Long-Term Setmelanotide for Obesity

No longer recruiting at 30 trial locations

Overseen By Rushika Conroy

Age: Any Age

Sex: Any

Travel: May Be Covered

Trial Phase: Phase 2 & 3

Sponsor: Rhythm Pharmaceuticals, Inc.

Must be taking: Setmelanotide

Disqualifiers: Pregnancy, Melanoma, Psychiatric disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 5 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety and effectiveness of setmelanotide for individuals with obesity linked to specific genetic issues. It focuses on those who completed a previous trial with setmelanotide and benefited from it. Participants must have a history of obesity due to genetic factors affecting the MC4 receptor pathway and must have shown positive results in earlier trials. The trial aims to determine if ongoing treatment remains safe and effective. As a Phase 2, Phase 3 trial, it measures the treatment's effectiveness in a smaller group and represents the final step before FDA approval, offering participants a chance to contribute to groundbreaking research.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team to get a clear answer.

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

Research shows that setmelanotide is generally well-tolerated by patients. Studies have not identified any new safety concerns during the trials. However, more information is needed to fully understand its long-term safety.

The FDA has already approved setmelanotide for helping individuals aged 2 and older lose weight and maintain weight loss. This approval indicates the treatment is considered safe for its intended use. However, ongoing trials continue to gather more information.

Overall, while setmelanotide appears safe based on current data, participating in a clinical trial like this can provide more insights into how well the treatment is tolerated over time. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Setmelanotide is unique because it targets the melanocortin-4 receptor (MC4R) pathway, which plays a key role in regulating hunger and energy expenditure. Unlike most treatments for obesity that focus on reducing calorie intake or increasing physical activity, setmelanotide directly addresses genetic factors that contribute to severe obesity. Researchers are excited about this treatment because it offers a new mechanism of action that could be particularly effective for individuals whose obesity is driven by rare genetic conditions, offering a tailored approach where traditional options might fall short.

What evidence suggests that setmelanotide might be an effective treatment for obesity?

Research has shown that setmelanotide, the treatment under study in this trial, can aid weight loss and reduce hunger in individuals with obesity caused by certain genetic issues. In previous studies, patients with LEPR-deficiency, a specific type of obesity, lost weight effectively; one adult reduced their BMI by 14.8% in six months. Setmelanotide has also improved weight-related outcomes for conditions like Bardet-Biedl syndrome (BBS). Overall, setmelanotide shows promise in managing obesity linked to specific genetic problems. ^{2 4 5 6 7}

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 2 or older who have previously been treated with setmelanotide for obesity linked to genetic defects and saw benefits. They must be able to follow the study plan, use effective birth control, and not have significant liver disease, severe kidney issues, certain psychiatric disorders, a history of melanoma or suicidal behavior.

Inclusion Criteria

If the patient exhibited a clinically meaningful benefit (efficacy) to setmelanotide treatment, and would benefit from continued treatment, he/she may be eligible for the extension study, after discussion with the Sponsor.

I am over 2 years old and have safely benefited from a prior setmelanotide trial.

Agree to use a highly effective form of contraception throughout the trial

Exclusion Criteria

You have thought about or attempted suicide in the past.

I or my close family have had melanoma, or I have a history of albinism.

You are pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants continue receiving setmelanotide treatment via once daily subcutaneous injection

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 years

Long-term extension

Exploration of long-term safety and tolerability of setmelanotide

Up to 7 years

What Are the Treatments Tested in This Trial?

Interventions

• Setmelanotide

Trial Overview The trial is testing the long-term safety and effectiveness of continued setmelanotide treatment in those who've shown positive results from earlier trials. Setmelanotide targets specific genetic causes of obesity by acting on a pathway that controls weight.

How Is the Trial Designed?

Group I: Setmelanotide Experimental Treatment 1 Intervention

Participants received setmelanotide at the same starting dose as received in the index trials as a Subcutaneous (SC) injection once daily for up to 5.6 years in this extension trial.

Setmelanotide is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Imcivree for:

• Chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

Approved in European Union as Imcivree for:

• Chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

Approved in Canada as Imcivree for:

• Chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).

Who Is Running the Clinical Trial?

Rhythm Pharmaceuticals, Inc.

Lead Sponsor

Trials

31

Recruited

10,400+

Citations

1. 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 ...

2. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2020/213793Orig1s000MedR.pdf

213793Orig1s000 - accessdata.fda.gov LEPR-deficiency obesity, demonstrated that setmelanotide was effective on the primary endpoint in adults and children 6 years of age and ...

3. ir.rhythmtx.com ir.rhythmtx.com/news-releases/news-release-details/rhythm-pharmaceuticals-announces-new-data-presentations-patients

Release Details One adult patient with congenital hypothalamic obesity achieved a -14.8% BMI reduction baseline at month 6 of setmelanotide therapy.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03287960?term=AREA%5BBasicSearch%5D(AREA%5BBasicSearch%5D(AREA%5BInterventionSearch%5D(BIM-22493)%20AND%20AREA%5BStudyType%5D(INTERVENTIONAL)))&rank=7

Setmelanotide for the Treatment of Leptin Receptor (LEPR) ... A group of independent scientists who monitor the safety and scientific integrity of a clinical trial. The DMC can recommend to the sponsor that the trial be ...

5. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK599840/

Clinical Review - Setmelanotide (Imcivree) - NCBI - NIH Overall, the results of Study 023 and Study 022 were consistent with setmelanotide improving important weight-related outcomes for patients with BBS, but the ...

6. imcivree.com imcivree.com/hcp/ppl/safety-profile/

Safety Profile | IMCIVREE® (setmelanotide) injection IMCIVREE is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 2 years and older.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03651765

NCT03651765 | Long Term Extension Trial of Setmelanotide This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous ...