -Patients were not eligible for enrollment in the extension study if they had any of the following key exclusion criteria at the extension study Baseline visit: active chronic immune system disease other than MS (or stable disease treated with immune therapy); known immunodeficiency syndrome; active infection; uncontrolled diabetes mellitus; macularedema; treatment with Class Ia or III antiarrhythmic drugs; any of the specified cardiac, pulmonary, or hepatic conditions; or any medically unstable condition

Trial locations and contacts

157 sites in 18 countries.

157 sites in 18 countries

United States33 sites

  • Novartis Investigative Site

    Atlanta, Georgia, 30302

  • Novartis Investigative Site

    Aurora, Colorado, 80045

  • Novartis Investigative Site

    Baltimore, Maryland, 21287

  • Novartis Investigative Site

    Boston, Massachusetts, 02135

  • Novartis Investigative Site

    Brookline, Massachusetts, 02445

  • Novartis Investigative Site

    Buffalo, New York, 14203

  • Novartis Investigative Site

    Burlington, Vermont, 05401

  • Novartis Investigative Site

    Charlotte, North Carolina, 28207

  • Novartis Investigative Site

    Charlottesville, Virginia, 22904

  • Novartis Investigative Site

    Chicago, Illinois, 60611

  • Novartis Investigative Site

    Chicago, Illinois, 60637

  • Novartis Investigative Site

    Cleveland, Ohio, 44195

  • Novartis Investigative Site

    Columbus, Ohio, 43221

  • Novartis Investigative Site

    Dallas, Texas, 75390-9034

  • Novartis Investigative Site

    Detroit, Michigan, 48201

  • Novartis Investigative Site

    Durham, North Carolina, 27705

  • Novartis Investigative Site

    Houston, Texas, 77030

  • Novartis Investigative Site

    Kansas City, Kansas, 66160

  • Novartis Investigative Site

    Knoxville, Tennessee, 37934

  • Novartis Investigative Site

    Madison, Wisconsin, 53792

  • Novartis Investigative Site

    Nashville, Tennessee, 37212

  • Novartis Investigative Site

    New York, New York, 10029

  • Novartis Investigative Site

    Newport Beach, California, 92660

  • Novartis Investigative Site

    Pittsburgh, Pennsylvania, 15213

  • Novartis Investigative Site

    Pompano Beach, Florida, 33060

  • Novartis Investigative Site

    Rochester, New York, 14642

  • Novartis Investigative Site

    Sacramento, California, 95817

  • Novartis Investigative Site

    San Antonio, Texas, 78229

  • Novartis Investigative Site

    Seattle, Washington, 98122

  • Novartis Investigative Site

    St Louis, Missouri, 63110

  • Novartis Investigative Site

    Stony Brook, New York, 11794

  • Novartis Investigative Site

    Tampa, Florida, 33609

  • Novartis Investigative Site

    Teaneck, New Jersey, 07666

Other trials in United States

Germany17 sites

  • Novartis Investigative Site

    Berlin, 10713

  • Novartis Investigative Site

    Berlin, 13347

  • Novartis Investigative Site

    Bochum, 44791

  • Novartis Investigative Site

    Dresden, 01307

  • Novartis Investigative Site

    Düsseldorf, D-40225

  • Novartis Investigative Site

    Erlangen, 91054

  • Novartis Investigative Site

    Essen, 45147

  • Novartis Investigative Site

    Freiburg im Breisgau, 79106

  • Novartis Investigative Site

    Hanover, 30625

  • Novartis Investigative Site

    Hennigsdorf, 16761

  • Novartis Investigative Site

    Magdeburg, 39120

  • Novartis Investigative Site

    München, 81377

  • Novartis Investigative Site

    München, 81675

  • Novartis Investigative Site

    Münster, 48149

  • Novartis Investigative Site

    Teupitz, 15755

  • Novartis Investigative Site

    Trier, 54292

  • Novartis Investigative Site

    Würzburg, 97080

Other trials in Germany

Canada13 sites

  • Novartis Investigative Site

    Burnaby, British Columbia, V5G 2X6

  • Novartis Investigative Site

    Calgary, Alberta, T2N 2T9

  • Novartis Investigative Site

    Edmonton, Alberta, T6R 2B7

  • Novartis Investigative Site

    Gatineau, Quebec, J9J 0A5

  • Novartis Investigative Site

    Greenfield Park, Quebec, J4V 2J2

  • Novartis Investigative Site

    Halifax, Nova Scotia, B3H 4K4

  • Novartis Investigative Site

    Montreal, Quebec, H2L 4M1

  • Novartis Investigative Site

    Montreal, Quebec, H3A 2B4

  • Novartis Investigative Site

    Ottawa, Ontario, K1H 8L6

  • Novartis Investigative Site

    Regina, Saskatchewan, S4T 1A5

  • Novartis Investigative Site

    Toronto, Ontario, M4N 3M5

  • Novartis Investigative Site

    Toronto, Ontario, M5B 1W8

  • Novartis Investigative Site

    Vancouver, British Columbia, V6T 2B5

Other trials in Canada

Italy13 sites

  • Novartis Investigative Site

    Bari, BA, 70124

  • Novartis Investigative Site

    Catania, CT, 95123

  • Novartis Investigative Site

    Cefalù, PA, 90015

  • Novartis Investigative Site

    Chieti, CH, 66100

  • Novartis Investigative Site

    Gallarate, VA, 21013

  • Novartis Investigative Site

    Genova, GE, 16132

  • Novartis Investigative Site

    Milan, MI, 20122

  • Novartis Investigative Site

    Milan, MI, 20132

  • Novartis Investigative Site

    Montichiari, BS, 25018

  • Novartis Investigative Site

    Orbassano, TO, 10043

  • Novartis Investigative Site

    Padova, PD, 35128

  • Novartis Investigative Site

    Roma, RM, 00133

  • Novartis Investigative Site

    Roma, RM, 00189

Other trials in Italy

Spain12 sites

  • Novartis Investigative Site

    Badalona, Catalonia, 08916

  • Novartis Investigative Site

    Barcelona, Catalonia, 08035

  • Novartis Investigative Site

    Barcelona, Catalonia, 08036

  • Novartis Investigative Site

    Bilbao, Basque Country, 48013

  • Novartis Investigative Site

    Girona, Catalonia, 17007

  • Novartis Investigative Site

    L'Hospitalet de Llobregat, Catalonia, 08907

  • Novartis Investigative Site

    Lleida, Catalonia, 25198

  • Novartis Investigative Site

    Madrid, 28040

  • Novartis Investigative Site

    Madrid, 28046

  • Novartis Investigative Site

    Majadahonda, Madrid, 28222

  • Novartis Investigative Site

    Seville, Andalusia, 41009

  • Novartis Investigative Site

    Valencia, Valencia, 46026

Other trials in Spain

United Kingdom9 sites

  • Novartis Investigative Site

    Bristol, BS10 5NB

  • Novartis Investigative Site

    London, EC1A 7BE

  • Novartis Investigative Site

    London, NW1 2BU

  • Novartis Investigative Site

    London, SE5 9RS

  • Novartis Investigative Site

    London, W8 6RF

  • Novartis Investigative Site

    Newcastle upon Tyne, NE1 4LP

  • Novartis Investigative Site

    Norwich, NR4 7UY

  • Novartis Investigative Site

    Salford, Manchester, M6 8HD

  • Novartis Investigative Site

    Sheffield, South Yorkshire, S10 2JF

Other trials in United Kingdom

France8 sites

  • Novartis Investigative Site

    Bordeaux, F-33076

  • Novartis Investigative Site

    Lille, 59037

  • Novartis Investigative Site

    Marseille, 13385

  • Novartis Investigative Site

    Montpellier, 34295

  • Novartis Investigative Site

    Nantes, 44093

  • Novartis Investigative Site

    Paris, 75651

  • Novartis Investigative Site

    Rennes, 35043

  • Novartis Investigative Site

    Strasbourg, 67091

Other trials in France

Czechia7 sites

  • Novartis Investigative Site

    Brno, Czech Republic, 656 91

  • Novartis Investigative Site

    Olomouc, CZE, 775 20

  • Novartis Investigative Site

    Ostrava-Poruba, 708 00

  • Novartis Investigative Site

    Pilsen, 301 00

  • Novartis Investigative Site

    Prague, 128 08

  • Novartis Investigative Site

    Rychnov nad Kněžnou, Czech Republic, 516 01

  • Novartis Investigative Site

    Teplice, 415 29

Other trials in Czechia

Australia6 sites

  • Novartis Investigative Site

    Box Hill, Victoria, 3128

  • Novartis Investigative Site

    Camperdown, New South Wales, 2050

  • Novartis Investigative Site

    Heidelberg, Victoria, 3084

  • Novartis Investigative Site

