Chronic Inflammatory Demyelinating PolyradiculoneuropathyPhase 3NCT01625182
Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.
A Phase 3 study testing Fingolimod in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Novartis Pharmaceuticals at 67 sites in 15 countries.
Trial summary
The study was designed to evaluate the efficacy and safety of fingolimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy compared with placebo. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- What is being tested?
- Fingolimod
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 106 participants
- Where
- 67 sitesin 15 countries
- Study dates
- 22 Dec 2012to 3 Sep 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
67 sites in 15 countries.
67 sites in 15 countries
United States12 sites
Novartis Investigative Site
Boston, Massachusetts, 02115
Novartis Investigative Site
Burlington, Vermont, 05401
Novartis Investigative Site
Chicago, Illinois, 60637
Novartis Investigative Site
Columbus, Ohio, 43210
Novartis Investigative Site
Houston, Texas, 77030
Novartis Investigative Site
Louisville, Kentucky, 40202
Novartis Investigative Site
Miami, Florida, 33136
Novartis Investigative Site
New York, New York, 10032
Novartis Investigative Site
Orange, California, 92868
Novartis Investigative Site
Patchogue, New York, 11772
Novartis Investigative Site
Plainview, New York, 11803
Novartis Investigative Site
St. Petersburg, Florida, 33713
Italy7 sites
Novartis Investigative Site
Cefalù, PA, 90015
Novartis Investigative Site
Ferrara, 44100
Novartis Investigative Site
Legnano, MI, 20025
Novartis Investigative Site
Milan
Novartis Investigative Site
Pisa, 56126
Novartis Investigative Site
Rome, 00168
Novartis Investigative Site
Rozzano, MI, 20089
France6 sites
Novartis Investigative Site
Limoges, 87042
Novartis Investigative Site
Marseille, 13385
Novartis Investigative Site
Montpellier, 34295
Novartis Investigative Site
Paris, 75013
Novartis Investigative Site
Pessac, 33604
Novartis Investigative Site
Strasbourg, 67091
Japan6 sites
Novartis Investigative Site
Aomori, Aomori, 030-8553
Novartis Investigative Site
Bunkyo, Tokyo, 113-8519
Novartis Investigative Site
Chiba, 260-8677
Novartis Investigative Site
Kodaira, Tokyo, 187-8551
Novartis Investigative Site
Nagoya, Aichi-ken, 466-8560
Novartis Investigative Site
Sayama, Osaka, 589-8511
Germany5 sites
Novartis Investigative Site
Bochum, 44791
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Düsseldorf, 40225
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Göttingen, 37075
United Kingdom5 sites
Novartis Investigative Site
Glasgow, G51 4TF
Novartis Investigative Site
Headington, Oxfordshire, OX3 9DU
Novartis Investigative Site
Liverpool, L9 7LJ
Novartis Investigative Site
London, WC1N 3BG
Novartis Investigative Site
Newcastle upon Tyne, NE1 4LP
Australia4 sites
Novartis Investigative Site
Auchenflower, Queensland, 4066
Novartis Investigative Site
Fitzroy, Victoria, 3065
Novartis Investigative Site
Parkville, Victoria, 3050
Novartis Investigative Site
Sydney, New South Wales, 2050
Canada4 sites
Novartis Investigative Site
Greenfield Park, J4V 2J2
Novartis Investigative Site
Kingston, Ontario, K7L 2V7
Novartis Investigative Site
Montreal, H3A 2B4
Novartis Investigative Site
Québec, Quebec, G1J 1Z4
Belgium3 sites
Novartis Investigative Site
Brussels, 1200
Novartis Investigative Site
Leuven, 3000
Novartis Investigative Site
Liège, 4000
Greece3 sites
Novartis Investigative Site
Athens, GR 151 25
Novartis Investigative Site
Thessaloniki, 546 36
Novartis Investigative Site
Thessaloniki, 57010
Israel3 sites
Novartis Investigative Site
Haifa
Novartis Investigative Site
Ramat Gan, 52621
Novartis Investigative Site
Tel Aviv, 64239
Poland3 sites
Novartis Investigative Site
Gdansk, 80-803
Novartis Investigative Site
Katowice, 40-662
Novartis Investigative Site
Lodz, 93-121
Spain3 sites
Novartis Investigative Site
Barcelona, Catalonia, 08025
Novartis Investigative Site
L'Hospitalet de Llobregat, Catalonia, 08907
Novartis Investigative Site
Madrid, 28040
Netherlands2 sites
Novartis Investigative Site
Amsterdam, 1105 AZ
Novartis Investigative Site
Maastricht, 5800
About this study
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)
The study was designed to evaluate the efficacy and safety of fingolimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy compared with placebo.
Read the 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.
- Being tested
- Fingolimod, Placebo Comparator
- Keywords
- chronic inflammatory demyelinating polyradiculoneuropathy, CIDP, FTY720, fingolimod.
