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

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)

Sponsors

Lead Sponsor: Novartis Pharmaceuticals

Collaborator: Mitsubishi Tanabe Pharma Corporation

Source Novartis
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.

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.

Overall Status Completed
Start Date December 22, 2012
Completion Date September 3, 2016
Primary Completion Date September 3, 2016
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale Month 12
Secondary Outcome
Measure Time Frame
Change From Baseline for Grip Strength, Dominant Hand baseline, Month 6, Month 12
Change From Baseline for Grip Strength, Non-dominant Hand baseline, Month 6, Month 12
Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS) baseline, Month 6, Month 12
Enrollment 106
Condition
Intervention

Intervention Type: Drug

Intervention Name: Fingolimod

Description: Fingolimod 0.5 mg capsules

Arm Group Label: Fingolimod (FTY720)

Intervention Type: Drug

Intervention Name: Placebo Comparator

Description: Matching placebo capsules

Arm Group Label: Placebo

Eligibility

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

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

Gender: All

Minimum Age: 18 Years

Maximum Age: 75 Years

Healthy Volunteers: No

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

Australia

Belgium

Canada

Czechia

France

Germany

Greece

Israel

Italy

Japan

Netherlands

Poland

Spain

United Kingdom

United States

Verification Date

September 2017

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Fingolimod (FTY720)

Type: Experimental

Description: Participants received Fingolimod 0.5 mg orally once daily.

Label: Placebo

Type: Placebo Comparator

Description: Participants received matching placebo to Fingolimod orally once daily.

Patient Data Undecided
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov