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Study on Carbetocin Nasal Spray for Reducing Uncontrollable Hunger in Patients with Prader-Willi Syndrome

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What is this study about?

This clinical trial is focused on studying a condition called Prader-Willi Syndrome (PWS), which is a genetic disorder that can cause a strong, uncontrollable hunger known as hyperphagia. The trial is testing a new treatment called Carbetocin Nasal Spray, which is a nasal spray solution designed to help reduce this excessive hunger. The study will compare the effects of the Carbetocin Nasal Spray, also known by its code name ACP-101, to a placebo, which is a substance with no active medication.

The purpose of the study is to see how effective the Carbetocin Nasal Spray is in managing hyperphagia in people with Prader-Willi Syndrome. Participants in the study will be randomly assigned to receive either the Carbetocin Nasal Spray or the placebo. The study will last for a period of 12 weeks, during which participants will use the nasal spray and attend regular study visits to monitor their progress. The nasal spray is administered through the nose using a special device called the Aptar CPS Nasal Spray Pump.

Throughout the study, researchers will assess changes in the participants’ hunger levels and overall condition using specific questionnaires and clinical evaluations. The goal is to determine if the Carbetocin Nasal Spray can significantly reduce the symptoms of hyperphagia in individuals with Prader-Willi Syndrome compared to those receiving the placebo. This study aims to provide valuable insights into a potential new treatment option for managing this challenging aspect of Prader-Willi Syndrome.

1 Joining the study

Upon joining the study, the participant will be required to provide informed consent. If the participant is a minor, consent will be obtained from a legally acceptable representative. The participant may also need to provide assent, which is a form of agreement to participate.

A caregiver must also provide written consent to participate as an informant in study assessments. The caregiver will be responsible for providing medical records and ensuring adherence to study procedures.

2 Initial assessment

The participant will undergo an initial assessment to confirm eligibility. This includes verifying the presence of Prader-Willi syndrome and associated symptoms such as increased appetite and food-seeking behavior.

The participant’s caregiver will be involved in this process and must be able to complete assessments and use electronic media for study visits.

3 Medication administration

The participant will receive either the carbetocin nasal spray or a placebo. The nasal spray is administered intranasally, meaning it is sprayed into the nose.

The dosage of the carbetocin nasal spray is 11.4 mg/ml, and it is provided in a 2 ml vial. The frequency and duration of administration will be determined by the study protocol.

4 Ongoing assessments

Throughout the study, the participant will undergo regular assessments to monitor changes in symptoms. This includes evaluating the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score and the Clinical Global Impression–Severity (CGI-S) score.

These assessments will occur at various intervals, with a primary focus on changes from the baseline at Week 12.

5 Completion of the study

The study is expected to conclude by April 30, 2025. Upon completion, the participant’s response to the treatment will be evaluated based on the improvement in symptoms.

The participant and caregiver will be informed of the study’s findings and any potential next steps.

Who Can Join the Study?

  • The patient or their legal representative must give written permission to participate in the study. If the patient is a minor, their legal representative must provide this permission. If the patient is an adult, they must provide their own permission unless they are under guardianship.
  • The patient’s caregiver must agree to participate in the study and provide necessary medical records.
  • The patient must have been taking any allowed long-term medications at a stable dose for at least 3 months before the study starts. Small changes in growth hormone dose or other non-significant medication changes are allowed.
  • If the patient is female, she must not be pregnant or breastfeeding. Females who can have children must avoid sexual activity or use a highly effective birth control method during the study and for at least 30 days after.
  • If the patient is male and sexually active, he must use a condom during the study and for 90 days after the last dose of the study drug. His female partner must also use a highly effective birth control method during this time, or he must not have a female partner who can have children. He must also agree not to donate sperm during the study and for 90 days after.
  • The patient must be between 5 and 30 years old.
  • The patient must have Prader-Willi syndrome with a confirmed genetic mutation causing the condition.
  • The patient must have an increased appetite and decreased feeling of fullness, along with food-seeking behavior.
  • The patient must have a specific score on a questionnaire that measures appetite-related behavior.
  • The patient must have a specific score on a scale that measures the severity of their appetite-related behavior.
  • The patient must live with a caregiver who understands the study procedures and is willing to participate in all study visits. The caregiver must be a family member or someone who has cared for the patient for at least 6 months and spends time with them regularly.
  • The caregiver must be able to receive and store the study medication as instructed.
  • The caregiver must be able to attach the nasal spray device to the medication vial.

Who Cannot Join the Study?

  • Individuals who do not have Prader-Willi Syndrome (PWS), a genetic condition that affects many parts of the body.
  • Individuals who are not experiencing hyperphagia, which means having an abnormally strong feeling of hunger or desire to eat.
  • Individuals who are outside the specified age range for the study.
  • Individuals who are not able to follow the study procedures or instructions.
  • Individuals who have other medical conditions that might interfere with the study.
  • Individuals who are taking medications that could affect the study results.
  • Individuals who are pregnant or breastfeeding.
  • Individuals who have participated in another clinical trial recently.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Oncopole Claudius Regaud Toulouse France
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Universitair Ziekenhuis Gent Gent Belgium
Hospital General Universitario Gregorio Maranon Madrid Spain
Ospedale San Raffaele S.r.l. Milan Italy
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Uzulbwqdrkjjwsrlldghu Eeufm Adt Essen Germany
Kcu Kalqxz Jibtuctem glzxc Augsburg Germany
Pzyt Tnatu Hmgoxuis Udldkfvzogsd Sabadell Spain

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Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
01.07.2024
France France
Not recruiting
01.07.2024
Germany Germany
Not recruiting
01.07.2024
Italy Italy
Not recruiting
01.07.2024
Spain Spain
Not recruiting
01.07.2024

Trial locations

Investigated drugs:

Carbetocin Nasal Spray is being studied for its potential to help manage hyperphagia, which is an excessive hunger or increased appetite, in individuals with Prader-Willi Syndrome. This medication is administered through the nose and is being tested to see if it can effectively reduce the symptoms of hyperphagia in patients with this condition.

Investigated diseases:

Prader-Willi Syndrome (PWS) – Prader-Willi Syndrome is a genetic disorder that affects many parts of the body. One of the hallmark features of this condition is hyperphagia, which is an insatiable appetite leading to chronic overeating and obesity. Individuals with PWS often experience developmental delays, intellectual disabilities, and behavioral problems. The syndrome is caused by the loss of function of specific genes on chromosome 15. As children with PWS grow, they may develop a strong interest in food, which can lead to significant weight gain if not managed. The condition requires careful management of diet and behavior to prevent complications associated with obesity.

Trial ID:
2023-506200-24-00
Protocol code:
ACP-101-302
Trial Phase:
Therapeutic confirmatory (Phase III)

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