Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease
This research trial is investigating a medication called apremilast for children and teenagers between 2 and 17 years old who have Behçet's disease and frequently suffer from painful mouth ulcers. Behçet's disease is a rare condition that causes inflammation in blood vessels, leading to various symptoms including these ulcers. The study aims to compare apremilast with a placebo (a dummy medicine with no active drug) to see how effective apremilast is at reducing oral ulcers over a 12-week period. Participants will be carefully monitored, and information gathered will help understand if this treatment could be a helpful option for managing this symptom in young people.
Överblick
Vad handlar denna studie om?
Behçet's disease is a rare, long-term condition that causes inflammation in blood vessels throughout the body. While it can affect many different parts of the body, one common and often distressing symptom is recurring, painful mouth ulcers. These ulcers can make eating, drinking, and speaking difficult, significantly impacting a child's quality of life.
This study, called the Apremilast Pediatric Study, is specifically designed for children and teenagers aged 2 to 17 years who have Behçet's disease and regularly experience these mouth ulcers. The main goal is to find out if a medication called apremilast can help to reduce the number and severity of these ulcers. Apremilast is already used for some other inflammatory conditions, but its effectiveness specifically for mouth ulcers in children with Behçet's disease needs to be thoroughly investigated.
Participants in the study will be given either apremilast or a placebo (a medicine that looks identical but contains no active drug). This is a standard and important part of clinical trials, as it helps researchers accurately determine if any improvements seen are genuinely due to the study medicine or if they might happen anyway. All participants will be closely monitored by a medical team to ensure their safety and track how their ulcers respond to the treatment.
Viktiga punkter
- The study is for children aged 2-17 with Behçet's disease and recurring mouth ulcers.
- It tests apremilast a medication, against a placebo (dummy medicine).
- Aims to see if apremilast can reduce mouth ulcers.
- Participants will have regular clinic visits and monitoring.
- Potential benefits include access to a new treatment, but no guarantee of success.
- Deltagande är frivilligt, och du kan dra dig ur när som helst.
Vem kan vara berättigad?
To be considered for this study, your child must be between 2 and 17 years old. They need to have a confirmed diagnosis of Behçet's disease and have experienced mouth ulcers at least three times in the year before the study starts. Importantly, they must also have at least two active mouth ulcers both when they are first screened and on the day they officially join the study.
Your child should also have tried at least one other non-biologic treatment for their Behçet's disease in the past, such as steroid creams for their mouth or other general medications. This research is not suitable if your child has severe Behçet's disease affecting major organs like their lungs, gut, or brain, or if their eyes are badly affected and require strong immune-suppressing medicines right now. However, if these severe issues happened a while ago (at least a year) and are currently stable, they might still be able to join. Mild eye problems or Behçet's-related joint pain or skin rashes are generally allowed.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Är ditt barn mellan 2 och 17 år gammalt?
- Does your child have a confirmed diagnosis of Behçet's disease?
- Has your child had mouth ulcers at least 3 times in the last year?
- Does your child currently have at least two mouth ulcers?
- Has your child tried at least one non-biologic treatment for Behçet's disease in the past?
- Does your child currently have severe Behçet's disease affecting major organs like lungs, gut, or brain, or severe eye problems (requiring strong treatment)?
Vad innebär deltagande?
If your child takes part, they will receive either apremilast tablets or a placebo, which they will take regularly. Over a 12-week period, they will have several visits to the study clinic where the medical team will check their overall health, monitor their mouth ulcers, and record any changes. These visits will involve physical examinations, potentially blood tests, and discussions about how your child is feeling and how their ulcers are progressing. After the initial 12 weeks, there will be further follow-up appointments to ensure continued safety and gather more information about the long-term effects of the treatment. The total duration of participation in the study will be explained in full detail by the study team.
Potentiella risker och fördelar
Platser (27)
- Hospices Civils de Lyon Hopital Femme Mere EnfantVerifierat postnummerBron, Frankrike· Rekrytering
- Hopital Necker Enfants MaladesVerifierat postnummerParis, Frankrike· Avslutad
- Hopital Robert DebreVerifierat postnummerParis, Frankrike· Rekrytering
- Agia Sofia Children HospitalVerifierat postnummerAten, Grekland· Rekrytering
- Attikon University General HospitalVerifierat postnummerAten, Grekland· Rekrytering
- General Hospital of Thessaloniki IppokrateioVerifierat postnummerThessaloniki, Grekland· Rekrytering
- Meir Medical CenterVerifierat postnummerKfar Saba, Israel· Rekrytering
- Ospedale Santissima AnnunziataVerifierat postnummerChieti, Italy· Avslutad
- IRCCS Istituto Giannina GasliniVerifierat postnummerGenova, Italy· Rekrytering
- Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano PiniVerifierat postnummerMilano, Italien· Avslutad
- IRCCS Ospedale Pediatrico Bambino GesuVerifierat postnummerRom, Italien· Rekrytering
- Hospital Universitario Virgen del RocioVerifierat postnummerSeville, Spain· Rekrytering
Vanliga frågor
What is Behçet's disease?
Behçet's disease is a rare, lifelong condition that causes inflammation in blood vessels throughout the body, leading to various symptoms including mouth ulcers.
What is apremilast?
Apremilast is a medication that aims to reduce inflammation in the body. This study is testing if it can help with mouth ulcers caused by Behçet's disease in children.
Vad är en placebo?
A placebo is a 'dummy' medicine that looks like the active drug but contains no medicine. It's used to compare against the real treatment to see if the drug is truly effective.
Will my child definitely get the study medicine?
No, participants are randomly assigned to receive either apremilast or a placebo. This is to ensure fair comparison of the treatment's effects.
Can I remove my child from the study earlier?
Yes, you have the right to withdraw your child from the study at any time, for any reason, without affecting their future medical care.
Hur du får reda på mer
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