EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
Study Phase: Phase 3
Recruitment Status: Recruiting
Start Date: December 10, 2021
End Date: December 01, 2024
Inclusion Criteria:
- Patients must have a pre-identified:
- Heterozygous genetic variant in the POMC gene or PCSK1 gene
- Heterozygous genetic variant in the LEPR gene
- Homozygous, heterozygous, or compound heterozygous variant in the NCOA1 (SRC1)
- Homozygous, heterozygous, or compound heterozygous variant in SH2B1 gene, or chromosomal 16p11.2 deletion encompassing the SH2B1 gene
- Between 6 and 65 years of age at the time of provision of informed consent/assent
- 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
- 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
- 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
- Agree to use a highly effective form of contraception throughout the study and for 90 days following the study
- Reported history of lifestyle intervention of diet and exercise
- Reported history of hyperphagia Key
Exclusion Criteria:
- Weight loss of 2% or greater in the previous 3 months
- Recent history of bariatric surgery
- Significant psychiatric disorder(s)
- Suicidal ideation, attempt or behavior
- Clinically significant pulmonary, cardiac, endocrine/metabolic, hepatic or oncologic disease
- Glycated hemoglobin (HbA1C) >10% at Screening
- History of significant liver disease or severe kidney disease
- History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing
- Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide
- Significant hypersensitivity to any excipient in the study drug
- If female, pregnant or breastfeeding
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Conditions:
- Obesity