Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results

What Was Tested

This clinical trial, phase iii paper looks at Alpha-MSH and Setmelanotide.

There is limited evidence on how hyperphagia and obesity affect health-related quality of life in patients with Bardet-Biedl syndrome, and on how management of these symptoms may influence disease burden.

This analysis evaluated changes in health-related quality of life in adults and children with Bardet-Biedl syndrome in a Phase 3 trial following 1 year of setmelanotide treatment (ClinicalTrials.gov identifier: NCT03746522).

Who Took Part

PubMed indexes the participants as children, teenagers and adults.

Patients with Bardet-Biedl syndrome and obesity received 52 weeks of treatment with setmelanotide and completed various self-reported health-related quality of life measures.

Descriptive outcomes were reported in patients with data both at active treatment baseline and after 52 weeks of treatment.

How the Study Was Run

A time span of 52 weeks appears in the methods.

What Was Measured

This analysis evaluated changes in health-related quality of life in adults and children with Bardet-Biedl syndrome in a Phase 3 trial following 1 year of setmelanotide treatment (ClinicalTrials.gov identifier: NCT03746522).

PubMed files the paper under topics including Quality of Life, Bardet-Biedl Syndrome, Obesity, Hyperphagia and alpha-MSH.

The authors tagged it with the keywords BBS, Genetic obesity, IWQOL-Lite, PedsQL and Setmelanotide.

What This Means in Plain Language

This is a clinical trial, phase iii of Alpha-MSH and Setmelanotide over 52 weeks.

Results are reported in around 8 sentences of the abstract; we leave them to the paper itself rather than summarise them.

The publication is from 2023 (Orphanet journal of rare diseases), a journal based in England.

More context: the Alpha-MSH compound page and the Setmelanotide compound page, all human trials.