A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

Conditions

Hyperphagia related behaviour associated with Prader-Willi Syndrome (PWS)

Brief summary

Treatment-emergent adverse events (TEAEs), Serious adverse events (SAEs), Withdrawals due to adverse events (AEs), Potentially clinically important changes in other safety assessments, Device incidents, or device malfunctions

Detailed description

Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score – change from Baseline at each visit, Clinical Global Impression–Severity (CGI-S) for PWS score – change from Baseline at each visit, Percentage of subjects with treatment response based on the HQ-CT (defined as improvement from Baseline ≥8 points) at each visit, PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) score – change from Baseline at each visit, Clinical Global Impression – Change (CGI-C) for PWS score at each visit, CGI-S for hyperphagia in PWS score – change from Baseline at each visit, Food Safe Zone score – change from Baseline at each visit, Zarit Burden Interview (ZBI) score – change from Baseline at each visit, PWS-specific Healthcare Resource Use and Caregiver Burden Questionnaire (PWS-HRUQ) – change from Baseline at each visit

Related papers

No related papers found yet.

Interventions

DRUGCarbetocin Nasal Spray

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Treatment-emergent adverse events (TEAEs), Serious adverse events (SAEs), Withdrawals due to adverse events (AEs), Potentially clinically important changes in other safety assessments, Device incidents, or device malfunctions	—

Secondary

Measure	Time frame
Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score – change from Baseline at each visit, Clinical Global Impression–Severity (CGI-S) for PWS score – change from Baseline at each visit, Percentage of subjects with treatment response based on the HQ-CT (defined as improvement from Baseline ≥8 points) at each visit, PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) score – change from Baseline at each visit, Clinical Global Impression – Change (CGI-C) for PWS score at each visit, CGI-S for hyperphagia in PWS score – change from Baseline at each visit, Food Safe Zone score – change from Baseline at each visit, Zarit Burden Interview (ZBI) score – change from Baseline at each visit, PWS-specific Healthcare Resource Use and Caregiver Burden Questionnaire (PWS-HRUQ) – change from Baseline at each visit	—

Measure

Time frame

Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score – change from Baseline at each visit, Clinical Global Impression–Severity (CGI-S) for PWS score – change from Baseline at each visit, Percentage of subjects with treatment response based on the HQ-CT (defined as improvement from Baseline ≥8 points) at each visit, PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) score – change from Baseline at each visit, Clinical Global Impression – Change (CGI-C) for PWS score at each visit, CGI-S for hyperphagia in PWS score – change from Baseline at each visit, Food Safe Zone score – change from Baseline at each visit, Zarit Burden Interview (ZBI) score – change from Baseline at each visit, PWS-specific Healthcare Resource Use and Caregiver Burden Questionnaire (PWS-HRUQ) – change from Baseline at each visit

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Countries

France, Germany, Spain

Outcome results

None listed