What is the mechanism of action of this treatment?

The most common treatments for Hypothalamic Obesity (HO) often target the central melanocortin pathways, which play a crucial role in regulating energy balance and appetite. Setmelanotide, an MC4R agonist, works by activating the melanocortin 4 receptor, which helps reduce hunger and promote weight loss. This is particularly important for HO patients, as their condition is characterized by severe obesity and hyperphagia due to hypothalamic damage. By targeting these pathways, treatments like Setmelanotide can help manage the symptoms of HO more effectively, improving both weight and quality of life for patients.

What side effects are associated with this type of intervention?

Setmelanotide, an MC4R agonist, is being studied for its efficacy in treating Hypothalamic Obesity. Common side effects of MC4R agonists like Setmelanotide include injection site reactions, nausea, and headache. Other potential side effects may include increased blood pressure and heart rate, hyperpigmentation, and gastrointestinal issues. It is important for patients to discuss these potential side effects with their healthcare provider to weigh the benefits and risks of the treatment.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of Hypothalamic Obesity in the provided context. Setmelanotide, an MC4R agonist, is currently being studied for its efficacy in weight reduction, hunger control, and quality of life improvement in patients with acquired Hypothalamic Obesity. Other treatments for obesity and related metabolic conditions, such as GLP-1 receptor agonists like liraglutide and semaglutide, have been approved for weight management and type 2 diabetes but are not specifically indicated for Hypothalamic Obesity.

What other types of research exist?

Promising alternatives to Setmelanotide for Hypothalamic Obesity patients include GLP-1 receptor agonists such as liraglutide and combination therapies involving neurotensin and GLP-1 mimetics. These treatments have shown efficacy in weight management and could be considered for HO treatment in 2024.

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Melanocortin 4 Receptor (MC4R) Agonist

Setmelanotide for Hypothalamic Obesity

Phase 3

Waitlist Available

Research Sponsored by Rhythm Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial tests if Setmelanotide, an injection, can help reduce weight and hunger in patients aged 4 and older with acquired Hypothalamic Obesity. The medication works by targeting brain pathways that control hunger and energy use. Setmelanotide has been approved for treating obesity in rare genetic conditions and is being developed for other obesity-related disorders.

Who is the study for?

This trial is for people aged 4 and older with acquired Hypothalamic Obesity, marked by weight gain after hypothalamic injury. Adults must have a BMI of ≥30 kg/m2, while children need a BMI in the top 95th percentile. Participants should agree to use effective contraception during and post-trial.

What is being tested?

The study tests Setmelanotide's effectiveness on weight reduction, hunger control, and life quality in HO patients compared to placebo. It involves daily injections and assessments over up to 60 weeks.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions at the injection site, general discomfort or changes related to hormone activity due to Setmelanotide's role as an appetite regulator.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2022 Phase 4 trial • 77 Patients • NCT05046132

46%

Nausea

39%

Injection site pain

29%

Vomiting

25%

Injection site erythema

21%

Ephelides

21%

Decreased appetite

18%

Skin hyperpigmentation

14%

Injection site swelling

14%

Headache

14%

Erection increased

11%

Injection site pruritus

11%

Fatigue

Abdominal pain

Constipation

Pancreatitis acute

Hepatitis acute

Asthenia

Chills

Injection site induration

Medical device site dermatitis

Aphthous ulcer

Change of bowel habit

Chapped lips

Diarrhoea

Gingival discolouration

Hyperaesthesia teeth

Pruritus

Rash

Cough

Dry throat

Dyspnoea

Rhinorrhoea

Muscle spasms

Pain in extremity

Palpitations

Skin abrasion

Blood creatine phosphokinase increased

Abnormal dreams

100%

80%

60%

40%

20%

Study treatment Arm

Group 1: Setmelanotide 2 mg

Group 1: Setmelanotide 3 mg

Group 2: Moxifloxacin 400mg

Group 1: Setmelanotide 5 mg

Group 1: Setmelanotide 7 mg

Group 2: Placebo

Group 3: Placebo

Group 3: Moxifloxacin 400mg

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: SetemelanotideExperimental Treatment1 Intervention

Randomized 2:1 (Setmelanotide: Placebo)

Group II: PlaceboPlacebo Group1 Intervention

Randomized 2:1 (Setmelanotide: Placebo)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Setmelanotide

2017

Completed Phase 4

~800

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Hypothalamic Obesity (HO) often target the central melanocortin pathways, which play a crucial role in regulating energy balance and appetite. Setmelanotide, an MC4R agonist, works by activating the melanocortin 4 receptor, which helps reduce hunger and promote weight loss. This is particularly important for HO patients, as their condition is characterized by severe obesity and hyperphagia due to hypothalamic damage. By targeting these pathways, treatments like Setmelanotide can help manage the symptoms of HO more effectively, improving both weight and quality of life for patients.

Role of secretin peptide family and their receptors in the hypothalamic control of energy homeostasis.New central targets for the treatment of obesity.Melanocortin 4 receptor-mediated hyperphagia and activation of neuropeptide Y expression in the dorsomedial hypothalamus during lactation.