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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03013543
Recruitment Status : Completed
First Posted : January 6, 2017
Last Update Posted : December 27, 2022
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Genetic Obesity Obesity Obesity Due to Melanocortin 4 Receptor Deficiency Drug: Setmelanotide Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 213 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
Actual Study Start Date : February 10, 2017
Actual Primary Completion Date : March 1, 2022
Actual Study Completion Date : March 1, 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Setmelanotide
Setmelanotide subcutaneous injection once daily
Drug: Setmelanotide
RM-493 once daily subcutaneous injection
Other Name: RM-493

Primary Outcome Measures :
  1. Effect of Setmelanotide on Body Weight Reduction [ Time Frame: Baseline to 3 months ]
    The proportion of patients in each subgroup of RGDO who achieve at least 5% body weight reduction from baseline at ~3 months treatment with setmelanotide.

Secondary Outcome Measures :
  1. Change and percentage change in body weight [ Time Frame: Baseline to 3 months ]
    Change and percentage change from baseline in body weight

  2. Change in daily most hunger scores [ Time Frame: Baseline to 3 months ]
    Change in the weekly average of the daily hunger score from Baseline at 3 months

  3. Change in waist circumference [ Time Frame: Baseline to 3 months ]
    Change from baseline in waist circumference

  4. Mean percent change in BMI [ Time Frame: Baseline to 3 months ]
    Mean percent change in body mass index from baseline at 3 months

  5. Mean change in BMI-Z score in patients <12 years old [ Time Frame: Baseline to 3 months ]
    Mean change in BMI-Z score from Baseline at 3 months

  6. Mean change in BMI-Z score in patients ≥12 to <18 years old [ Time Frame: Baseline to 3 months ]
    Mean change in BMI-Z score in patients ≥12 to <18 years old from Baseline at 3 months

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients with the following genotypes and/or clinical assessment:

    1. POMC/PCSK1/LEPR heterozygous - not currently enrolling new patients
    2. POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or homozygous deficiency obesity
    3. POMC/PCSK1/LEPR composite heterozygous (two or more mutations in two or more genes) deficiency obesity
    4. Smith-Magenis Syndrome (SMS)
    5. SH2B1 deficiency obesity
    6. Chromosomal rearrangement of the 16p11.2 locus causing obesity
    7. CPE compound heterozygous or homozygous deficiency obesity
    8. Leptin deficiency obesity with loss of response to metreleptin
    9. SRC1 deficiency obesity
    10. MC4R deficiency obesity
  2. Age 6 years and above
  3. Obese, defined as Body Mass Index (BMI) ≥ 30 kg/m2 for patients ≥16 years of age or BMI≥ 95th percentile for age and gender for patients 6 up to 16 years of age.
  4. 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
  5. Female participants of child-bearing potential must be confirmed non-pregnant, and agree to use contraception as outlined in the protocol.
  6. Male participants with female partners of childbearing potential must agree to a double barrier method if they become sexually active during the study. Male patients must not donate sperm during and for 90 days following their participation in the study.

Key Exclusion Criteria:

  1. 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.
  2. 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).
  3. Gastric bypass surgery within the previous six months or any prior gastric bypass surgery resulting in >10% weight loss durably maintained
  4. Diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
  5. Suicidal ideation, attempt or behavior
  6. Clinically significant pulmonary, cardiac, or oncologic disease
  7. HbA1c >9.0% at Screening
  8. History of significant liver disease
  9. Glomerular filtration rate (GFR) <30 mL/min at Screening.
  10. History or close family history of melanoma or patient history of oculocutaneous albinism
  11. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
  12. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
  13. Patients previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
  14. Inability to comply with QD injection regimen.
  15. Females who are breastfeeding or nursing.

Other protocol defined Inclusion/Exclusion criteria may apply.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03013543

Hide Hide 58 study locations
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United States, Alabama
Synexus Clinical Research US, Inc. - Simon Williamson Clinic, PC
Birmingham, Alabama, United States, 35211
United States, Arizona
Synexus Clinical Research US, Inc. - Phoenix Southeast
Chandler, Arizona, United States, 85224
Synexus Clinical Research US, Inc. - Central Arizona Medical Associates, PC
Mesa, Arizona, United States, 85206
Honor Health Research Institute
Scottsdale, Arizona, United States, 85258
United States, California
Axis Clinical Trials-Downtown
Los Angeles, California, United States, 90017
Axis Clinical Trials Headquarters
Los Angeles, California, United States, 90036
San Diego Wake Research
San Diego, California, United States, 92108
United States, Colorado
Anschutz Health and Wellness Center University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States, 80045
United States, District of Columbia
Division of Endocrinology and Diabetes Children's National Hospital
Washington, District of Columbia, United States, 20010
United States, Florida
University of Florida College of Medicine
Gainesville, Florida, United States, 32610
AXIS South Florida Clinical Trials
Hialeah, Florida, United States, 33016
Florida Hospital
Orlando, Florida, United States, 32804
Synexus Clinical Research US, Inc. - St. Petersburg
Pinellas Park, Florida, United States, 33781
United States, Illinois
Synexus Clinical Research US, Inc. - Chicago
Chicago, Illinois, United States, 60602
United States, Maine
Maine Medical Partners
Portland, Maine, United States, 04102
United States, Maryland
NIH Hatfield Clinical Research Center
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Baystate Medical Center
Springfield, Massachusetts, United States, 01107
United States, Michigan
University of Michigan Medicine
Ann Arbor, Michigan, United States, 48105
United States, Minnesota
Precision Medicine for Obesity Research: Gastroenterology & Hepatology Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
Washington University St. Louis
Saint Louis, Missouri, United States, 63110
United States, Nevada
Impact Clinical Trials
Las Vegas, Nevada, United States, 89106
United States, New York
AXIS New York Clinical Trials
Brooklyn, New York, United States, 11201
University at Buffalo
Buffalo, New York, United States, 14203
AXIS Clinical Trials
New York, New York, United States, 10022
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10025
Columbia University
New York, New York, United States, 10032
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
Wake Research Inc.
Raleigh, North Carolina, United States, 27612
United States, Ohio
Synexus Clinical Research US, Inc. - Akron
Akron, Ohio, United States, 44311
Synexus Clinical Research US, Inc. - Cincinnati
Cincinnati, Ohio, United States, 45236
Synexus Clinical Research US, Inc. - Columbus
Columbus, Ohio, United States, 43016
United States, Pennsylvania
Obesity Institute, Geisinger Clinic
Danville, Pennsylvania, United States, 17822
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Synexus Clinical Research US, Inc. - Primary Care Associates, PC
Anderson, South Carolina, United States, 29621
United States, Tennessee
Wake Research TN
Chattanooga, Tennessee, United States, 37421
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Vanderbilt University School of Medicine
Nashville, Tennessee, United States, 37212-3157
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Synexus Clinical Research US, Inc. - Plano
Plano, Texas, United States, 75093
Synexus Clinical Research US, Inc. - San Antonio
San Antonio, Texas, United States, 78229
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
United States, Washington
Seattle Children's Research Institute
Seattle, Washington, United States, 98101
United States, Wisconsin
Marshfield Clinic Research Institute
Marshfield, Wisconsin, United States, 54449
University of Alberta
Edmonton, Canada, T6G 2E1
Hopital Trousseau - Nutrition et Gastroentérologie
Paris, France, 75012
Hopital de la Pitié-Salpêtrière
Paris, France, 75013
Service de pédiatrie CHU de la Réunion - Hôpital Félix Guyon
Saint-Denis, France, 97405
Charité Berlin
Berlin, Germany, 13354
University of Leipzig
Leipzig, Germany, 04103
University of Ulm
Ulm, Germany, 89075
University General Hospital of Patras
Río, Patras, Greece, 26504
Edmond and Lily Safra Children's Hospital
Ramat Gan, Israel, 52621
Erasmus MC
Rotterdam, Netherlands, 3015 CE
Hospital Infantil Universitario Niño Jesús
Madrid, Spain, 65 28009
United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2TH
Addenbrooke's Hospital
Cambridge, United Kingdom, CB2 0QQ
Hammersmith Hospital
London, United Kingdom, W12 0NN
Hammersmith Hospital
London, United Kingdom
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
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Study Chair: David Meeker, MD Rhythm Pharmaceuticals, Inc.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Rhythm Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03013543    
Other Study ID Numbers: RM-493-014
First Posted: January 6, 2017    Key Record Dates
Last Update Posted: December 27, 2022
Last Verified: December 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rhythm Pharmaceuticals, Inc.:
Pro-opiomelanocortin (POMC) deficiency obesity
LepR deficiency obesity
Smith-Magenis Syndrome
MC4R deficiency obesity
SRC1 deficiency obesity
SH2B1 deficiency obesity
Additional relevant MeSH terms:
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Genetic Diseases, Inborn
Nutrition Disorders
Body Weight