Open-Label Study of Setmelanotide in Hypothalamic Obesity

What Was Tested

This registered phase 2 trial tested Setmelanotide.

Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).

It had 1 arm (groups): Setmelanotide (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 18 actual participants took part.

It was open to people of any sex aged from 6 years up to 40 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Participant has documented evidence of HO, including:. Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND. Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND. Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening. Participant has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.

The full record lists 11 inclusion and 15 exclusion criteria; among the exclusions: bariatric surgery or procedure within the last 6 months; diagnosis of severe psychiatric disorders; history or close family history (parents or siblings) of skin cancer or melanoma.

How the Study Was Run

Design: single-group and open-label.

Nothing was hidden in this open-label Setmelanotide study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2021-06-07 with completion listed for 2022-06-28.

It ran at 5 sites in United States (5).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 9 secondary outcomes, for example Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment, Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment, Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment, Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years, Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years and Number of Participants With Adverse Events (AEs).

What This Means in Plain Language

Put simply, this is a phase 2 trial of Setmelanotide in 18 people.

Its size is small, so on its own it can only give an early look.

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.