Setmelanotide for Obesity

Recruiting in Palo Alto (17 mi)

+33 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Rhythm Pharmaceuticals, Inc.

Prior Safety Data

Breakthrough Therapy

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests setmelanotide, a medication to help control weight, in obese patients with specific genetic defects. These patients have issues that make typical treatments less effective. Setmelanotide helps reduce hunger and increase energy use. It has been approved in the USA for weight management.

Research Team

David Meeker, MD

Principal Investigator

Rhythm Pharmaceuticals, Inc.

Eligibility Criteria

This trial is for people aged 6-65 with severe obesity linked to specific genetic changes in the MC4R pathway. Participants must understand the study, use effective birth control, and have a BMI ≥40 kg/m2 (adults) or ≥97th percentile for their age/gender (children). Excluded are those with certain other genetic conditions, recent bariatric surgery, significant health issues like liver disease or melanoma risk, psychiatric disorders, or recent weight loss treatments.

Inclusion Criteria

I have a genetic variant linked to obesity.

Use of highly effective contraception

I am between 6 and 65 years old.

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Exclusion Criteria

HbA1C >10.0% at Screening

I have never participated in a study or taken setmelanotide before.

Any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) or Patient Health Questionnaire 9 (PHQ 9) score of ≥15 during Screening, any suicide attempt in patient's lifetime years, or any suicidal behavior in the last month

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Treatment Details

Interventions

Placebo (Other)
Setmelanotide (Melanocortin-4 Receptor Agonist)

Trial OverviewThe DAYBREAK study tests Setmelanotide's effectiveness on obesity due to MC4R pathway gene variants. It starts with an open-label phase where everyone gets Setmelanotide followed by a blind phase where participants randomly receive either Setmelanotide or a placebo without knowing which one they're getting.

Participant Groups

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Stage 2: Setmelanotide (Double-Blind)Experimental Treatment1 Intervention

Participants from Stage 1 who were eligible for Stage 2 received once daily SC injection of setmelanotide 3.0 mg from Week 16 to Week 40 in a double-blind treatment Stage (Stage 2).

Group II: Stage 1: Setmelanotide (Open-Label)Experimental Treatment1 Intervention

Participants received once daily SC injection of setmelanotide from Day 1 to Week 16 in an open-label treatment stage (Stage 1). Participants ≥12 years of age started on setmelanotide 2.0 milligrams (mg) once daily for approximately 2 weeks, then increased to 3.0 mg once daily. Participants \<12 years of age started on setmelanotide 1.0 mg once daily for approximately 1 week, then increased to 2.0 mg once daily for approximately 1 week, then increased to 3.0 mg once daily.

Group III: Stage 2: Placebo (Double-Blind)Placebo Group1 Intervention

Participants from Stage 1 who were eligible for Stage 2 received once daily SC injection of matching placebo 3.0 mg from Week 16 to Week 40 in a double-blind treatment Stage (Stage 2).

Setmelanotide is already approved in Canada for the following indications:

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).