Primary: Safety and tolerability of fingolimod 0,5 mg.Secundairy: Long-term efficacy.Exploratory: Patient-Reported Outcomes Indices for Multiple Sclerosis (PRIMuS) and Short Form Health Survey*12 (SF-12), Treatment Satisfaction Questionnaire for…
ID
Source
Brief title
Condition
- Neurological disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Side effects. Special attention for ophthalmic and skin abnormalities.
Secondary outcome
Patient-Reported Outcomes Indices for Multiple Sclerosis (PRIMuS) en Short Form
Health Survey*12 (SF-12), Treatment Satisfaction Questionnaire for Medication
(TSQM-9).
Background summary
Fingolimod (FTY720) is a new oral treatment for multiple sclerosis (MS). The
application for a market authorization has been submitted to the authorities.
It is an immunosupperssant. Fingolimod decreases the number of activated
T-cells in blood and in the CNS by binding to the sphingosin-1-phosphate
receptor-1 (S1P1) on circulating lymfocytes. This binding results in a
reversible sequestration of T-cells, thus *trapping* autoagressive T-cells in
peripheral lymoid tissues. Therefore they are not able to migrate to areas of
inflammation in the CNS.
Fingolimod reduces the number of MS relapses and improves the MRI findings and
inflammatory markers.
The current study is a follow-up study in order to collect additional long-term
safety data and to enable patients from currently ongoing studies (relevant
studies in NL: CFTY720D2316 en CFTY720D2301E1 (new patients are not eligible))
to continue using finglimod for up to 60 months (5 years) or until 30Jun2016.
The duration of 60 months (5 year) will be achieved first by all Dutch
patients. Patients who are not eligible for reimbursement by health insurance
at this time (and this is the situation in the Netherlands) are still allowed
to use an additional 2 years fingolimod in this study. In the Netherlands, the
end date of this study will be 30Jun2018.
Study objective
Primary: Safety and tolerability of fingolimod 0,5 mg.
Secundairy: Long-term efficacy.
Exploratory: Patient-Reported Outcomes Indices for Multiple Sclerosis (PRIMuS)
and Short Form Health Survey*12 (SF-12), Treatment Satisfaction Questionnaire
for Medication (TSQM-9).
Study design
Open, non-comparative phase IIIB safety study for patients coming from 2
ongoing studies in the Netherlands, with fingolimod 0,5 mg daily until the drug
has obtained the registration and reimbursement status in the Netherlands. 1st
dose of study medication will be given in the clinic. Monitoring during at
least 6 h post intake of the 1st dose in case the patients did not use
fingolimod during 14 days or more.
Approx. 5000 patients.
Intervention
Treatment with fingolimod.
Study burden and risks
Risks: Adverse effects of study medication.
Burden:
Part 1:
Visits every 3 months (year 1) and every 6 months thereafter. First visit =
last visit of preceding study. During all visits vital signs, blood tests
(10-15 ml per visits, during screening incl. HIV and hepatitis B-C) and
pregnancy test (if relevant). Physical examination every 6 months and
ophthalmalogical examination (incl. OCT measurement) yearly. Completion of SF12
and TSQM-9 (ex-CFTY720D2316) or EQ5D and Multiple Sclerosis Functional
Composite (MSFC) (ex-CFTY720D2301E1) every 6 months.
Advice for monthly self-inspection of the skin.
MRI yearly for ex-CFTY720D2301E1 patients.
Part 2:
Deel 2:
Visits every 6 months. First visit = last visit of part 1. During all visits,
vital signs, physical - and neurological examination, blood collection (10-15
ml each time + onze optional 10 ml extra for biomarker assessment). ECG at
restart fingolimod. Only is case necessary according to investigator's opinion:
PFT, dermatological examination, ophthalmalogical examination.
Raapopseweg 1
Arnhem 6824 DP
NL
Raapopseweg 1
Arnhem 6824 DP
NL
Listed location countries
Age
Inclusion criteria
1. Written informed consent must be obtained before any assessment is performed.
2. Patients who have completed designated ongoing or planned Novartis global clinical
trials with fingolimod and are unable to obtain fingolimod outside a clinical trial.
Exclusion criteria
* Premature permanent discontinuation of a previous fingolimod study due to:
a. An adverse event or serious adverse event or laboratory abnormality.
b. Conditions leading to permanent study drug discontinuation such as macular edema, elevated liver enzymes five times ULN (upper limit of normal), malignancy of any organ system.
c. Unsatisfactory therapeutic result in the prceding fingolimod study (2 or more relapses per year as defined by the Dutch certified EC).
* Patients with a history of chronic disease of the immune system other than MS.
* Uncontrolled diabetes mellitus (HbA1c > 8%), certain forms of diabetic retinopathy.
* Patients with active systemic bacterial, viral or fungal infections. Positive HIV antibody, Hepatitis B surface antigen or Hepatitis C antibody tests.
* Voorgaande behandeling met cladribine, cyclophosphamide of mitoxantrone.
* Immunosuppressive medications within 3 months prior to baseline.
* Immunoglobulins and/or monoclonal antibodies within 3 months prior to baseline.
* Cladribine, cyclophosphamide or mitoxantrone at any time.
* Any of the following cardiovascular conditions that have developed during the previous fingolimod study: history of cardiac arrest, MI in the last 6 months, unstable ischemic heart disease, cardiac failure (NYHA Class III), patients receiving current treatment with Class III antiarrhythmic drugs, history or presence of second type II or third degree AV block or corrected QTc interval >450 msec in males and >470 msec in females, proven history of sick sinus syndrome or sino-atrial heart block, uncontrolled hypertension, resting heart rate <45 bom, angina pectoris due to coronary spasm or Raynaud*s phenomenon.
* Pulmonary fibrosis, active tuberculosis.
* Pregnancy, lactation, inadequate contraception.
Patients who temporarily or permanently discontinued from any fingolimod study because of pregnancy or nursing (lactating) can be re-enrolled.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2010-020515-37-NL |
| ClinicalTrials.gov | NCT01201356 |
| CCMO | NL33502.029.10 |