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Carbetocin

Phase 3

Hyperphagia in Prader-Willi Syndrome | Small molecule | Other |ACADIA Pharmaceuticals Inc.|Last Updated: Sep 10, 2025

Target and mechanism

Molecular targetOXTR
Target classAgonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment170

FDA Designations

No designations recorded

Clinical trial landscape

Carbetocin · 1 trial · 1 indication

Phase 3 1
NCT06173531Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi SyndromeHyperphagia in Prader-Willi Syndrome
ACTIVE NOT_RECRUITING170 Analytics
PHASE3ACTIVE NOT_RECRUITING
Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Hyperphagia in Prader-Willi SyndromeUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
Baseline to Week 12

The HQ-CT is a nine-item questionnaire designed to be completed by caregivers of subjects with PWS. It is a revision of the 11-item HPWSQ-R and has been further validated. The Foundation for Prader-Willi Research has made the HQ-CT available for clinical studies in PWS, and it is the consensus instrument within the PWS research community for measuring observable behaviors that stem from subjects' excessive drive to eat. The HQ-CT should be completed by the same caregiver throughout the study. The HQ-CT will be administered to the caregiver by a rater using standardized prompts. The Food Safe Zone should be administered immediately before administration of the HQ-CT. A higher score on the HQ-CT indicates greater severity of hyperphagia.

Secondary Endpoints

Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS
Baseline to Week 12
Clinical Global Impression-Change (CGI-C) for PWS score at Week 12
Score at Week 12
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CarbetocinEXPERIMENTALCarbetocin nasal spray 3.2 mg three times daily (TID)
PlaceboPLACEBO_COMPARATORPlacebo

Interventions

NameTypeDescription
CarbetocinDRUGCarbetocin nasal spray 3.2 mg three times daily (TID)
PlaceboDRUGPlacebo given TID, identical in appearance respective to carbetocin treatment
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Eligibility Criteria

Age Range5 Years to 30 Years
SexALL
Healthy VolunteersNo
Study Sites30

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...

Countries:United StatesCanadaFranceGermanySpainUnited Kingdom
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Frequently asked questions about Carbetocin

What is Carbetocin used for in Prader-Willi Syndrome?

Carbetocin is an investigational small molecule being developed for the treatment of hyperphagia in Prader-Willi Syndrome. Hyperphagia is a hallmark symptom of the condition characterized by an insatiable appetite. Carbetocin is administered as a nasal spray and is currently in Phase 3 clinical development.

How does Carbetocin work?

Carbetocin is a synthetic analog of oxytocin, a hormone involved in social bonding and appetite regulation. It is designed to target pathways related to hyperphagia in Prader-Willi Syndrome, potentially reducing the excessive hunger and food-seeking behaviors associated with the condition. The exact mechanism is under investigation in clinical trials.

Who is developing Carbetocin?

Carbetocin is being developed by ACADIA Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ACAD. ACADIA is conducting a Phase 3 clinical trial to evaluate the safety and efficacy of Carbetocin nasal spray for hyperphagia in Prader-Willi Syndrome.

What phase is Carbetocin in?

Carbetocin is in Phase 3 clinical development for the treatment of hyperphagia in Prader-Willi Syndrome. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 3 trial is active but not recruiting participants, with an enrollment target of 170 patients.

What clinical trials is Carbetocin in?

Carbetocin is being studied in a Phase 3 clinical trial with the identifier NCT06173531, titled 'Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome.' The trial is randomized, double-blind, and placebo-controlled, enrolling 170 participants aged 5 years and older across the United States, Canada, France, Germany, Spain, and the United Kingdom.