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Phase 3 N=106 Randomized Quadruple-blind Treatment

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

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Enrolled (actual)
106
Serious AEs
12.3%
Results posted
Sep 2017
Primary outcome: Primary: Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale — 721.0; 540.0 Days — p=0.9838

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Fingolimod (Drug); Placebo Comparator (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Novartis Pharmaceuticals
Primary completion
Sep 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
721.0; 540.0 0.9838
SECONDARY
Change From Baseline for Grip Strength, Dominant Hand
-2.6; -3.8; -0.8; -3.9
SECONDARY
Change From Baseline for Grip Strength, Non-dominant Hand
-2.7; -6.1; -1.2; -5.0
SECONDARY
Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS)
-6.4; -5.5; -5.7; -5.1

Summary

The study was designed to evaluate the efficacy and safety of fingolimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy compared with 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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01625182). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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