Trial reportThe Journal of clinical endocrinology and metabolism2023
Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial.
Trial report in The Journal of clinical endocrinology and metabolism, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03649477 (Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin), which is not on this map. Cited by 25 papers, 3 of them syntheses that pooled it.
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.
Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin (LV-101) in Prader-Willi Syndrome (PWS) With Long Term Follow-Up (CARE-PWS)
Who cites it
25 citing papers in PubMed, 3 syntheses or guidelines pooled it, 45 citations in OpenAlex.
- The burden of illness in Prader-Willi syndrome: a systematic literature review.Orphanet journal of rare diseases · 2025Pooled it
- A comprehensive analysis of oxytocin: a potential brain-based treatment to regulate obesity.Frontiers in endocrinology · 2025Pooled it
- The oxytocin system in patients with craniopharyngioma: A systematic review.Frontiers in neuroendocrinology · 2025Pooled it
- Diazoxide Choline Extended-release Tablets in Prader-Willi Syndrome: A Randomized, Double-blind, Withdrawal Period Study.The Journal of clinical endocrinology and metabolism · 2026Trial
- Hyperphagia in Prader-Willi syndrome: linking hypothalamic dysfunction to clinical assessment and management across the lifespan.Orphanet journal of rare diseases · 2026Review
- Therapeutic peptides and proteins: Status and developments in drug delivery.Journal of controlled release : official journal of the Controlled Release Society · 2026Review
- From Molecules to Meaning: Integrating Neuropeptides, Sociostasis, and Hormesis in the Brain-Heart Axis.Current issues in molecular biology · 2026Review
- Hyperphagia in craniopharyngioma- a real-world study from the international hypothalamic-pituitary brain tumors patient registry.Scientific reports · 2026Article
- Patient advocacy group perspectives on treatment priorities and clinical trials for the rare neurodevelopmental condition, Prader-Willi syndrome.Orphanet journal of rare diseases · 2026Article
- Clinical Presentation, Genetics, and Laboratory Testing with Integrated Genetic Analysis of Molecular Mechanisms in Prader-Willi and Angelman Syndromes: A Review.International journal of molecular sciences · 2026Review
- Efficacy and safety of semaglutide for obesity and hyperphagia in adults with Prader-Willi syndrome.Frontiers in endocrinology · 2026Observational
- Oxytocin Deficiency in Childhood and Adolescence: Clinical Features, Diagnostic Challenges and Therapeutic Perspectives.Current issues in molecular biology · 2025Review
- 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
- Validation of the Food Safe Zone questionnaire for families of individuals with Prader-Willi syndrome.Journal of neurodevelopmental disorders · 2025Article
- Review
- Is Oxytocin a Contributor to Behavioral and Metabolic Features in Prader-Willi Syndrome?Current issues in molecular biology · 2024Review
- The Prader-Willi syndrome Profile: validation of a new measure of behavioral and emotional problems in Prader-Willi syndrome.Orphanet journal of rare diseases · 2024Article
Corrections and comments
- Commented on by
Authors and funding
37 authors at 20 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
contextPrader-Willi syndrome (PWS) is a rare genetic disorder characterized by endocrine and neuropsychiatric problems including hyperphagia, anxiousness, and distress. Intranasal carbetocin, an oxytocin analog, was investigated as a selective oxytocin replacement therapy.
objectiveTo evaluate safety and efficacy of intranasal carbetocin in PWS.
designRandomized, double-blind, placebo-controlled phase 3 trial with long-term follow-up.
settingTwenty-four ambulatory clinics at academic medical centers.
participantsA total of 130 participants with PWS aged 7 to 18 years.
interventionsParticipants were randomized to 9.6 mg/dose carbetocin, 3.2 mg/dose carbetocin, or placebo 3 times daily during an 8-week placebo-controlled period (PCP). During a subsequent 56-week long-term follow-up period, placebo participants were randomly assigned to 9.6 mg or 3.2 mg carbetocin, with carbetocin participants continuing at their previous dose.
main outcome measuresPrimary endpoints assessed change in hyperphagia (Hyperphagia Questionnaire for Clinical Trials [HQ-CT]) and obsessive-compulsive symptoms (Children's Yale-Brown Obsessive-Compulsive Scale [CY-BOCS]) during the PCP for 9.6 mg vs placebo, and the first secondary endpoints assessed these same outcomes for 3.2 mg vs placebo. Additional secondary endpoints included assessments of anxiousness and distress behaviors (PWS Anxiousness and Distress Behaviors Questionnaire [PADQ]) and clinical global impression of change (CGI-C).
resultsBecause of onset of the COVID-19 pandemic, enrollment was stopped prematurely. The primary endpoints showed numeric improvements in both HQ-CT and CY-BOCS which were not statistically significant; however, the 3.2-mg arm showed nominally significant improvements in HQ-CT, PADQ, and CGI-C scores vs placebo. Improvements were sustained in the long-term follow-up period. The most common adverse event during the PCP was mild to moderate flushing.
conclusionsCarbetocin was well tolerated, and the 3.2-mg dose was associated with clinically meaningful improvements in hyperphagia and anxiousness and distress behaviors in participants with PWS. CLINICAL TRIALS REGISTRATION NUMBER: NCT03649477.
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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.