Setmelanotide for Obesity

Recruiting at 7 trial locations

Overseen By Rhythm Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Rhythm Pharmaceuticals, Inc.

Disqualifiers: Prader-Willi, ROHHADNET, Severe psychiatric, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

39 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called setmelanotide to determine if it can help individuals with congenital Hypothalamic Obesity (cHO) lose weight, feel less hungry, and improve their quality of life. Participants will receive either setmelanotide or a placebo (a substance with no active medication) through daily injections for up to 26 weeks. Suitable candidates may have conditions like multiple pituitary hormone deficiency and experience significant weight gain or high body mass index (BMI). As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are on hormone replacement therapy, your dose must have been stable for at least 2 months before joining the study.

Is there any evidence suggesting that setmelanotide is likely to be safe for humans?

Research has shown that setmelanotide is generally well-tolerated. One study demonstrated significant weight loss and better hunger control with the treatment. Most participants managed the treatment well, with no serious safety issues reported. This suggests that setmelanotide could be a safe option for treating obesity related to certain genetic conditions. While minor side effects can occur, previous research indicates a favorable overall safety profile. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for obesity?

Setmelanotide is unique because it targets the melanocortin-4 receptor (MC4R) pathway, which plays a key role in regulating hunger and energy expenditure. Unlike other obesity treatments that primarily focus on appetite suppression or calorie absorption, setmelanotide works by addressing the underlying genetic factors contributing to obesity. Researchers are excited about its potential to offer a more effective, targeted approach for individuals with specific genetic causes of obesity, potentially leading to significant weight loss and improved metabolic health.

What evidence suggests that setmelanotide might be an effective treatment for congenital Hypothalamic Obesity?

Research has shown that setmelanotide, which participants in this trial may receive, can help individuals with certain types of obesity lose weight and control hunger. One study found that participants lost about 20% of their highest weight in the first year of treatment. Another study reported a noticeable drop in BMI, a measure of body fat using height and weight. Participants also noted improvements in hunger symptoms. These findings suggest that setmelanotide could be effective for those with congenital hypothalamic obesity (cHO), aiding in weight loss and better hunger management. ^{3 4 6 7 8}

Who Is on the Research Team?

David Meeker, MD

Principal Investigator

Rhythm Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for patients aged 4 and older with congenital Hypothalamic Obesity, which includes conditions like sexual infantilism, septo-optic dysplasia, optic nerve hypoplasia, and panhypopituitarism. Participants must agree to use effective contraception during the study and for 90 days after.

Inclusion Criteria

I am at least 4 years old.

I agree to use effective birth control during and for 3 months after the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a daily injection of either setmelanotide or placebo for up to 26 weeks

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Setmelanotide

Trial Overview The trial tests Setmelanotide's effectiveness in reducing weight, hunger, and improving quality of life against a placebo. Patients receive daily injections of either Setmelanotide or placebo for up to 26 weeks while their health outcomes are monitored.

How Is the Trial Designed?

Group I: Setemelanotide Experimental Treatment 1 Intervention

Randomized 2:1 (Setmelanotide: Placebo)

Group II: Placebo Placebo Group 1 Intervention

Randomized 2:1 (Setmelanotide: Placebo)

Who Is Running the Clinical Trial?

Rhythm Pharmaceuticals, Inc.

Lead Sponsor

Trials

31

Recruited

10,400+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40897637/

Real-World Efficacy and Safety of Setmelanotide in Adults ... Results: Patients experienced a clinically significant weight reduction of 20% from their highest pre-treatment weight within the first year.

2. ir.rhythmtx.com ir.rhythmtx.com/news-releases/news-release-details/rhythm-pharmaceuticals-presents-data-mc4r-agonists-setmelanotide

Rhythm Pharmaceuticals Presents Data on MC4R Agonists ... Rhythm Pharmaceuticals Presents Data on MC4R Agonists Setmelanotide and Bivamelagon at ENDO 2025 · -9.3% BMI reduction from baseline in the 600mg ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2213858724002730

Setmelanotide in 2-5yr olds w/ MC4R obesity (VENTURE trial) Based on this real-world survey of patients with BBS and their caregivers, setmelanotide improved key symptoms related to insatiable hunger and obesity.

4. gastroenterologyadvisor.com gastroenterologyadvisor.com/news/setmelanotide-under-review-for-acquired-hypothalamic-obesity/

Setmelanotide Under Review for Acquired Hypothalamic ... Findings showed treatment with setmelanotide resulted in a statistically significant and clinically meaningful reduction in BMI compared with ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT02896192

NCT02896192 | Setmelanotide for the Treatment of Early- ... Participants who achieved at least a 5 kilograms (kg) weight loss (or at least 5% weight loss if baseline body weight was <100 kg) at the end of the open label ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10608339/

Efficacy and Safety of Setmelanotide, a Melanocortin-4 ... Our results show that setmelanotide (MC4R agonist) is associated with significant weight loss, body measurement, and hunger improvements in individuals who ...

7. ir.rhythmtx.com ir.rhythmtx.com/news-releases/news-release-details/rhythm-pharmaceuticals-announces-pivotal-phase-3-transcend-trial

Rhythm Pharmaceuticals Announces Pivotal Phase 3 ... 83% percent of patients on setmelanotide therapy achieved 5% or greater reduction in BMI (n=33 patients 18 or older) or BMI Z-score reduction of ...

8. thelancet.com thelancet.com/journals/landia/article/PIIS2213-8587(24)00273-0/abstract

Setmelanotide in patients aged 2–5 years with rare MC4R ... Ten (83%) of the 12 overall participants reached a 0·2-point reduction or more in BMI Z score per WHO methodology at week 52 (95% CI 58·7–99·8).