Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

Completed · Phase 2

Conditions studied: Genetic Obesity, Obesity, Obesity Due to Melanocortin 4 Receptor Deficiency

In brief

The purpose of the study was to determine the effect of setmelanotide (RM-493) on weight, hunger assessments, and other factors in participants with rare genetic disorders of obesity.

Key facts

Study ID

NCT03013543

Run by

Rhythm Pharmaceuticals, Inc.

People needed

213

Starts

2017-02-10

Expected to finish

2022-03-01

Last updated by the study team

2023-08-18

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants with the following genotypes and/or clinical assessment:
• POMC/PCSK1/LEPR heterozygous - not currently enrolling new participants
• POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or homozygous deficiency obesity
• POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more genes) deficiency obesity
• SMS
• SH2B1 deficiency obesity
• Chromosomal rearrangement of the 16p11.2 locus causing obesity
• Carboxypeptidase E (CPE) compound heterozygous or homozygous deficiency obesity
• Leptin deficiency obesity with loss of response to metreleptin
• SRC1 deficiency obesity
• MC4R deficiency obesity
• Age 6 years and above
• Obese, defined as Body Mass Index (BMI) ≥ 30 kilogram per meter square (kg/m\^2) for participants ≥16 years of age or BMI≥ 95th percentile for age and gender for participants 6 up to 16 years of age.
• Participant 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
• Female participants of childbearing potential must be confirmed non-pregnant, and agree to use contraception as outlined in the protocol.
• Male participants with female partners of childbearing potential must agree to a double barrier method if they become sexually active during the study. Male participants must not donate sperm during and for 90 days following their participation in the study.
• Key Exclusion Criteria:
• Recent intensive (within 2 months) diet and/or exercise regimen with or without the use of weight loss agents that has resulted in > 2% weight loss.
• Use of any medication that is approved to treat obesity within three months of first dose of study drug (e.g., orlistat, lorcaserin, phentermine-topiramate, naltrexone-bupropion).
• Gastric bypass surgery within the previous six months or any prior gastric bypass surgery resulting in >10% weight loss durably maintained
• Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
• Suicidal ideation, attempt or behavior
• Clinically significant pulmonary, cardiac, or oncologic disease
• hemoglobin A1c (HbA1c) > 9.0% at Screening
• History of significant liver disease

Where it is running

• Synexus Clinical Research US, Inc. - Phoenix Southeast — Chandler, Arizona, United States
• Synexus Clinical Research US, Inc. - Central Arizona Medical Associates, PC — Mesa, Arizona, United States
• Honor Health Research Institute — Scottsdale, Arizona, United States
• Axis Clinical Trials-Downtown — Los Angeles, California, United States
• Axis Clinical Trials Headquarters — Los Angeles, California, United States
• San Diego Wake Research — San Diego, California, United States
• Anschutz Health and Wellness Center University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• Division of Endocrinology and Diabetes Children's National Hospital — Washington D.C., District of Columbia, United States
• University of Florida College of Medicine — Gainesville, Florida, United States
• AXIS South Florida Clinical Trials — Hialeah, Florida, United States
• Florida Hospital — Orlando, Florida, United States
• Synexus Clinical Research US, Inc. - St. Petersburg — Pinellas Park, Florida, United States
• Synexus Clinical Research US, Inc. - Chicago — Chicago, Illinois, United States
• Maine Medical Partners — Portland, Maine, United States
• NIH Hatfield Clinical Research Center — Bethesda, Maryland, United States
• Baystate Medical Center — Springfield, Massachusetts, United States
• University of Michigan Medicine — Ann Arbor, Michigan, United States
• Precision Medicine for Obesity Research: Gastroenterology & Hepatology Mayo Clinic — Rochester, Minnesota, United States
• Washington University St. Louis — St Louis, Missouri, United States
• Impact Clinical Trials — Las Vegas, Nevada, United States
• AXIS New York Clinical Trials — Brooklyn, New York, United States
• University at Buffalo — Buffalo, New York, United States
• AXIS Clinical Trials — New York, New York, United States
• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• Synexus Clinical Research US, Inc. - Simon Williamson Clinic, PC — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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