    Hobart, Tasmania, 7000

  • Novartis Investigative Site

    Liverpool, New South Wales, 2170

  • Novartis Investigative Site

    Parkville, Victoria, 3050

Other trials in Australia

Belgium6 sites

  • Novartis Investigative Site

    Charleroi, 6000

  • Novartis Investigative Site

    Edegem, 2650

  • Novartis Investigative Site

    Leuven, 3000

  • Novartis Investigative Site

    Liège, 4000

  • Novartis Investigative Site

    Melsbroek, 1820

  • Novartis Investigative Site

    Sint-Truiden, 3800

Other trials in Belgium

Hungary6 sites

  • Novartis Investigative Site

    Budapest, 1076

  • Novartis Investigative Site

    Budapest, 1145

  • Novartis Investigative Site

    Debrecen, 4032

  • Novartis Investigative Site

    Győr, 9024

  • Novartis Investigative Site

    Miskolc, 3526

  • Novartis Investigative Site

    Veszprém, H-8200

Other trials in Hungary

Netherlands6 sites

  • Novartis Investigative Site

    Amsterdam, 1105 AZ

  • Novartis Investigative Site

    Breda, CK, 4818

  • Novartis Investigative Site

    Eindhoven, 5623 EJ

  • Novartis Investigative Site

    Nieuwegein, 3435 CM

  • Novartis Investigative Site

    Nijmegen, 6525 GC

  • Novartis Investigative Site

    Sittard-Geleen, 6162 BG

Other trials in Netherlands

Switzerland5 sites

  • Novartis Investigative Site

    Basel, 4031

  • Novartis Investigative Site

    Bern, 3010

  • Novartis Investigative Site

    Lausanne, 1011

  • Novartis Investigative Site

    Lugano, 6900

  • Novartis Investigative Site

    Zurich, 8091

Other trials in Switzerland

Turkey (Türkiye)5 sites

  • Novartis Investigative Site

    Ankara, 06100

  • Novartis Investigative Site

    Atakum / Samsun, 55139

  • Novartis Investigative Site

    Balcova / Izmir, 35340

  • Novartis Investigative Site

    Istanbul, 34093

  • Novartis Investigative Site

    Yenisehir / Izmir

Other trials in Turkey (Türkiye)

Poland4 sites

  • Novartis Investigative Site

    Lodz, 90-153

  • Novartis Investigative Site

    Lublin, 20-954

  • Novartis Investigative Site

    Warsaw, 02-097

  • Novartis Investigative Site

    Warsaw, 02-957

Other trials in Poland

Finland3 sites

  • Novartis Investigative Site

    Helsinki, 00930

  • Novartis Investigative Site

    Tampere, 33100

  • Novartis Investigative Site

    Turku, 20520

Other trials in Finland

Denmark2 sites

  • Novartis Investigative Site

    Aarhus, 8000 C

  • Novartis Investigative Site

    Sønderborg, 6400

Other trials in Denmark

Sweden2 sites

  • Novartis Investigative Site

    Gothenburg, 413 45

  • Novartis Investigative Site

    Stockholm, 17176

Other trials in Sweden

About this study

Official title: A Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel-group Study Comparing the Efficacy and Safety of 0.5mg Fingolimod Administered Orally Once Daily Versus Placebo in Patients With Primary Progressive Multiple Sclerosis and An Open-label, Single-arm Extension Study to the Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel-group Study Comparing the Efficacy and Safety of0.5 mg FTY720 Administered Orally Once Daily Versus Placebo in Patients With Primary Progressive Multiple Sclerosis

The purpose of this study is to evaluate whether FTY720 is effective in delaying MS disability progression compared to placebo in patients with PPMS. This was an open-label, single-arm extension study to a double-blind, randomized multicenter, placebo-controlled, parallel-group core study. The core study completed and eligible patients enrolled into the extension study at the next scheduled or unscheduled core study visit. All patients, regardless of their treatment in the core study, received fingolimod 0.5 mg in the extension study. The extension study was terminated early after the results of the core study became available showing that the study did not meet its primary endpoint which was defined as confirmed disability progression in this population

