Long-Term Setmelanotide for Obesity

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

205 Participants Needed

Trial Summary

What is the purpose of this trial?

This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous clinical trial on treatment with setmelanotide for obesity associated with genetic defects upstream of the Melanocortin-4 (MC4) receptor in the leptin-melanocortin pathway.

Research Team

David Meeker, MD

Principal Investigator

Rhythm Pharmaceuticals, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+