Setmelanotide for the Treatment of Early-Onset POMC Deficiency Obesity
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|ClinicalTrials.gov Identifier: NCT02896192|
Recruitment Status : Active, not recruiting
First Posted : September 12, 2016
Last Update Posted : April 14, 2020
|Condition or disease||Intervention/treatment||Phase|
|Pro-opiomelanocortin (POMC) Deficiency Obesity||Drug: Setmelanotide Drug: Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of RM-493, a MC4R Agonist, in Early Onset POMC Deficiency Obesity Due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic Mutation|
|Actual Study Start Date :||February 14, 2017|
|Estimated Primary Completion Date :||July 30, 2020|
|Estimated Study Completion Date :||July 30, 2020|
Setmelanotide subcutaneous injection once daily
RM-493 once daily subcutaneous injection
Other Name: RM-493
Placebo Comparator: Placebo
Placebo subcutaneous injection once daily
During the double blind placebo withdrawal period, patients will receive RM-493 or placebo at variable times over an 8 week period in order for patients to serve as their own control.
- Effect on weight loss [ Time Frame: 1 year ]Measurement of the effect of RM-493 on weight loss.
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 1 year ]Assessment of Adverse Events related to treatment
- Effect on Body Fat Mass [ Time Frame: 1 year ]Assessment of body composition as measured by bioelectrical impedance (BIA) or Dual-energy x-ray absorptiometry (DXA).
- Effect on Hunger [ Time Frame: 1 year ]Assessment of hunger using a Hunger Questionnaire.
- Improvements in insulin resistance [ Time Frame: 1 year ]Assessment of fasting glucose, glycated hemoglobin (HbA1c), and oral glucose tolerance test (OGTT).
- Effect on Waist Circumference [ Time Frame: 1 year ]Assessment of waist circumference.
- Reversal of weight during the double-blind placebo controlled withdrawal phase [ Time Frame: 8 weeks ]Assessment of weight regain during the double blind withdrawal phase
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): NCT02896192
|United States, Arizona|
|Honor Health Research Institute|
|Scottsdale, Arizona, United States, 85258|
|Gent, Belgium, 9000|
|Peel Memorial Hospital|
|Brampton, Ontario, Canada, L6W 2Z8|
|Institute of Cardiometabolism and Nutrition / Hopital de la Pitié-Salpêtrière|
|Paris, France, 75013|
|Charité Campus Virchow-Klinikum / Institute for Experimental Pediatric Endocrinology|
|Berlin, Germany, 13353|
|Hospital Universitario Niño Jesús|
|Madrid, Spain, 28009|
|University of Cambridge Metabolic Research Laboratories|
|Cambridge, United Kingdom, CB2 0QQ|
|Study Chair:||Liz Stoner||Rhythm Pharmceuticals, Inc.|