Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
2 other identifiers
interventional
213
9 countries
58
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Feb 2017
Longer than P75 for phase_2
58 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 3, 2017
CompletedFirst Posted
Study publicly available on registry
January 6, 2017
CompletedStudy Start
First participant enrolled
February 10, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2022
CompletedResults Posted
Study results publicly available
August 18, 2023
CompletedAugust 18, 2023
July 1, 2023
5.1 years
January 3, 2017
July 25, 2023
July 25, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants With ≥ 5% Reduction in Body Weight From Baseline After 3 Months of Setmelanotide Treatment
Baseline to Month 3
Secondary Outcomes (8)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
From first dose up to Month 16
Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment
Baseline, Month 3
Percent Change From Baseline in Body Weight After 3 Months of Setmelanotide Treatment
Baseline, Month 3
Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged ≥ 12 Years
Baseline, Month 3
Change From Baseline in Daily Hunger Questionnaire Scores After 3 Months of Setmelanotide Treatment in Participants Aged < 12 Years
Baseline, Month 3
- +3 more secondary outcomes
Study Arms (10)
16p11.2 Cohort
EXPERIMENTALParticipants with chromosomal rearrangement of the p11.2 region of chromosome 16 (16p11.2) locus causing obesity received setmelanotide once daily (QD) via subcutaneous (SC) injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
AS Cohort
EXPERIMENTALParticipants with Alström syndrome (AS) received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
BBS Cohort
EXPERIMENTALParticipants with Bardet-Biedl syndrome (BBS) received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
MC4R Cohort
EXPERIMENTALParticipants with melanocortin-4 receptor (MC4R) deficiency obesity received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
POMC/PCSK1/LEPR Heterozygous Cohort
EXPERIMENTALParticipants with pro-opiomelanocortin (POMC)/proprotein convertase subtilisin/kexin type 1 (PCSK1)/leptin receptor (LEPR) heterozygous mutations received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
POMC/PCSK1/LEPR Composite Heterozygous Cohort
EXPERIMENTALParticipants with POMC/PCSK1/LEPR composite heterozygous mutations received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
POMC/PCSK1/LEPR Compound Heterozygous Cohort
EXPERIMENTALParticipants with POMC/PCSK1/LEPR compound heterozygous mutations received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
SH2B1 Cohort
EXPERIMENTALParticipants with steroid receptor coactivator (SRC) homology 2B adapter protein 1 (SH2B1) haploinsufficiency received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
SMS Cohort
EXPERIMENTALParticipants with Smith-Magenis Syndrome (SMS) received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
SRC1 Cohort
EXPERIMENTALParticipants with steroid receptor coactivator 1 (SRC1) mutations received setmelanotide QD via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and the dose was escalated up to a maximum dose of 3.0 mg QD. Participants either continued setmelanotide treatment by enrolling in an extension study (RM-493-022; NCT03651765) immediately following the last dose in this study or if the extension study was not open at the current clinic site, participants continued treatment in the current study for up to 1 year, resulting in treatment duration of up to 16 months.
Interventions
RM-493 QD SC injection
Eligibility Criteria
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.
- +1 more criteria
You may not qualify if:
- 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
- Glomerular filtration rate (GFR) \< 30 milliliter/minute (mL/min) at Screening.
- History or close family history of melanoma or participant history of oculocutaneous albinism
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
- Participants previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
- Inability to comply with QD injection regimen.
