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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Resultados
Resultados de este estudio
Publicado September 2018Se han publicado los resultados de este estudio.
¿De qué trata este estudio?
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.
Puntos clave
- 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.
¿Quién puede ser elegible?
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.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- 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?
¿En qué consiste la participación?
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.
Riesgos y beneficios potenciales
Ubicaciones (73)
- UAB Childrens Hospital Harbor Center Neurology DeptCódigo postal verificadoBirmingham, United States
- Childrens Hospital Los AngelesCódigo postal verificadoLos Angeles, United States
- UCSFCódigo postal verificadoSan Francisco, Estados Unidos
- University of MiamiCódigo postal verificadoMiami, United States
- AMO CorporationCódigo postal verificadoTallahassee, United States
- Hospital General de MassachusettsCódigo postal verificadoBoston, Estados Unidos
- Wayne State UniversityCódigo postal verificadoDetroit, Estados Unidos
- Robert Wood Johnson Medical SchoolCódigo postal verificadoNew Brunswick, United States
- Childrens Hospital of PhiladelphiaCódigo postal verificadoFiladelfia, Estados Unidos
- University of Utah Clinical Trials OfficeCódigo postal verificadoSalt Lake City, Estados Unidos
- Sitio de Investigación de NovartisCódigo postal verificadoParkville, Australia
- Sitio de Investigación de NovartisCódigo postal verificadoViena, Austria
Preguntas comunes
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.
Will I know which treatment I'm getting?
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.
¿Cuánto tiempo durará el estudio?
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.
Can I stop participating if I want to?
Yes, you are free to leave the study at any time, for any reason, without it affecting your medical care.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.