Being tested
FTY720, Placebo
Keywords
FTY720, primary progressive multiple sclerosis,PPMS
Medical subject headings
Multiple Sclerosis, Chronic Progressive

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FTY720Drug

    Fingolimod capsules at doses of 1.25 mg (prior to implementation of Amendment 5) and 0.5 mg (after Amendment 5) were administered orally once daily

  • PlaceboDrug

    Matching placebo capsules were administered orally once daily

Study groups in NCT00731692
Study groupRoleWhat they receive
FTY720D 0.5 mgExperimentalCohort 2: The 0.5 mg group consists of patients who were directly randomized to fingolimod 0.5 mg (i.e. AFTER the amendment
Receives: Drug: FTY720
PlaceboPlacebo comparatorCohort 1 and 2: Patients randomized to placebo continued on placebo after re-randomization
Receives: Drug: Placebo
FTY720D 1.25 mg switch to 0.5 mgExperimentalCohort 1: fingolimod 1.25 group consists of patients who were initially randomized to fingolimod 1.25 mg and switched to fingolimod 0.5 mg after amendment on Nov 2009
Receives: Drug: FTY720

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Kaplan-Meier Estimate of the Risk of 3-month Confirmed Disability Progression Based on Composite Endpoint

    3-month sustained increase from Baseline in EDSS (at least 1 point increase from Baseline for patients with a Baseline value of 5 or less or at least 0.5 point increase from Baseline for patients with a Baseline value of 5.5 or more) or 3-month sustained increase of at least 20% from BL in the time taken to complete the timed 25-foot walk test (25' TWT); or 3-month sustained increase of at least 20% from BL in the time taken to complete the 9-HPT. The 25' TWT is a quantitative measure of lower extremity function. The EDSS is a scale assessing neurologic impairment, including a series of scores in each of 8 functional systems: Visual, Brain Stem, Pyramidal, Cerebellar, Sensory, Bowel and Bladder, Cerebral and Other functions. The score ranges from 0 (normal) to 10 (death due to MS)). The 9-hole peg test (9-HPT) is a quantitative measure of upper extremity (arm and hand) function.

    Measured: up to 36 months after the last patient was randomized

Secondary outcomes (14)

  1. Kaplan-Meier Estimate of the Risk of 3- Month Confirmed Disability Progression Based on Expanded Disability Status Scale (EDSS)

    The Expanded Disability Status Scale (EDSS) is a scale for assessing neurologic impairment in MS (Kurtzke 1983) and includes a series of scores in each of 8 functional systems and the EDSS steps (ranging from 0 (normal) to 10 (death due to MS)). The functional systems are Visual, Brain Stem, Pyramidal, Cerebellar, Sensory, Bowel and Bladder, Cerebral and Other functions. Fatigue is not included in the Cerebral score of the EDSS. The score ranges from 0 (normal) to 10 (death due to MS)

    Measured: up to 36 months after the last patient was randomized

  2. Percent Change From Baseline in Brain Volume at Month 36

    The percent change from Baseline in brain volume was analyzed using a random coefficients model. The model included: 1) fixed effects: treatment and region and 2) continuous covariates: time, number of Gd enhancing lesions at Baseline, Baseline T2 volume, and normalized brain volume at Baseline. Time as a continuous covariate allowed for the estimation of different slopes and intercepts among treatment groups.

    Measured: Baseline to month 36

  3. Kaplan Meier Estimate -Percentage of Participants With 3- Month Confirmed Disability Progression Based on 9-HPT.

    The 9-HPT is a quantitative measure of upper extremity (arm and hand) function designed and validated for evaluation of MS patients. N= Total number of patients included in the analysis

    Measured: up to 36 months after the last patient was randomized

  4. Kaplan Meier Estimate -Percentage of Participants With 3- Month Confirmed Disability Progression Based on 25' TWT.

    The 25' TWT is a quantitative measure of lower extremity function designed and validated for evaluation of MS patients. N= Total number of patients included in the analysis

    Measured: up to 36 months after the last patient was randomized

  5. Number of New/Enlarging T2 Lesions Per Year Measured From Baseline to Month 36

    Inflammatory disease, as measured by number of new or newly-enlarging T2 lesions, was assessed by Magnetic resonance Imaging (MRI) scanning of the brain and full spinal cord. N= Total number of patients included in the analysis

    Measured: Baseline to 36 months

  6. Number of Gd-enhancing Lesions at Month 36

    Inflammatory disease, as measured by number of T1 Gd-enhancing lesions, was assessed by MRI scanning of the brain and full spinal cord. N= Total number of patients included in the analysis

    Measured: Baseline to 36 months

  7. Percent Change in Total T2 Lesion Volume From Baseline to Month 36

    Inflammatory disease as measured by percent change in total T2 lesion volume (mm3) was assessed by MRI. N= Total number of patients included in the analysis

    Measured: Baseline to month 36

  8. Change From Baseline in the Patient Reported Indices in Multiple Sclerosis (PRIMUS-QoL Score)

    The quality of life scale contains 22 items. Each item will be given a score of 1 or 0. A score of 1 (or 0) indicates the presence (or absence) of the symptom or adverse quality of life. All 22 item scores will be summed to obtain a total score ranging from 0 (good) to 22 (poor), which is the PRIMUS QoL scale score

    Measured: Baseline, 36 months

  9. Change From Baseline in PRIMUS-Activities

    The activities subscale of PRIMUS contains 15 items and each item is given a score of 0 (able to do on own without difficulties), 1 (able to do on own with difficulties), or 2 (unable to do on own). All 15 items were summed to obtain a total score ranging from 0 (good) to 30 (poor).

    Measured: Baseline, 36 months

  10. Change From Baseline in Unidimensional Fatigue Impact (U-FIS) Score

    Unidimensional Fatigue Impact Scale (U-FIS), contains 22 patient-reported items that assess the impact of fatigue on cognitive, physical, and psychosocial functioning. Responses formed a single unidimensional scale measuring fatigue impact. The U-FIS was calculated and analyzed according to the U-FIS scoring manual. The U-FIS scale contains 22 items with 5 possible outcomes for each item. Two response categories (about half the time and a lot of the time) were combined into 1 category to obtain 4 possible outcomes: 0 (never), 1 (a little of the time), 2 (about half the time/a lot of the time), and 3 (all the time). The 22 condensed item scores were summed to obtain a total score ranging from 0 (no fatigue) to 66 (severe fatigue impact).

    Measured: Baseline, 36 months

  11. Change From Baseline in European Quality of Life - 5 Dimensions (EQ-5D Score)

    EQ-5D: participant rated questionnaire to assess health-related quality of life in terms of a single utility score. Health State Profile component assesses level of current health for 5 domains: mobility, self care, usual activities, pain and discomfort, and anxiety and depression; 1 indicates better health state (no problems); 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQoL Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; higher score indicates a better health state.

    Measured: Baseline, 36 months

  12. Change From Baseline in Multiple Sclerosis Walking Scale (MSWS-12 Score)

    The Multiple Sclerosis Walking Scaleis a patient reported measure of walking quality (Hobart et al 2003), consisting of 12 items asking patients to rate the impact of MS upon their walking ability. Responses were captured on a 3-point scale ranging from 1 (Not at all) to 3 (A lot) for items 1 to 3 and on a 5-point scale ranging from 1 (not limited) to 5 (extremely) for items 4 to 12. All 12 item scores were summed to obtain a total score ranging from 12 (good) to 54 (poor) which is the MSWS-12 scale score. The total score was transformed to a 0 to 100 scale score. The MSWS-12 scale score will be transformed to a 0-100 scale score before any summaries or statistical analyses are performed. The transformed score is obtained by subtracting 12 and divided by 42 and multiplying by 100 (i.e., transformed scale score = (raw scale score- 12)/42*100).

    Measured: Baseline, 36 months

  13. Blood Concentrations of Fingolimod and Fingolimod-phosphate

    Concentrations of fingolimod and fingolimod-phosphate in whole blood were determined by validated liquid chromatography methods with tandem mass spectrometry. The lower limits of quantification were 0.08 ng/ml for fingolimod and 0.1 ng/ml for fingolimod-phosphate.

    Venous blood samples were collected for the analysis.

    Measured: Month 3 up to 36 months

  14. Change in MSFC Z-score and Subscale Scores From Baseline to Month 36

    The Multiple Sclerosis Functional Composite (MSFC) is a multidimensional clinical outcome measure that includes quantitative tests of leg function/ambulation (Timed 25-Foot Walk), arm function (9-Hole Peg Test), and cognitive function (Paced Auditory Serial Addition Test). The overall MSFC z-score as an average of the three standardized scores derived using baseline data pooled over each treatment arm as reference population. Higher scores reflect better neurological function and a positive change from Baseline indicates improvement.

    Measured: Baseline to Month 36

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Aug 2008 (estimated)
  2. Study start28 Jul 2008
  3. Primary completion22 Jun 2015
  4. Study completion22 Jun 2015

Why it stopped: The extension study was terminated early after the results of the core study showed the study did not meet primary endpoint; confirmed disability progression

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
970 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00731692
Sponsor's protocol ID
CFTY720D2306
Other IDs
2007-002627-32
Acronym
INFORMS
Status last verified
May 2017
First submitted
7 Aug 2008
Last updated
14 Jun 2017
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

  • Calvi A, Mendelsohn Z, Hamed W, Chard D, Tur C, Stutters J, MacManus D, Kanber B, Wheeler-Kingshott CAMG, Barkhof F, Prados F. Treatment reduces the incidence of newly appearing multiple sclerosis lesions evolving into chronic active, slowly expanding lesions: A retrospective analysis. Eur J Neurol. 2024 Jan;31(1):e16092. doi: 10.1111/ene.16092. Epub 2023 Oct 12.

    PubMed 37823722

  • Leppert D, Kropshofer H, Haring DA, Dahlke F, Patil A, Meinert R, Tomic D, Kappos L, Kuhle J. Blood Neurofilament Light in Progressive Multiple Sclerosis: Post Hoc Analysis of 2 Randomized Controlled Trials. Neurology. 2022 May 24;98(21):e2120-e2131. doi: 10.1212/WNL.0000000000200258. Epub 2022 Apr 4.

    PubMed 35379762

  • Koch MW, Mostert J, Repovic P, Bowen JD, Strijbis E, Uitdehaag B, Cutter G. Smoking, obesity, and disability worsening in PPMS: an analysis of the INFORMS original trial dataset. J Neurol. 2022 Mar;269(3):1663-1669. doi: 10.1007/s00415-021-10750-z. Epub 2021 Aug 15.

    PubMed 34392376

  • Koch MW, Mostert J, Zhang Y, Wolinsky JS, Lublin FD, Strijbis E, Cutter G. Association of Age With Contrast-Enhancing Lesions Across the Multiple Sclerosis Disease Spectrum. Neurology. 2021 Sep 28;97(13):e1334-e1342. doi: 10.1212/WNL.0000000000012603. Epub 2021 Aug 10.

    PubMed 34376508

  • Miller DH, Lublin FD, Sormani MP, Kappos L, Yaldizli O, Freedman MS, Cree BAC, Weiner HL, Lubetzki C, Hartung HP, Montalban X, Uitdehaag BMJ, MacManus DG, Yousry TA, Gandini Wheeler-Kingshott CAM, Li B, Putzki N, Merschhemke M, Haring DA, Wolinsky JS. Brain atrophy and disability worsening in primary progressive multiple sclerosis: insights from the INFORMS study. Ann Clin Transl Neurol. 2018 Jan 30;5(3):346-356. doi: 10.1002/acn3.534. eCollection 2018 Mar.

    PubMed 29560379

  • Lublin F, Miller DH, Freedman MS, Cree BAC, Wolinsky JS, Weiner H, Lubetzki C, Hartung HP, Montalban X, Uitdehaag BMJ, Merschhemke M, Li B, Putzki N, Liu FC, Haring DA, Kappos L; INFORMS study investigators. Oral fingolimod in primary progressive multiple sclerosis (INFORMS): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet. 2016 Mar 12;387(10023):1075-1084. doi: 10.1016/S0140-6736(15)01314-8. Epub 2016 Jan 28.

    PubMed 26827074

  • Hartung HP, Aktas O. Bleak prospects for primary progressive multiple sclerosis therapy: downs and downs, but a glimmer of hope. Ann Neurol. 2009 Oct;66(4):429-32. doi: 10.1002/ana.21880. No abstract available.

    PubMed 19847907

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing FTY720 in people with Primary Progressive Multiple Sclerosis, run by Novartis Pharmaceuticals at 157 sites in 18 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 57 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 14 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 157 sites in United States, Germany, Canada, Italy, Spain and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Jun 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Kaplan-Meier Estimate of the Risk of 3-month Confirmed Disability Progression Based on Composite Endpoint (time frame: up to 36 months after the last patient was randomized). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00731692, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jun 2017.

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