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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Novartis
Sponsor:
Collaborator:
Mitsubishi Tanabe Pharma Corporation
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01625182
First received: June 19, 2012
Last updated: July 18, 2014
Last verified: July 2014
  Purpose

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


Condition Intervention Phase
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Drug: Fingolimod
Drug: Placebo Comparator
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • time to first confirmed worsening on the adjusted INCAT Disability Scale by 1 point or more from the value at Baseline, in patients who being treated with IVIg and/or corticosteroids prior to the study start. [ Time Frame: Month 36 ] [ Designated as safety issue: No ]
    The time to the first confirmed worsening on the adjusted INCAT Disability Scale Score has to be confirmed by the Independent Evaluating Physician at any scheduled or unscheduled visit. The INCAT Disability Scale has been applied to measure disability in inflammatory neuropathies. The INCAT Disability Scale has been demonstrated to possess good clinimetric properties, has proven to correlate well with patients' perception of their disease state, and, importantly, is easily administered and requires minimal training.


Secondary Outcome Measures:
  • The change from Baseline for grip strength [ Time Frame: Month 6/ Month 36 ] [ Designated as safety issue: No ]
    Grip strength measurements will be done using a vigorimeter. With this device, the pressure in the bulb exercised by the patient is registered on a manometer via a rubber junction tube. The grip strength has been demonstrated to be responsive in various studies examining patients with inflammatory neuropathies.

  • The change from Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS) [ Time Frame: Month 6/ Month 36 ] [ Designated as safety issue: No ]
    This questionnaire was constructed using the patients' perception of their ability to perform daily and social activities. The final 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).

  • safety and tolerability of fingolimod compared with placebo in patients with CIDP [ Time Frame: Month 36 ] [ Designated as safety issue: Yes ]
    Measured by AE/SAE, hematology and biochemistry lab tests, vital signs, ECG, and pulmonary function test


Estimated Enrollment: 156
Study Start Date: December 2012
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: November 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fingolimod 0.5mg/day Drug: Fingolimod
Placebo Comparator: Placebo Drug: Placebo Comparator

Detailed Description:

This study is 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 will be randomly assigned in a ratio of 1:1 to receive oral fingolimod (0.5 mg/day) or matching placebo.

The study will consist 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.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 1 year prior to Screening
  • stable CIDP symptoms for the 2 months 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, other cytotoxic immunosuppressive medications with sustained effects (including mitoxantrone, alemtuzumab, cladribine) at any time, hematopoietic stem cell transplantation at any time
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01625182

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

  Show 87 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Mitsubishi Tanabe Pharma Corporation
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01625182     History of Changes
Other Study ID Numbers: CFTY720I2201, 2011-005280-24
Study First Received: June 19, 2012
Last Updated: July 18, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
chronic inflammatory demyelinating polyradiculoneuropathy, CIDP, FTY720, fingolimod.

Additional relevant MeSH terms:
Polyradiculoneuropathy
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Fingolimod
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 16, 2014