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Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity
Plain-language guide to NCT03013543: phase 2 non-randomized trial of Setmelanotide, 213 participants. Who took part, design and what was measured.
Key Facts
- Registry ID
- NCT03013543
- Phase
- phase 2
- Status
- completed
- Start
- 2017-02-10
- Completion (planned or actual)
- 2022-03-01
- Participants
- 213 (actual)
- Design
- non-randomized, open label (everyone knows who gets what), treatment
- Conditions studied
- Genetic Obesity; Obesity; Obesity Due to Melanocortin 4 Receptor Deficiency
- Results posted
- yes
What This Study Did, in Plain Language
What Was Tested
In this phase 2 trial, researchers set out to test Setmelanotide.
The purpose of the study was to determine the effect of setmelanotide (RM-493) on weight, hunger assessments, and other factors in participants with rare genetic disorders of obesity.
It had ten arms (groups): 16p11.2 Cohort (experimental), AS Cohort (experimental), BBS Cohort (experimental), MC4R Cohort (experimental) and POMC/PCSK1/LEPR Heterozygous Cohort (experimental).
The registry classes the interventions as drug.
Who Took Part
213 actual participants took part.
It was open to people of any sex aged from 6 years with no upper age limit.
Healthy volunteers were not accepted; participants needed the condition being studied.
Examples of who could join: Participants with the following genotypes and/or clinical assessment:. POMC/PCSK1/LEPR heterozygous - not currently enrolling new participants. POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or homozygous deficiency obesity. POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more genes) deficiency obesity. SH2B1 deficiency obesity.
How the Study Was Run
Design: non-randomized, parallel-group and open-label.
Groups ran in parallel: each of the participants stayed in one arm throughout.
Being open-label, the Setmelanotide study did not mask who received what.
It started in 2017-02-10 with completion listed for 2022-03-01.
It ran at 58 sites in United States (43), United Kingdom (4), France (3), Germany (3), Canada (1) and Greece (1).
Its stated primary purpose is treatment.
What Was Measured
It also tracked 8 secondary outcomes, for example Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment, Percent Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment, Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged ≥ 12 Years, Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged < 12 Years and Percent Change From Baseline in Waist Circumference After 3 Months of Setmelanotide Treatment.
What This Means in Plain Language
Put simply, this is a phase 2, non-randomized trial of Setmelanotide in 213 people.
The trial is marked completed; results are posted on the registry, and this page does not restate them.
More context: the Setmelanotide compound page, all human trials.
Our Notes on the Compound
Setmelanotide: An FDA-approved melanocortin-4 receptor agonist for rare genetic obesity (POMC, PCSK1, LEPR deficiency and Bardet-Biedl syndrome).
Related Pages
Source: ClinicalTrials.gov record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.