Condition médicale (spécialité visée)
Choix aire thérapeutique
Endocrinologie et métabolisme
Profil des participants
Sexe(s) des participants
TOUS
Aptitude des participants
Mineurs
Critères de sélection
Critères d'inclusion
Critères d'inclusion:
* Homme ou femme âgé de 5 à 30 ans
* Syndrome de Prader-Willi avec une mutation documentée causant la maladie
* Appétit accru avec satiété diminuée accompagnée de recherche de nourriture (conforme à la Phase Nutritionnelle 3 du PWS)
* Score total HQ-CT ≥13 au dépistage et à la ligne de base
* Score CGI-S pour l'hyperphagie dans PWS ≥4 au dépistage et à la ligne de base
* Vit avec un soignant qui comprend et est prêt et capable de suivre les procédures liées à l'étude et est prêt à participer à toutes les visites de l'étude
Critères d'exclusion:
* Diagnostic génétique du syndrome de Schaaf-Yang ou d'un autre trouble cognitif génétique, hormonal ou chromosomique autre que le PWS
* Infection respiratoire supérieure active lors de la visite de dépistage ou de la visite à la ligne de base
* Tout trouble cardiovasculaire, rénal, hépatique, gastro-intestinal ou respiratoire cliniquement significatif, y compris l'asthme sévère
* Antécédents de maladie cérébrovasculaire, traumatisme crânien, épilepsie ou migraines fréquentes. Des antécédents de convulsions fébriles ne sont pas exclusifs
* Chirurgie nasale dans le mois précédant la visite de dépistage ou planification d'une chirurgie nasale pendant l'étude.
* Refus de s'abstenir de sérum physiologique nasal, d'autres irrigations nasales et d'autres médicaments intranasaux pendant la période de dépistage et pendant la période de traitement de l'étude
* Irritabilité ou agitation cliniquement significative, nécessitant le début ou l'augmentation de la dose de médicament antipsychotique, au cours des 6 mois précédant la visite de dépistage
* Utilisation de prostaglandines, d'analogues de prostaglandines ou d'agonistes de prostaglandines dans les 3 mois précédant la visite à la ligne de base. Les inhibiteurs de la synthèse des prostaglandines, tels que les anti-inflammatoires non stéroïdiens, ne sont pas exclus.
* Début d'un agoniste du peptide 1 de type glucagon (GLP-1) dans les 6 mois précédant la visite de dépistage. Le traitement par agoniste du GLP-1 est autorisé si le sujet le prend depuis plus de 6 mois avant le dépistage.
* Utilisation d'ocytocine, de desmopressine (DDAVP) ou de tésofensine dans les 6 mois précédant la visite à la ligne de base
* Symptômes psychotiques actifs, antécédents de symptômes psychotiques ou trouble psychotique
* Tentative de suicide ou hospitalisation psychiatrique en milieu hospitalier
* Nouvelles interventions liées à l'alimentation, y compris des restrictions environnementales ou alimentaires, dans le mois précédant la visite de dépistage
Des critères d'inclusion/exclusion supplémentaires s'appliquent. Les sujets seront évalués lors du dépistage pour s'assurer que tous les critères de participation à l'étude sont remplis.
Inclusion Criteria:
* Male or female and 5 through 30 years of age
* Prader-Willi syndrome with a documented disease-causing mutation
* Increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)
* HQ-CT total score of ≥13 at Screening and Baseline
* CGI-S score for hyperphagia in PWS of ≥4 at Screening and Baseline
* Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits
Exclusion Criteria:
* Genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS
* An active upper respiratory infection at the Screening visit or the Baseline visit
* Any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma
* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary
* Nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study.
* Unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study
* Clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit
* Used prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary.
* Started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening.
* Used oxytocin, desmopressin (DDAVP), or tesofensine within 6 months prior to the Baseline visit
* Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
* History of suicide attempt or inpatient psychiatric hospitalization
* New food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit
Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.
Critères d'exclusion
1. Is genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS.
2. Has an active upper respiratory infection at the Screening visit or the Baseline visit.
3. Has any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma.
4. Has a history of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
5. Has significant, uncorrected visual or uncorrected hearing impairment.
6. Has had major surgery within 1 month of the Screening visit or planning to have surgery during the study.
7. Has had nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study.
8. Is unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study.
9. Has had more than three episodes of sinusitis in the 12 months prior to the Baseline visit.
10. Has another nasal disorder that may affect deposition of intranasal medication.
11. Has known hypersensitivity to any component of study drug.
12. Has been diagnosed with cancer (except managed basal cell carcinoma or squamous cell carcinoma of the skin).
13. Has had clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit.
14. Has used systemic prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary.
15. Has started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening.
16. Has used oxytocin, desmopressin (DDAVP), or tesofensine within 6 months prior to the Baseline visit.
17. Has active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder.
18. Has a history of suicide attempt or inpatient psychiatric hospitalization.
19. Has new food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit.
20. Has participated in an interventional research study involving another investigational medication or device in the 6 months prior to the Screening visit.
21. Has a history of or current abuse of/dependence on alcohol or illicit drugs.
22. Has a clinically significant abnormal laboratory value at Screening. Laboratory testing may be repeated during the Screening period with agreement of the Medical Monitor.
23. Has serum potassium below the normal range (according to the central laboratory) at Screening. Serum potassium may be repeated during the Screening period with the agreement of the Medical Monitor.
24. Has a clinically significant thyroid function test result at Screening (as measured by thyroid stimulating hormone [TSH] and reflex free thyroxine [T4]). If TSH is abnormal and the reflex free T4 is normal, the subject may be randomized.
25. Has clinically significant abnormality in vital signs at Screening or Baseline.
26. Has any of the following:
a. QTcF interval of >450 ms at Screening or Baseline (before dosing)
b. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
c. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
d. Has any other clinically significant finding on ECG at Screening or Baseline (before dosing)
27. Has a positive pregnancy test at Screening.
28. Is an employee or is a family member of an employee of Acadia Pharmaceuticals Inc.
29. Is judged by the Investigator or the Medical Monitor to be inappropriate for the study for any reason.