Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis
This research study is investigating two treatments for Multiple Sclerosis (MS) in young people aged 10 to 17: fingolimod and interferon beta-1a. The main goal is to find out how safe and effective fingolimod is compared to interferon beta-1a. The study has two main parts: a core part where treatments are compared, and a longer-term extension part where everyone receives fingolimod. This helps doctors understand which treatment works best and has the fewest side effects for younger patients with MS, ultimately aiming to improve care for them. It's a key step in finding better ways to manage MS in this age group.
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Ergebnisse
Ergebnisse dieser Studie
Veröffentlicht September 2018Die Ergebnisse dieser Studie wurden veröffentlicht.
Worum geht es in dieser Studie?
This study is designed to carefully check two medications for children and teenagers with Multiple Sclerosis (MS). MS is a condition that affects the brain and spinal cord, and it can cause various symptoms. The two medications being looked at are called fingolimod and interferon beta-1a. Doctors want to understand if fingolimod is as safe and effective as interferon beta-1a, which is a common treatment, in helping young people manage their MS.
The study is divided into two main parts. The first part, called the 'Core Phase,' lasts for two years. During this time, some young people will receive fingolimod, and others will receive interferon beta-1a. This is done with a 'double-blind' approach, meaning neither the patient nor the doctors know who is getting which treatment until the study is over. This helps make sure the results are fair. After the core phase, there's a longer 'Extension Phase' that can last up to five years. In this phase, everyone in the study will be given fingolimod, so researchers can continue to monitor their health and the effects of the treatment over a longer period.
This research is really important because it focuses specifically on younger patients, from 10 to 17 years old. Treating MS in children and teenagers can be different from treating adults, so finding out which treatments are safest and work best for them is crucial. The study also includes a small group of even younger patients (under 12 or weighing less than 40kg, or before puberty) in the extension phase to gather more information.
Wichtige Erkenntnisse
- This study evaluates two MS medications, fingolimod and interferon beta-1a, in young people.
- It aims to understand the safety and effectiveness of fingolimod in patients aged 10-17.
- The study has a two-year initial phase comparing treatments and a five-year follow-up phase.
- Many safety checks and tests will be carried out to monitor participants' health.
- It's a 'blinded' study, meaning neither patients nor doctors know which drug is given initially.
- This research hopes to improve MS treatment options for children and teenagers.
Wer könnte berechtigt sein?
To be considered for the main part of the study (Core Phase), you would generally need to have a diagnosis of multiple sclerosis. Your MS should also have shown some activity recently, such as having at least one flare-up in the past year, or two in the last two years, or signs of new activity on an MRI scan within the last six months. Your MS symptoms should also be within a certain range of mild to moderate.
There are also reasons why someone might not be able to join. For example, if you have a type of MS called 'progressive MS,' have another active long-term immune system condition, certain severe heart problems, or severe kidney problems, you wouldn't be eligible. Also, if you have a specific condition called ADEM (Acute Disseminated Encephalomyelitis), you would not be able to take part.
For the second part of the study (Extension Phase), patients who successfully completed the Core Phase can continue. New younger patients might also be able to join the Extension Phase directly if they meet specific criteria for their age group and MS diagnosis, as approved by local health authorities.
Könnte diese Studie für Sie geeignet sein?
Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.
- Are you between 10 and 17 years old?
- Do you have a confirmed diagnosis of multiple sclerosis?
- Have you had recent MS flare-ups or signs of new activity on an MRI scan?
- Do you have a type of MS that isn't 'progressive'?
- Do you have any severe heart or kidney conditions or other serious long-term immune diseases?
- Are you willing to commit to regular hospital visits and check-ups for several years?
Was beinhaltet die Teilnahme?
If you decide to take part in this study, it's a long-term commitment. The first part, called the Core Phase, lasts for two years. During this time, you would regularly visit the study clinic for check-ups, assessments, and to receive your study medication, which would be either fingolimod or interferon beta-1a. You wouldn't know which one you're getting, and neither would your doctors, to ensure fair results. You'll likely have regular MRI scans and other tests to monitor your MS and your general health.
After the two-year Core Phase, if you meet the requirements, you can then join the Extension Phase, which can last for up to five more years. In this longer phase, everyone in the study receives fingolimod. You would continue to have regular clinic visits, assessments, and tests to see how you're responding to the treatment over the long term. The total duration for someone who completes both phases could be up to seven years. Throughout the study, your health will be closely monitored, and any side effects will be carefully recorded.
Potenzielle Risiken und Vorteile
Locations (73)
- UAB Childrens Hospital Harbor Center Neurology DeptVerifizierte PostleitzahlBirmingham, Vereinigte Staaten
- Childrens Hospital Los AngelesVerifizierte PostleitzahlLos Angeles, Vereinigte Staaten
- UCSFVerifizierte PostleitzahlSan Francisco, Vereinigte Staaten
- Universität von MiamiVerifizierte PostleitzahlMiami, Vereinigte Staaten
- AMO CorporationVerifizierte PostleitzahlTallahassee, Vereinigte Staaten
- Massachusetts General HospitalVerifizierte PostleitzahlBoston, Vereinigte Staaten
- Wayne State UniversityVerifizierte PostleitzahlDetroit, Vereinigte Staaten
- Robert Wood Johnson Medical SchoolVerifizierte PostleitzahlNew Brunswick, United States
- Childrens Hospital of PhiladelphiaVerifizierte PostleitzahlPhiladelphia, Vereinigte Staaten
- University of Utah Clinical Trials OfficeVerifizierte PostleitzahlSalt Lake City, Vereinigte Staaten
- Novartis UntersuchungsstelleVerifizierte PostleitzahlParkville, Australien
- Novartis UntersuchungsstelleVerifizierte PostleitzahlWien, Österreich
Häufige Fragen
What is multiple sclerosis (MS)?
MS is a long-lasting condition affecting the brain and spinal cord, which are parts of your nervous system. It can cause various symptoms like tiredness, problems with balance, and changes in vision.
What are fingolimod and interferon beta-1a?
These are both medications used to treat MS. Fingolimod is a daily capsule, and interferon beta-1a is an injection, usually given into muscle.
Werde ich wissen, welche Behandlung ich bekomme?
For the first two years of the study, neither you nor your doctor will know if you're receiving fingolimod or interferon beta-1a. This is done to make the study results fair.
Wie lange wird die Studie dauern?
The main part of the study lasts for two years, and there's an optional longer follow-up part that can last an additional five years, making it potentially seven years in total.
Kann ich die Teilnahme beenden, wenn ich möchte?
Yes, you are free to leave the study at any time, for any reason, without it affecting your medical care.
Wie Sie mehr erfahren können
Sprechen Sie immer mit Ihrem Hausarzt oder Spezialisten, bevor Sie sich entscheiden, an einer Studie teilzunehmen.
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