Evidence map›Paper›PMID 36961653›Full record

ArticleAdvances in therapy2023

Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome.

Claire Ervin, Lindsey Norcross, Usha G Mallya, Sheri Fehnel, Robert S Mittleman, Matthew Webster, Andrea M Haqq, Robert M Haws

2 registry-linked trialsOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 16 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 1 pooled it
5.6field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

NCT03013543 phase2completednot on this map

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity

TypeinterventionalSponsorRhythm Pharmaceuticals, Inc.Ran2017 to 2022Enrolled213ConditionsGenetic Obesity, Obesity, Obesity Due to Melanocortin 4 Receptor DeficiencyArmsSetmelanotide
NCT03746522 phase3completednot on this map

A Phase 3 Trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity

TypeinterventionalSponsorRhythm Pharmaceuticals, Inc.Ran2018 to 2021Enrolled52ConditionsBardet Biedl Syndrome (BBS), Alström Syndrome (AS)ArmsSetmelanotide, Placebo
3 · Its place in the literature

Who cites it

16 citing papers in PubMed, 1 synthesis or guideline pooled it, 18 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Management of Childhood Obesity.International journal of molecular sciences · 2026
    Review
  5. Review
  6. Article
  7. Review
  8. Article
  9. Hyperphagia in Bardet-Biedl syndrome: Pathophysiology, burden, and management.Obesity reviews : an official journal of the International Association for the Study of Obesity · 2025
    Review
  10. Review
  11. Article
  12. Review
  13. Article
  14. Review
  15. Review
  16. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 4 institutions in 2 countries.

Claire ErvinRTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA. cervin@rti.org.ORCID 0000-0003-2546-8980
Lindsey NorcrossRTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA.
Usha G MallyaRhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Sheri FehnelRTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA.
Robert S MittlemanRhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Matthew WebsterRhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Andrea M Haqq *University of Alberta, 6-002E Li Ka Shing Centre for Health Research Innovation, Edmonton, AB, T6G 2E1, Canada.
Robert M Haws *Marshfield Clinic Research Institute, 1000 North Oak Avenue, Marshfield, WI, 54449, USA.
Jazz Pharmaceuticals (United States) · USRTI Health Solutions · USMarshfield Clinic · USUniversity of Alberta · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionBardet-Biedl syndrome (BBS) is a rare genetic disease associated with hyperphagia, a pathologic insatiable hunger, due to impaired signaling in the melanocortin-4 receptor (MC4R) pathway. The impact of hyperphagia on the lives of patients with BBS and their families has not been fully characterized.

methodsPatients with BBS or their caregivers who participated in clinical trials of the MC4R agonist setmelanotide (NCT03013543 and NCT03746522) were included in this qualitative study. Telephone interviews were conducted using a semistructured interview guide to explore patient experience and caregiver observations of hyperphagia before and during setmelanotide treatment.

resultsNineteen interviews (8 patients, 11 caregivers) were conducted. The term "hunger" (rather than "hyperphagia") was used in interviews to ensure common terminology. Before setmelanotide treatment, all participants described their (or their child's) hunger as all-consuming, leading to an obsessive focus on food. Nine participants recalled intense, continuous hunger, and most participants (5 patients, 10 caregivers) reported lack of control with eating. Negative impacts on patients' lives included difficulties with concentration, emotional and physical manifestations, and impaired relationships. All participants experienced or observed improvements in hunger and health outcomes during treatment, the most meaningful of which included weight loss and decrease in obsessive focus on food and food-seeking behaviors. All participants reported improvements in either physical and/or emotional well-being and being satisfied with setmelanotide.

conclusionsHyperphagia and resulting food-seeking behaviors have notable negative impacts on quality of life in patients with BBS and caregivers. Setmelanotide improved hyperphagia, reduced body weight and obsessive focus on food, and facilitated improvements in physical and emotional well-being for both patients and caregivers.

trial registrationNCT03013543 and NCT03746522.

Indexed as

Bardet-Biedl SyndromeQuality of Lifealpha-MSHCaregiversChildHumansalpha-MSHsetmelanotideBardet-Biedl syndromeDisease burdenHyperphagiaMC4R pathwayQuality of lifeSetmelanotide

Identifiers

PMID36961653
PMCPMC10036981
OpenAlexW4360815487

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.