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FingolimodDrug
Fingolimod 0.5 mg capsules
Placebo ComparatorDrug
Matching placebo capsules
| Study group | Role | What they receive |
|---|---|---|
| Fingolimod (FTY720) | Experimental | Participants received Fingolimod 0.5 mg orally once daily. Receives: Drug: Fingolimod |
| Placebo | Placebo comparator | Participants received matching placebo to Fingolimod orally once daily. Receives: Drug: Placebo Comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
Confirmed worsening in CIDP was measured by the adjusted INCAT Disability Scale. The adjusted INCAT disability scale measures arm disability and leg disability. For arm disability the scale ranges from 0 (no upper limb problems) to 5 (inability to use either arm for any purposeful movement). The leg disability scale ranges from 0 (walking not affected) to 5 (restricted to wheelchair, unable to stand and walk a few steps with help). The total adjusted INCAT disability score is calculated by the sum of the arm and leg disability scores where the total score ranges from 0 to 10. A confirmed worsening was defined as an increase by 1 or more points on the adjusted INCAT disability scale from the value at baseline.
Measured: Month 12
Secondary outcomes (3)
Change From Baseline for Grip Strength, Dominant Hand
Grip strength measurements were done using a vigorimeter. With this device, the pressure in the bulb exercised by the participant was registered on a manometer via a rubber junction tube. Both the dominant and non-dominant hands were tested. A negative change from baseline indicates deterioration.
Measured: baseline, Month 6, Month 12
Change From Baseline for Grip Strength, Non-dominant Hand
Grip strength measurements were done using a vigorimeter. With this device, the pressure in the bulb exercised by the participant was registered on a manometer via a rubber junction tube. Both the dominant and non-dominant hands were tested. A negative change from baseline indicates deterioration.
Measured: baseline, Month 6, Month 12
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. The 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). The obtained raw summed score was translated subsequently to a convenient centile metric score ranging from 0 (most severe disability) to 100 (no disability at all). A higher score indicated a better health status. A negative change from baseline indicates deterioration.
Measured: baseline, Month 6, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2012 (estimated)
- Study start22 Dec 2012
- Primary completion3 Sep 2016
- Study completion3 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Inflammatory Demyelinating PolyradiculoneuropathyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 106 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Tanabe Pharma Corporation
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01625182
- Sponsor's protocol ID
- CFTY720I2201
- Other IDs
- 2011-005280-24
- Status last verified
- Sep 2017
- First submitted
- 19 Jun 2012
- Last updated
- 30 Oct 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Publications
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.
PubMed 30001923
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT01625182
- Acronym / sponsor ID
- CFTY720I2201
- Keywords
- chronic inflammatory demyelinating polyradiculoneuropathy, CIDP, FTY720, fingolimod.
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 106 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Tanabe Pharma Corporation
- Study start
- 22 Dec 2012 (actual)
- Primary completion
- 3 Sep 2016 (actual)
- Study completion
- 3 Sep 2016 (actual)
- First posted
- 21 Jun 2012 (estimated)
- Results
- Posted 19 Sep 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fingolimod (FTY720) | Experimental | Drug: FingolimodParticipants received Fingolimod 0.5 mg orally once daily. |
| Placebo | Placebo comparator | Drug: Placebo ComparatorParticipants received matching placebo to Fingolimod orally once daily. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FingolimodFingolimod 0.5 mg capsules | Drug | Fingolimod (FTY720) |
| Placebo ComparatorMatching placebo capsules | Drug | Placebo |
Primary outcome
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
Time frame: Month 12
Confirmed worsening in CIDP was measured by the adjusted INCAT Disability Scale. The adjusted INCAT disability scale measures arm disability and leg disability. For arm disability the scale ranges from 0 (no upper limb problems) to 5 (inability to use either arm for any purposeful movement). The leg disability scale ranges from 0 (walking not affected) to 5 (restricted to wheelchair, unable to stand and walk a few steps with help). The total adjusted INCAT disability score is calculated by the sum of the arm and leg disability scores where the total score ranges from 0 to 10. A confirmed worsening was defined as an increase by 1 or more points on the adjusted INCAT disability scale from the value at baseline.
3 secondary outcomes
Change From Baseline for Grip Strength, Dominant Hand
Time frame: baseline, Month 6, Month 12
Change From Baseline for Grip Strength, Non-dominant Hand
Time frame: baseline, Month 6, Month 12
Change From Baseline for Rasch-Built Linearly Weighted Overall Disability Scale (R-ODS)
Time frame: baseline, Month 6, Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30001923Hughes 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.
- Individual participant dataSharing planned: Undecided. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
67 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fingolimod in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Novartis Pharmaceuticals at 67 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, Italy, France, Japan, Germany and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Sep 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale (time frame: Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01625182, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2017.