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ReclutamentoEsplorativo terapeutico (Fase II)Interventistico

An Exploratory, Multicenter, Randomized, double-blind, placebo-controlled study evaluating the effect and safety of pitolisant in children and adolescents with Autism Spectrum Disorders.

This research study is investigating a medicine called pitolisant (also known as Wakix) for children and teenagers who have autism spectrum disorders. The main goal is to find out if this medicine can help improve social skills and communication. It will also carefully check for any side effects and see how safe the medicine is. This is an early-stage study, meaning it's one of the first times this medicine is being tested for this condition. Some participants will receive the actual medicine, while others will receive a dummy pill (placebo) to help researchers understand if any changes are truly due to the medicine or something else.

A colpo d'occhio

Stato
Reclutamento
Fase
Esplorativo terapeutico (Fase II)
Sponsor
Bioprojet Pharma
Obiettivo di iscrizione
58
Inizio
14 Ago 2023

Di cosa tratta questo studio?

This research study is looking into a new use for a medicine called pitolisant, which is also known by the brand name Wakix. It's being tested in children and teenagers who have autism spectrum disorders. The main aim is to see if this medicine can help improve social skills and communication, which are areas many people with autism find challenging. Researchers will also be very carefully watching to make sure the medicine is safe and to track any side effects.

This is a 'Phase II' study, which means it's an early step in testing a new medicine. It's designed to explore whether the medicine works and find the right dose, rather than confirm its effectiveness. Participants will either receive the actual medicine or a 'dummy' pill (called a placebo) that looks the same but contains no active medicine. This helps researchers compare the two groups and figure out if any changes are truly caused by the study medicine.

To measure how well the medicine works, researchers will use special questionnaires and assessments that look at social skills, daily living abilities, and sleep patterns. They will also keep a close eye on behaviour and any changes in health.

Punti chiave

  • This study explores a new medicine (pitolisant) for autism in children and teenagers.
  • It aims to see if the medicine improves social skills and communication.
  • Participants will receive either the medicine or a dummy pill.
  • Safety and side effects will be carefully monitored throughout the study.
  • This is an early-stage study to gather initial information.
  • La partecipazione è volontaria e puoi ritirarti in qualsiasi momento.

Chi può essere idoneo?

This study is open to both boys and girls of all ages. You don't need to be a specific age to be considered for this study.

More detailed information about who can and cannot join will be provided by the study team. This might include specific medical conditions, current medications, or how severe your autism symptoms are.

If you're interested, the research team will ask you some questions and do some assessments to make sure the study is a safe and suitable option for you or your child.

Questo studio potrebbe fare al caso tuo?

Rispondi a queste domande rapide per vedere se potresti essere idoneo. Questo è solo una guida — il team di ricerca prende la decisione finale.

  1. Are you a child or teenager with an autism spectrum disorder?
  2. Are you willing to take study medicine or a dummy pill?
  3. Are you able to attend regular clinic visits?
  4. Are you comfortable with health assessments and questionnaires?
  5. Ti senti a tuo agio con gli esami del sangue?
  6. Is your doctor supportive of you joining a research study?
Rispondi a ogni domanda per vedere il tuo risultato.

Cosa comporta la partecipazione?

If you or your child join this study, it will involve taking either the study medicine (pitolisant) or a dummy pill for a set period. Neither you nor the study doctors will know which one you're receiving – this is called 'double-blind'. You'll have regular visits to the clinic for check-ups, where doctors will assess health, ask about social skills and daily activities using questionnaires, and monitor for side effects. They will also take blood samples to check how the medicine is affecting the body. The total length of time you'll be involved in the study will be explained in detail by the research team.

Rischi e benefici potenziali

Taking part in a study like this might offer a potential benefit if the study medicine helps improve social skills or other aspects of autism. However, there's no guarantee the medicine will work for everyone, or even at all. There are also potential risks, such as side effects from the medicine, which could be mild or, less commonly, more serious. All risks will be fully explained before you decide to join. Remember, taking part is completely voluntary, and you can withdraw from the study at any time without giving a reason, and this won't affect your future medical care.

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Domande comuni

What is pitolisant?

Pitolisant is a medicine that's already used for other conditions, but in this study, we're exploring if it can help children and teenagers with autism.

What is a 'dummy pill'?

A dummy pill, also called a placebo, looks exactly like the study medicine but doesn't contain any active ingredient. It helps us compare the effects of the real medicine.

Will I know if I'm getting the medicine or the dummy pill?

No, neither you nor your doctor will know during the study. This is called 'double-blind' and is a standard way to get accurate results.

Cosa succede se avverto effetti collaterali?

The study team will monitor you very closely for any side effects. You should report any new or unusual symptoms to them immediately.

Quanto durerà lo studio?

The full duration of your participation in the study will be explained in detail by the research team if you decide to join.

Come saperne di più

Parla sempre con il tuo medico di base o specialista prima di decidere di partecipare a uno studio.

Interessato a partecipare?

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