Prader-Willi syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:15:12 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Prader-Willi syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Effects of Pitolisant Hydrochloride for Patients with Prader-Willi Syndrome https://clinicaltrials.eu/trial/study-on-the-effects-of-pitolisant-hydrochloride-for-patients-with-prader-willi-syndrome/ Wed, 29 Apr 2026 15:03:59 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-pitolisant-hydrochloride-for-patients-with-prader-willi-syndrome/ This clinical trial is focused on studying Prader-Willi syndrome, a genetic condition that affects many parts of the body. The study will evaluate the effects of a medication called pitolisant hydrochloride, which is also known by the code name HBS-101 and is marketed under the name WAKIX. The purpose of the study is to assess how this medication impacts the severity of excessive daytime sleepiness in patients with Prader-Willi syndrome.

The study will be conducted in two main phases. Initially, participants will be randomly assigned to receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient. This phase is double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication. After this phase, all participants will have the opportunity to receive the medication in an open-label extension, where everyone knows they are receiving the active treatment. The study will last for several weeks, with regular check-ins to monitor the participants’ health and any changes in their symptoms.

Throughout the study, the effects of pitolisant hydrochloride on various symptoms of Prader-Willi syndrome will be closely monitored. This includes not only the primary focus on excessive daytime sleepiness but also other related symptoms such as irritable and disruptive behaviors. The safety of the medication will also be assessed by tracking any side effects experienced by participants. The study aims to provide valuable information on the potential benefits and safety of using pitolisant hydrochloride for managing symptoms in individuals with Prader-Willi syndrome.

]]> Long-Term Study on Oxytocin for Children with Prader-Willi Syndrome Up to Age 4 https://clinicaltrials.eu/trial/long-term-study-on-oxytocin-for-children-with-prader-willi-syndrome-up-to-age-4/ Wed, 29 Apr 2026 15:03:42 +0000 https://clinicaltrials.eu/trial/long-term-study-on-oxytocin-for-children-with-prader-willi-syndrome-up-to-age-4/ This clinical trial focuses on children with Prader-Willi Syndrome (PWS), a genetic condition that affects growth, metabolism, and development. The study involves a treatment using a nasal spray solution called Otwillo, which contains the active substance oxytocin. Oxytocin is a hormone that plays a role in social bonding and behavior. The purpose of the study is to confirm the long-term safety of this treatment in children who have previously participated in the OTBB3 study.

The study will follow children with Prader-Willi Syndrome up to the age of four. It will compare those who have been treated with Otwillo in the previous OTBB3 study to those who have not received this treatment. The study will monitor the occurrence of any adverse events, which are unwanted effects that may happen during the study, as well as any serious adverse events. It will also track the development of other health conditions that often occur alongside Prader-Willi Syndrome, known as comorbidities.

Throughout the study, information will be collected on any medications, surgeries, or rehabilitation therapies the children may undergo. This will include details such as the type of treatment, the age at which it started and stopped, and how often it was given. The study aims to provide valuable insights into the long-term effects and safety of using oxytocin nasal spray in managing Prader-Willi Syndrome in young children.

]]> Study on the Safety and Effects of PBF-999 for Patients with Prader-Willi Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pbf-999-for-patients-with-prader-willi-syndrome/ Wed, 29 Apr 2026 14:25:03 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pbf-999-for-patients-with-prader-willi-syndrome/ This clinical trial is focused on studying the effects of a new treatment for Prader-Willi Syndrome, a genetic disorder that affects many parts of the body and often leads to a constant feeling of hunger, among other symptoms. The treatment being tested is a medication called PBF-999, which is taken in capsule form. The purpose of the study is to evaluate the safety and tolerability of PBF-999 in patients with Prader-Willi Syndrome over a period of 28 days.

Participants in the study will receive either the PBF-999 capsules or a placebo, which looks like the medication but does not contain the active ingredient. The study will monitor how the body processes the medication and any side effects that may occur. Throughout the study, participants will be asked to complete questionnaires and undergo various assessments to help researchers understand the impact of the treatment on their condition.

The study aims to gather important information about the potential benefits and risks of PBF-999 for individuals with Prader-Willi Syndrome. This research could lead to new insights and possibly new treatment options for managing the symptoms of this condition. Participants will be closely monitored by healthcare professionals to ensure their safety and well-being during the trial.

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