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

Novartis

Status and phase

Completed

Phase 3

Conditions

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Treatments

Drug: Placebo Comparator

Drug: Fingolimod

Study type

Interventional

Funder types

Industry

Identifiers

NCT01625182

CFTY720I2201

2011-005280-24 (EudraCT Number)

Details and patient eligibility

About

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

Full 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.

Enrollment

106 patients

Sex

All

Ages

18 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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