For Healthcare Professionals

EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway


About the study

The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor pathway: POMC or PCSK1 (Sub-study 035a) LEPR (Sub-study 035b) SRC1 (Sub-study 035c) SH2B1 (Sub-study 035d) The objectives and endpoints are identical for these sub-studies.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


Inclusion Criteria:

Patients must have a pre-identified:

  1. Heterozygous genetic variant in the POMC gene or PCSK1 gene
  2. Heterozygous genetic variant in the LEPR gene
  3. Homozygous, heterozygous, or compound heterozygous variant in the NCOA1 (SRC1)
  4. Homozygous, heterozygous, or compound heterozygous variant in SH2B1 gene, or chromosomal 16p11.2 deletion encompassing the SH2B1 gene
  5. Between 6 and 65 years of age at the time of provision of informed consent/assent
  6. Obesity, defined as BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 up to 17 years of age
  7. Patient and/or parent or guardian is able to understand and comply with the requirements of the study and is able to understand and sign the written informed consent/assent
  8. Patient and/or parent or guardian reports that patient experienced childhood obesity, defined as the patient and/or parent or guardian reporting that the patient was significantly overweight during childhood
  9. Agree to use a highly effective form of contraception throughout the study and for 90 days following the study
  10. Reported history of lifestyle intervention of diet and exercise
  11. Reported history of hyperphagia


Key Exclusion Criteria:

  1. Weight loss of 2% or greater in the previous 3 months
  2. Recent history of bariatric surgery
  3. Significant psychiatric disorder(s)
  4. Suicidal ideation, attempt or behavior
  5. Clinically significant pulmonary, cardiac, endocrine/metabolic, hepatic or oncologic disease
  6. Glycated hemoglobin (HbA1C) >10% at Screening
  7. History of significant liver disease or severe kidney disease
  8. History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism
  9. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
  10. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing
  11. Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide
  12. Significant hypersensitivity to any excipient in the study drug
  13. If female, pregnant or breastfeeding
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Study Locations

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How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Obesity,Genetic Obesity

Age (in years)

6 - 65


Phase 3

Participants needed


Est. Completion Date

Dec 31, 2024

Treatment type



Rhythm Pharmaceuticals, Inc. identifier


Study number


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