Trial reportJCI insight2018
Intranasal carbetocin reduces hyperphagia in individuals with Prader-Willi syndrome.
Trial report in JCI insight, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
43 citing papers in PubMed, 82 citations in OpenAlex.
- Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial.The Journal of clinical endocrinology and metabolism · 2023Trial
- Oxytocin in young children with Prader-Willi syndrome: Results of a randomized, double-blind, placebo-controlled, crossover trial investigating 3 months of oxytocin.Clinical endocrinology · 2021Trial
- A randomized pilot efficacy and safety trial of diazoxide choline controlled-release in patients with Prader-Willi syndrome.PloS one · 2019Trial
- Hyperphagia in craniopharyngioma- a real-world study from the international hypothalamic-pituitary brain tumors patient registry.Scientific reports · 2026Article
- Efficacy and safety of semaglutide for obesity and hyperphagia in adults with Prader-Willi syndrome.Frontiers in endocrinology · 2026Observational
- Article
- Long-term intranasal oxytocin therapy in patients with hypothalamic syndrome: case series and literature review.Endocrine connections · 2025Article
- The Oxytocin System and Implications for Oxytocin Deficiency in Hypothalamic-Pituitary Disease.Endocrine reviews · 2025Review
- Pharmacological Aspects in the Management of Children and Adolescents with Prader-Willi Syndrome.Paediatric drugs · 2025Review
- The expanding landscape of genetic causes of obesity.Pediatric research · 2025Review
- Defining Hyperphagia for Improved Diagnosis and Management of MC4R Pathway-Associated Disease: A Roundtable Summary.Current obesity reports · 2025Review
- Is Oxytocin a Contributor to Behavioral and Metabolic Features in Prader-Willi Syndrome?Current issues in molecular biology · 2024Review
- A bibliometric analysis of Prader-Willi syndrome from 2002 to 2022.Open medicine (Warsaw, Poland) · 2024Article
- Updates on Obesity in Prader-Willi Syndrome: From Genetics to Management.Ewha medical journal · 2023Review
- Management of Hyperphagia and Obesity in Prader-Willi Syndrome.Ewha medical journal · 2023Review
- Oxytocin's Regulation of Thermogenesis May Be the Link to Prader-Willi Syndrome.Current issues in molecular biology · 2023Review
- 1,5-Anhydro-D-Fructose Exhibits Satiety Effects via the Activation of Oxytocin Neurons in the Paraventricular Nucleus.International journal of molecular sciences · 2023Article
- A Pilot Randomized Clinical Trial of Intranasal Oxytocin to Promote Weight Loss in Individuals With Hypothalamic Obesity.Journal of the Endocrine Society · 2023Article
- Hyperphagia in Prader-Willi syndrome with obesity: From development to pharmacological treatment.Intractable & rare diseases research · 2023Review
- Clinical Trials in Prader-Willi Syndrome: A Review.International journal of molecular sciences · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 2 countries.
Funding
Abstract
backgroundPrader-Willi syndrome (PWS) is a genetic neurodevelopmental disorder of life-threatening hyperphagia, obesity, intellectual deficits, compulsivity, and other behavioral problems. The efficacy and safety of i.n. carbetocin, an oxytocin analog, was evaluated in a prospective, randomized, double-blinded trial in adolescents with PWS.
methodsEligible patients aged 10-18 years with genetically confirmed PWS were randomized (1:1) to i.n. carbetocin or placebo 3 times daily for 14 days. The primary efficacy endpoint was change in parent/caregiver-rated Hyperphagia in PWS Questionnaire-Responsiveness (HPWSQ-R) total score. Secondary efficacy endpoints included HPWSQ-R behavior, drive, and severity domains; clinician-rated HPWSQ; Children's Yale-Brown Obsessive-Compulsive Severity Scale; food domain of the Reiss Profile; and Clinical Global Impression-Improvement scale. Endpoints were assessed using analysis of covariance. Relationship between primary and secondary endpoints was assessed using Pearson correlation coefficients. Safety was assessed throughout the study.
resultsDemographics and clinical characteristics were similar between treatment groups (carbetocin, n = 17; placebo, n = 20). Patients receiving carbetocin had statistically significant reductions in HPWSQ-R total score at study end (-15.6) versus patients receiving placebo (-8.9; P = 0.029); several secondary efficacy endpoints also demonstrated significant differences (P < 0.05). Treatment effects for the primary and secondary endpoints were highly correlated (P ≤ 0.0001). Incidence of adverse events (AEs) was similar between treatment groups.
conclusionI.n. carbetocin was well tolerated and improved hyperphagia and behavioral symptoms of PWS.
trial registrationClinicalTrials.gov: NCT01968187FUNDING. The study was funded by Ferring Pharmaceuticals. Recruitment was aided by ongoing work in PWS performed through Eunice Kennedy Shriver National Institute of Child Health and Human Development grant U54 HD083211.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.