- Females who are breastfeeding or nursing.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (58)
Synexus Clinical Research US, Inc. - Simon Williamson Clinic, PC
Birmingham, Alabama, 35211, United States
Synexus Clinical Research US, Inc. - Phoenix Southeast
Chandler, Arizona, 85224, United States
Synexus Clinical Research US, Inc. - Central Arizona Medical Associates, PC
Mesa, Arizona, 85206, United States
Honor Health Research Institute
Scottsdale, Arizona, 85258, United States
Axis Clinical Trials-Downtown
Los Angeles, California, 90017, United States
Axis Clinical Trials Headquarters
Los Angeles, California, 90036, United States
San Diego Wake Research
San Diego, California, 92108, United States
Anschutz Health and Wellness Center University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
Division of Endocrinology and Diabetes Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
University of Florida College of Medicine
Gainesville, Florida, 32610, United States
AXIS South Florida Clinical Trials
Hialeah, Florida, 33016, United States
Florida Hospital
Orlando, Florida, 32804, United States
Synexus Clinical Research US, Inc. - St. Petersburg
Pinellas Park, Florida, 33781, United States
Synexus Clinical Research US, Inc. - Chicago
Chicago, Illinois, 60602, United States
Maine Medical Partners
Portland, Maine, 04102, United States
NIH Hatfield Clinical Research Center
Bethesda, Maryland, 20892, United States
Baystate Medical Center
Springfield, Massachusetts, 01107, United States
University of Michigan Medicine
Ann Arbor, Michigan, 48105, United States
Precision Medicine for Obesity Research: Gastroenterology & Hepatology Mayo Clinic
Rochester, Minnesota, 55905, United States
Washington University St. Louis
St Louis, Missouri, 63110, United States
Impact Clinical Trials
Las Vegas, Nevada, 89106, United States
AXIS New York Clinical Trials
Brooklyn, New York, 11201, United States
University at Buffalo
Buffalo, New York, 14203, United States
AXIS Clinical Trials
New York, New York, 10022, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10025, United States
Columbia University
New York, New York, 10032, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Wake Research Inc.
Raleigh, North Carolina, 27612, United States
Synexus Clinical Research US, Inc. - Akron
Akron, Ohio, 44311, United States
Synexus Clinical Research US, Inc. - Cincinnati
Cincinnati, Ohio, 45236, United States
Synexus Clinical Research US, Inc. - Columbus
Columbus, Ohio, 43016, United States
Obesity Institute, Geisinger Clinic
Danville, Pennsylvania, 17822, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Synexus Clinical Research US, Inc. - Primary Care Associates, PC
Anderson, South Carolina, 29621, United States
Wake Research TN
Chattanooga, Tennessee, 37421, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103, United States
Vanderbilt University School of Medicine
Nashville, Tennessee, 37212-3157, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Synexus Clinical Research US, Inc. - Plano
Plano, Texas, 75093, United States
Synexus Clinical Research US, Inc. - San Antonio
San Antonio, Texas, 78229, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Seattle Children's Research Institute
Seattle, Washington, 98101, United States
Marshfield Clinic Research Institute
Marshfield, Wisconsin, 54449, United States
University of Alberta
Edmonton, T6G 2E1, Canada
Hopital Trousseau - Nutrition et Gastroentérologie
Paris, 75012, France
Hopital de la Pitié-Salpêtrière
Paris, 75013, France
Service de pédiatrie CHU de la Réunion - Hôpital Félix Guyon
Saint-Denis, 97405, France
Charité Berlin
Berlin, 13354, Germany
University of Leipzig
Leipzig, 04103, Germany
University of Ulm
Ulm, 89075, Germany
University General Hospital of Patras
Rio, Patras, 26504, Greece
Edmond and Lily Safra Children's Hospital
Ramat Gan, 52621, Israel
Erasmus MC
Rotterdam, 3015 CE, Netherlands
Hospital Infantil Universitario Niño Jesús
Madrid, 65 28009, Spain
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH, United Kingdom
Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
Hammersmith Hospital
London, W12 0NN, United Kingdom
Hammersmith Hospital
London, United Kingdom
Related Publications (2)
Ervin C, Norcross L, Mallya UG, Fehnel S, Mittleman RS, Webster M, Haqq AM, Haws RM. Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome. Adv Ther. 2023 May;40(5):2394-2411. doi: 10.1007/s12325-023-02443-y. Epub 2023 Mar 24.
PMID: 36961653DERIVEDMeyer JR, Krentz AD, Berg RL, Richardson JG, Pomeroy J, Hebbring SJ, Haws RM. Kidney failure in Bardet-Biedl syndrome. Clin Genet. 2022 Apr;101(4):429-441. doi: 10.1111/cge.14119.
PMID: 35112343DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Rhythm Clinical Trials
- Organization
- Rhythm Pharmaceuticals, Inc.
Study Officials
- STUDY CHAIR
David Meeker, MD
Rhythm Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 3, 2017
First Posted
January 6, 2017
Study Start
February 10, 2017
Primary Completion
March 1, 2022
Study Completion
March 1, 2022
Last Updated
August 18, 2023
Results First Posted
August 18, 2023
Record last verified: 2023-07
Data Sharing
- IPD Sharing
- Will not share