Evaluate efficacy and safety of fingolimod 0.5 mg orally once daily versus placebo in chronic inflammatory demyelinating polyradiculoneuropathy

• Register: EUCTR
• Last refreshed on: 4 September 2017
• Main ID: EUCTR2011-005280-24-DE
• Date of registration: 30/07/2012
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Evaluate efficacy and safety of fingolimod 0.5 mg orally once daily versus placebo in chronic inflammatory demyelinating polyradiculoneuropathy
• Scientific title: A double-blind, randomized, multicenter, placebo-controlled, parallel-group study to evaluate the efficacy and safety of 0.5 mg fingolimod administered orally once daily versus placebo in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
• Date of first enrolment: 29/08/2012
• Target sample size: 156
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-005280-24
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Belgium, Canada, Czech Republic, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Norway, Poland, Spain, United Kingdom, United States

Contacts

• Name: Medizinischer Infoservice (MCC)
• Affiliation: Novartis Pharma GmbH

• Name: Medizinischer Infoservice (MCC)
• Affiliation: Novartis Pharma GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
Inclusion Criteria
1. written informed consent must be obtained before any assessment is
performed
2. The diagnosis of CIDP will use the definition of the the European
Federation of Neurological Societies/Peripheral Nerve Society
(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 forms of atypical CIDP:
•pure motor, or
•asymetrical [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 (Van
den Bergh et al 2010 and Erratum 2011)
3. 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
4. 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
5. History of documented clinically meaningful deterioration confirmed by clinical examination, during therapy or upone upon interruption or reduction of therapy within 18 months prior to Screening
6. Stable CIDP symptoms without significant change in treatment regimen for the 6 weeks before randomization.
7. Male or female, aged 18 years or older at Screening

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 78
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 78

Exclusion criteria:
Exclusion Criteria
1. other chronic demyelinating neuropathies, including: • Distal Acquired Demyelinating Symmetric Neuropathy (DADS) • Multifocal Motor Neuropathy with conduction block (MMN) • pure sensory CIDP • hematopoietic malignancy except for MGUS IgG or IgA
2. conditions in which the pathogenesis of the neuropathy may be different from CIDP such as: Lyme disease, POEMS syndrome, osteosclerotic myeloma, Castleman’s
disease
3. 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 prior to randomization should have B-cell (CD19/CD20) testing performed and if values are within normal range, patients are eligible to participate
• other cytotoxic immunosuppressive medications with sustained effects (including mitoxantrone, alemtuzumab, cladribine) at any time
• hematopoietic stem cell transplantation at any time



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
MedDRA version: 19.0 Level: PT Classification code 10057645 Term: Chronic inflammatory demyelinating polyradiculoneuropathy System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: fingolimod
Product Code: FTY720I
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: fingolimod
CAS Number: 162359-56-0
Current Sponsor code: FTY720
Other descriptive name: FINGOLIMOD HYDROCHLORIDE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.5-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: time to event

Main Objective: To evaluate the effect of fingolimod 0.5 mg daily compared with placebo on delaying disability progression, in patients with CIDP, measured by the time to the first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale. A confirmed worsening is defined as an increase by 1 point or more on the adjusted INCAT Disability Scale from the value at Baseline.

Primary end point(s): Delay of disease progression, measured by time to first confirmed worsening on the adjusted INCAT disability scale by 1 point or more from the value at baseline, in patients who were being treated with IVIG and/or corticosteroids prior to study start.

Secondary Objective: • to assess the change in grip strength from Baseline to Month 6/End-of-Treatment (whichever occurs first) in CIDP patients on fingolimod as compared with those on placebo
• to assess the change in Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS) from Baseline to Month 6/End-of-Treatment (whichever occurs first) in CIDP patients on fingolimod as compared with those on placebo
• to evaluate safety and tolerability of fingolimod compared with placebo in patients with CIDP

Secondary Outcome(s)

Secondary end point(s): 1) The change in grip strength from baseline in CIDP patients on fingolimod as compared with those on placebo. Grip strength measurements will be done using a vigorimeter
2) The change from baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS). This questionnaire was constructed using the patients perception of their ability to perform daily and social activities
3) Safety and tolerability of fingolimod compared with placebo in patients with CIDP. Measured by AE/SAE, hematology and biochemistry lab tests, vital signs, ECG, and pulmonary function test.

Timepoint(s) of evaluation of this end point: 1) Month 6/ End of treatment
2) Month 6/ End of treatment
3) End of study

Secondary ID(s)

CFTY720I2201

2011-005280-24-BE

NCT01625182

Source(s) of Monetary Support

Novartis Pharma Services AG