Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.

September 28, 2017 updated by: Novartis Pharmaceuticals

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)

The study was designed to evaluate the efficacy and safety of fingolimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy compared with placebo.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This study was a double-blind, randomized, multicenter, placebo-controlled, parallel-group study in patients with a diagnosis of chronic inflammatory demyelinating polyradiculoneuropathy and treated with IVIg, corticosteroids, or both therapies prior to study entry. Patients meeting the eligibility criteria were randomly assigned in a ratio of 1:1 to receive oral fingolimod (0.5 mg/day) or matching placebo.

The study consisted of 3 periods: a Screening Period, a Double-blind Treatment Period and a Follow-up Period after discontinuation of study drug treatment. Patients who complete the study will have an option to enter an extension.

Study Type

Interventional

Enrollment (Actual)

106

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2050
        • Novartis Investigative Site
    • Queensland
      • Auchenflower, Queensland, Australia, 4066
        • Novartis Investigative Site
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • Novartis Investigative Site
      • Parkville, Victoria, Australia, 3050
        • Novartis Investigative Site
  • Belgium
      • Bruxelles, Belgium, 1200
        • Novartis Investigative Site
      • Leuven, Belgium, 3000
        • Novartis Investigative Site
      • Liege, Belgium, 4000
        • Novartis Investigative Site
  • Canada
      • Greenfield Park, Canada, J4V 2J2
        • Novartis Investigative Site
      • Montreal, Canada, H3A 2B4
        • Novartis Investigative Site
    • Ontario
      • Kingston, Ontario, Canada, K7L 2V7
        • Novartis Investigative Site
    • Quebec
      • Québec, Quebec, Canada, G1J 1Z4
        • Novartis Investigative Site
  • Czechia
      • Praha 5, Czechia, 150 06
        • Novartis Investigative Site
  • France
      • Limoges, France, 87042
        • Novartis Investigative Site
      • Marseille cedex 05, France, 13385
        • Novartis Investigative Site
      • Montpellier, France, 34295
        • Novartis Investigative Site
      • Paris, France, 75013
        • Novartis Investigative Site
      • Pessac Cedex, France, 33604
        • Novartis Investigative Site
      • Strasbourg, France, 67091
        • Novartis Investigative Site
  • Germany
      • Bochum, Germany, 44791
        • Novartis Investigative Site
      • Düsseldorf, Germany, 40225
        • Novartis Investigative Site
      • Essen, Germany, 45147
        • Novartis Investigative Site
      • Göttingen, Germany, 37075
        • Novartis Investigative Site
    • Nordrhein-Westfalen
      • Koeln, Nordrhein-Westfalen, Germany, 50937
        • Novartis Investigative Site
  • Greece
      • Athens, Greece, GR 151 25
        • Novartis Investigative Site
      • Thessaloniki, Greece, 546 36
        • Novartis Investigative Site
      • Thessaloniki, Greece, 57010
        • Novartis Investigative Site
  • Israel
      • Haifa, Israel
        • Novartis Investigative Site
      • Ramat Gan, Israel, 52621
        • Novartis Investigative Site
      • Tel Aviv, Israel, 64239
        • Novartis Investigative Site
  • Italy
      • Ferrara, Italy, 44100
        • Novartis Investigative Site
      • Milano, Italy
        • Novartis Investigative Site
      • Pisa, Italy, 56126
        • Novartis Investigative Site
      • Rome, Italy, 00168
        • Novartis Investigative Site
    • MI
      • Legnano, MI, Italy, 20025
        • Novartis Investigative Site
      • Rozzano, MI, Italy, 20089
        • Novartis Investigative Site
    • PA
      • Cefalù, PA, Italy, 90015
        • Novartis Investigative Site
  • Japan
      • Aomori, Japan, 030-8553
        • Novartis Investigative Site
      • Chiba, Japan, 260-8677
        • Novartis Investigative Site
    • Aichi
      • Nagoya, Aichi, Japan, 466-8560
        • Novartis Investigative Site
    • Osaka
      • Sayama, Osaka, Japan, 589-8511
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo, Tokyo, Japan, 113-8519
        • Novartis Investigative Site
      • Kodaira, Tokyo, Japan, 187-8551
        • Novartis Investigative Site
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Novartis Investigative Site
      • Maastricht, Netherlands, 5800
        • Novartis Investigative Site
  • Poland
      • Gdansk, Poland, 80-803
        • Novartis Investigative Site
      • Katowice, Poland, 40-662
        • Novartis Investigative Site
      • Lodz, Poland, 93-121
        • Novartis Investigative Site
  • Spain
      • Madrid, Spain, 28040
        • Novartis Investigative Site
    • Cataluña
      • Barcelona, Cataluña, Spain, 08025
        • Novartis Investigative Site
      • L´Hospitalet de Llobregat, Cataluña, Spain, 08907
        • Novartis Investigative Site
  • United Kingdom
      • Glasgow, United Kingdom, G51 4TF
        • Novartis Investigative Site
      • Liverpool, United Kingdom, L9 7LJ
        • Novartis Investigative Site
      • London, United Kingdom, WC1N 3BG
        • Novartis Investigative Site
      • Newcastle Upon Tyne, United Kingdom, NE1 4LP
        • Novartis Investigative Site
    • Oxfordshire
      • Headington, Oxfordshire, United Kingdom, OX3 9DU
        • Novartis Investigative Site
  • United States
    • California
      • Orange, California, United States, 92868
        • Novartis Investigative Site
    • Florida
      • Miami, Florida, United States, 33136
        • Novartis Investigative Site
      • Saint Petersburg, Florida, United States, 33713
        • Novartis Investigative Site
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Novartis Investigative Site
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Novartis Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Novartis Investigative Site
    • New York
      • New York, New York, United States, 10032
        • Novartis Investigative Site
      • Patchogue, New York, United States, 11772
        • Novartis Investigative Site
      • Plainview, New York, United States, 11803
        • Novartis Investigative Site
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Novartis Investigative Site
    • Texas
      • Houston, Texas, United States, 77030
        • Novartis Investigative Site
    • Vermont
      • Burlington, Vermont, United States, 05401
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 73 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Fingolimod (FTY720)
Participants received Fingolimod 0.5 mg orally once daily.
Drug: Fingolimod
Fingolimod 0.5 mg capsules
Placebo Comparator: Placebo
Participants received matching placebo to Fingolimod orally once daily.
Drug: Placebo Comparator
Matching placebo capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
Time Frame: Month 12
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.
Month 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline for Grip Strength, Dominant Hand
Time Frame: baseline, Month 6, Month 12
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.
baseline, Month 6, Month 12
Change From Baseline for Grip Strength, Non-dominant Hand
Time Frame: baseline, Month 6, Month 12
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.
baseline, Month 6, Month 12
Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS)
Time Frame: baseline, Month 6, Month 12
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.
baseline, Month 6, Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Collaborators

Mitsubishi Tanabe Pharma Corporation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 22, 2012

Primary Completion (Actual)

September 3, 2016

Study Completion (Actual)

September 3, 2016

Study Registration Dates

First Submitted

June 19, 2012

First Submitted That Met QC Criteria

June 20, 2012

First Posted (Estimate)

June 21, 2012

Study Record Updates

Last Update Posted (Actual)

October 30, 2017

Last Update Submitted That Met QC Criteria

September 28, 2017

Last Verified

September 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CFTY720I2201
  • 2011-005280-24 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

IPD Plan Description

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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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