Clinical trial NCT01625182: Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.
Official 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Keywords: chronic inflammatory demyelinating polyradiculoneuropathy, CIDP, FTY720, fingolimod.
Lead sponsor: Novartis
Sponsor class: INDUSTRY
Collaborators: name: Mitsubishi Tanabe Pharma Corporation
First posted: 2012-06-21
Enrollment: 106
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
timeFrame: Month 12
Secondary outcomes:
measure: Change From Baseline for Grip Strength, Dominant Hand
timeFrame: baseline, Month 6, Month 12
measure: Change From Baseline for Grip Strength, Non-dominant Hand
timeFrame: baseline, Month 6, Month 12
measure: Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS)
timeFrame: baseline, Month 6, Month 12
Other outcomes:
Locations:
city: Orange
country: United States
facility: Novartis Investigative Site
state: California
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Saint Petersburg
country: United States
facility: Novartis Investigative Site
state: Florida
city: Chicago
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Louisville
country: United States
facility: Novartis Investigative Site
state: Kentucky
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: New York
country: United States
facility: Novartis Investigative Site
state: New York
city: Patchogue
country: United States
facility: Novartis Investigative Site
state: New York
city: Plainview
country: United States
facility: Novartis Investigative Site
state: New York
city: Columbus
country: United States
facility: Novartis Investigative Site
state: Ohio
city: Houston
country: United States
facility: Novartis Investigative Site
state: Texas
city: Burlington
country: United States
facility: Novartis Investigative Site
state: Vermont
city: Sydney
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Auchenflower
country: Australia
facility: Novartis Investigative Site
state: Queensland
city: Fitzroy
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Parkville
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Leuven
country: Belgium
facility: Novartis Investigative Site
city: Liege
country: Belgium
facility: Novartis Investigative Site
city: Kingston
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Québec
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Greenfield Park
country: Canada
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
city: Praha 5
country: Czechia
facility: Novartis Investigative Site
city: Limoges
country: France
facility: Novartis Investigative Site
city: Marseille cedex 05
country: France
facility: Novartis Investigative Site
city: Montpellier
country: France
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Pessac Cedex
country: France
facility: Novartis Investigative Site
city: Strasbourg
country: France
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
state: Nordrhein-Westfalen
city: Bochum
country: Germany
facility: Novartis Investigative Site
city: Düsseldorf
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Göttingen
country: Germany
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
city: Thessaloniki
country: Greece
facility: Novartis Investigative Site
city: Thessaloniki
country: Greece
facility: Novartis Investigative Site
city: Haifa
country: Israel
facility: Novartis Investigative Site
city: Ramat Gan
country: Israel
facility: Novartis Investigative Site
city: Tel Aviv
country: Israel
facility: Novartis Investigative Site
city: Legnano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Rozzano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Cefalù
country: Italy
facility: Novartis Investigative Site
state: PA
city: Ferrara
country: Italy
facility: Novartis Investigative Site
city: Milano
country: Italy
facility: Novartis Investigative Site
city: Pisa
country: Italy
facility: Novartis Investigative Site
city: Rome
country: Italy
facility: Novartis Investigative Site
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Sayama
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Bunkyo
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Kodaira
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Aomori
country: Japan
facility: Novartis Investigative Site
city: Chiba
country: Japan
facility: Novartis Investigative Site
city: Amsterdam
country: Netherlands
facility: Novartis Investigative Site
city: Maastricht
country: Netherlands
facility: Novartis Investigative Site
city: Gdansk
country: Poland
facility: Novartis Investigative Site
city: Katowice
country: Poland
facility: Novartis Investigative Site
city: Lodz
country: Poland
facility: Novartis Investigative Site
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Cataluña
city: L´Hospitalet de Llobregat
country: Spain
facility: Novartis Investigative Site
state: Cataluña
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Headington
country: United Kingdom
facility: Novartis Investigative Site
state: Oxfordshire
city: Glasgow
country: United Kingdom
facility: Novartis Investigative Site
city: Liverpool
country: United Kingdom
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: Newcastle Upon Tyne
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Hughes R, Dalakas MC, Merkies I, Latov N, Leger JM, Nobile-Orazio E, Sobue G, Genge A, Cornblath D, Merschhemke M, Ervin CM, Agoropoulou C, Hartung HP; FORCIDP Trial Investigators. Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial. Lancet Neurol. 2018 Aug;17(8):689-698. doi: 10.1016/S1474-4422(18)30202-3. Epub 2018 Jul 9.