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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01625182




Registration number
NCT01625182
Ethics application status
Date submitted
19/06/2012
Date registered
21/06/2012
Date last updated
30/10/2017

Titles & IDs
Public title
Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.
Scientific title
A Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Fingolimod 0.5 mg Administered Orally Once Daily Versus Placebo in Patients With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Secondary ID [1] 0 0
2011-005280-24
Secondary ID [2] 0 0
CFTY720I2201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Inflammatory Demyelinating Polyradiculoneuropathy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Fingolimod
Treatment: Drugs - Placebo Comparator

Experimental: Fingolimod (FTY720) - Participants received Fingolimod 0.5 mg orally once daily.

Placebo Comparator: Placebo - Participants received matching placebo to Fingolimod orally once daily.


Treatment: Drugs: Fingolimod
Fingolimod 0.5 mg capsules

Treatment: Drugs: Placebo Comparator
Matching placebo capsules

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale - Confirmed worsening in CIDP was measured by the adjusted INCAT Disability Scale. The adjusted INCAT disability scale measures arm disability and leg disability. For arm disability the scale ranges from 0 (no upper limb problems) to 5 (inability to use either arm for any purposeful movement). The leg disability scale ranges from 0 (walking not affected) to 5 (restricted to wheelchair, unable to stand and walk a few steps with help). The total adjusted INCAT disability score is calculated by the sum of the arm and leg disability scores where the total score ranges from 0 to 10. A confirmed worsening was defined as an increase by 1 or more points on the adjusted INCAT disability scale from the value at baseline.
Timepoint [1] 0 0
Month 12
Secondary outcome [1] 0 0
Change From Baseline for Grip Strength, Dominant Hand - Grip strength measurements were done using a vigorimeter. With this device, the pressure in the bulb exercised by the participant was registered on a manometer via a rubber junction tube. Both the dominant and non-dominant hands were tested. A negative change from baseline indicates deterioration.
Timepoint [1] 0 0
baseline, Month 6, Month 12
Secondary outcome [2] 0 0
Change From Baseline for Grip Strength, Non-dominant Hand - Grip strength measurements were done using a vigorimeter. With this device, the pressure in the bulb exercised by the participant was registered on a manometer via a rubber junction tube. Both the dominant and non-dominant hands were tested. A negative change from baseline indicates deterioration.
Timepoint [2] 0 0
baseline, Month 6, Month 12
Secondary outcome [3] 0 0
Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS) - This questionnaire was constructed using the patients' perception of their ability to perform daily and social activities. The questionnaire comprises 24 items ranging from ability to read a book or newspaper (as the easiest item to accomplish) to ability to run (most difficult item to accomplish). The obtained raw summed score was translated subsequently to a convenient centile metric score ranging from 0 (most severe disability) to 100 (no disability at all). A higher score indicated a better health status. A negative change from baseline indicates deterioration.
Timepoint [3] 0 0
baseline, Month 6, Month 12

Eligibility
Key inclusion criteria
Inclusion Criteria

- written informed consent must be obtained before any assessment is performed

- The diagnosis of CIDP will use the definition of the EFNS/PNS Task Force First
Revision. Patients must either have a clinical diagnosis of CIDP fulfilling the
clinical inclusion criteria for typical CIDP or one of the following atypical forms of
CIDP: pure motor, or asymmetrical (MADSAM [Lewis-Sumner syndrome]), or IgA or IgG (not
IgM) MGUS paraprotein associated.

- All patients must also fulfill the clinical exclusion criteria and the definite
electrodiagnostic criteria of the EFNS/PNS Task Force First Revision.

- disability defined by an INCAT Disability Scale score of 1-9 or, if INCAT score is 0,
a documented history of disability sufficient to require treatment within the past 2
years following reduction or interruption of CIDP treatment

- receiving IVIg treatment (minimal dose equivalent to 0.4 g/kg every 4 weeks for a
minimum of 12 weeks) or corticosteroids (minimal dose equivalent to prednisone 10
mg/day) treatment prior to the screening visit

- history of documented clinically meaningful deterioration confirmed by clinical
examination during therapy or upon interruption or reduction of therapy within 18
months prior to Screening

- stable CIDP symptoms for the 6 weeks before randomization
Minimum age
18 Years
Maximum age
75 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

- other chronic demyelinating neuropathies, including: Distal Acquired Demyelinating
Symmetric Neuropathy (DADS) Multifocal Motor Neuropathy (MMN) pure sensory CIDP
hematopoietic malignancy except for MGUS

- conditions in which the pathogenesis of the neuropathy may be different from CIDP such
as: Lyme disease, POEMS syndrome, osteosclerotic myeloma, Castleman's disease

- treatment with plasma exchange within 2 months of randomization,
immunosuppressive/chemotherapeutic medications: azathioprine, cyclophosphamide,
cyclosporine, mycophenolate, etanercept, methotrexate tacrolimus or other
immunosuppressive drugs within 6 months of randomization or 5 half-lives (whichever is
later), Rituximab in the 2 years prior to randomization (patients that have received
rituximab between 1 and 2 years should have B-cell levels within normal range), other
cytotoxic immunosuppressive medications with sustained effects (including
mitoxantrone, alemtuzumab, cladribine) at any time, hematopoietic stem cell
transplantation at any time

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Sydney
Recruitment hospital [2] 0 0
Novartis Investigative Site - Auchenflower
Recruitment hospital [3] 0 0
Novartis Investigative Site - Fitzroy
Recruitment hospital [4] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
4066 - Auchenflower
Recruitment postcode(s) [3] 0 0
3065 - Fitzroy
Recruitment postcode(s) [4] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
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United States of America
State/province [2] 0 0
Florida
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United States of America
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Illinois
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United States of America
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Kentucky
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United States of America
State/province [5] 0 0
Massachusetts
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United States of America
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New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Vermont
Country [10] 0 0
Belgium
State/province [10] 0 0
Bruxelles
Country [11] 0 0
Belgium
State/province [11] 0 0
Leuven
Country [12] 0 0
Belgium
State/province [12] 0 0
Liege
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Canada
State/province [14] 0 0
Quebec
Country [15] 0 0
Canada
State/province [15] 0 0
Greenfield Park
Country [16] 0 0
Canada
State/province [16] 0 0
Montreal
Country [17] 0 0
Czechia
State/province [17] 0 0
Praha 5
Country [18] 0 0
France
State/province [18] 0 0
Limoges
Country [19] 0 0
France
State/province [19] 0 0
Marseille cedex 05
Country [20] 0 0
France
State/province [20] 0 0
Montpellier
Country [21] 0 0
France
State/province [21] 0 0
Paris
Country [22] 0 0
France
State/province [22] 0 0
Pessac Cedex
Country [23] 0 0
France
State/province [23] 0 0
Strasbourg
Country [24] 0 0
Germany
State/province [24] 0 0
Nordrhein-Westfalen
Country [25] 0 0
Germany
State/province [25] 0 0
Bochum
Country [26] 0 0
Germany
State/province [26] 0 0
Düsseldorf
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Germany
State/province [27] 0 0
Essen
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Germany
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Göttingen
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Greece
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Athens
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Greece
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Thessaloniki
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Israel
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Haifa
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Israel
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Ramat Gan
Country [33] 0 0
Israel
State/province [33] 0 0
Tel Aviv
Country [34] 0 0
Italy
State/province [34] 0 0
MI
Country [35] 0 0
Italy
State/province [35] 0 0
PA
Country [36] 0 0
Italy
State/province [36] 0 0
Ferrara
Country [37] 0 0
Italy
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Milano
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Italy
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Pisa
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Italy
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Rome
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Japan
State/province [40] 0 0
Aichi
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Japan
State/province [41] 0 0
Osaka
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Japan
State/province [42] 0 0
Tokyo
Country [43] 0 0
Japan
State/province [43] 0 0
Aomori
Country [44] 0 0
Japan
State/province [44] 0 0
Chiba
Country [45] 0 0
Netherlands
State/province [45] 0 0
Amsterdam
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Netherlands
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Maastricht
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Poland
State/province [47] 0 0
Gdansk
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Poland
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Katowice
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Poland
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Lodz
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Spain
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Cataluña
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Spain
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Madrid
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United Kingdom
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Oxfordshire
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United Kingdom
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Glasgow
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United Kingdom
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Liverpool
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United Kingdom
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London
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United Kingdom
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Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Mitsubishi Tanabe Pharma Corporation
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The study was designed to evaluate the efficacy and safety of fingolimod in the treatment of
chronic inflammatory demyelinating polyradiculoneuropathy compared with placebo.
Trial website
https://clinicaltrials.gov/show/NCT01625182
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications