Ph 2 Trial to Evaluate Safety & Efficacy of RM-493 in Obese Patients With Prader-Willi Syndrome
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|ClinicalTrials.gov Identifier: NCT02311673|
Recruitment Status : Completed
First Posted : December 8, 2014
Last Update Posted : February 9, 2018
The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injectable formulation of RM-493 in obese subjects with Prader-Willi syndrome on tolerability, weight loss and hyperphagia-related behavior. The study drug (RM-493 and placebo) will be administered in a blinded fashion.
Funding Source - FDA OOPD
|Condition or disease||Intervention/treatment||Phase|
|Prader-Willi Syndrome||Drug: RM-493 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||40 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Ph 2, Randomized, Double-Blind, Placebo-controlled Pilot Study to Assess the Effects of RM-493, a Melanocortin 4 Receptor (MC4R) Agonist, in Obese Subjects With Prader-Willi Syndrome (PWS) on Safety, Weight Reduction, and Food-Related Behaviors|
|Study Start Date :||February 2015|
|Actual Primary Completion Date :||November 2016|
|Actual Study Completion Date :||December 2016|
Active Comparator: RM-493 Once Daily Dose 1
Dose 1 once daily in the morning
Other Name: setmelanotide
Active Comparator: RM-493 Once Daily Dose 2
Dose 2 once daily in the morning
Other Name: setmelanotide
Placebo Comparator: Placebo
Placebo in the morning
- Assess safety and tolerability of RM-493 (adverse events and clinical laboratory evaluations) [ Time Frame: Baseline to Day 70 or until any adverse events are resolved ]Assessment of adverse events and clinical laboratory evaluations.
- Effect on weight loss [ Time Frame: Baseline to Day 56 ]Measurement of the effect of RM-493 on weight loss.
- Effect on hyperphagia-related behavior (using the PWS Hyperphagia Questionnaire) [ Time Frame: Baseline to Day 56 ]Measurement of the effect of RM-493 on hyperphagia-related behavior using the PWS Hyperphagia Questionnaire.
- Effect on quality of life. [ Time Frame: Baseline to Day 56 ]Measurement of the effect of RM-493 on quality of life as measured by thee Food Related Problem Questionnaire and/or the Hospital Anxiety and Depression Questionnaire
- Effect on food-related behavior. (daily journal and/or Rate of Eating Questionnaire) [ Time Frame: Baseline to Day 56 ]Measurement of the effect of RM-493 on food-related behavior. A daily journal will be used to quantify aberrant behavior and/or a Rate of Eating Questionnaire will be used
- Effect on psychiatric status. (C-SSRS and PHQ-9 Questionnaires) [ Time Frame: Baseline to Day 70 ]Measurement of the effect of RM-493 on psychiatric status as assessed using the C-SSRS and PHQ-9 Questionnaires.
- Effect of on Pharmacokinetics (PK) (Frequent PK (trough) sampling) [ Time Frame: Baseline to Day 70 ]Frequent PK (trough) sampling throughout each stage of the study, including a 12-hour PK profile.
- Effect of dose adjustment of RM-493 [ Time Frame: Baseline to Day 56 ]Measurement of the effect of dose adjustment of RM-493 as assessed by measuring weight fluctuations from Baseline to Day 56.
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): NCT02311673
|United States, California|
|University of California Irvine|
|Irvine, California, United States, 92617|
|United States, Florida|
|University of Florida|
|Gainesville, Florida, United States, 32610|
|United States, Kansas|
|Kansas University Medical Center|
|Kansas City, Kansas, United States, 66160|
|United States, New York|
|Winthrop University Hospital|
|Mineola, New York, United States, 11501|
|United States, Tennessee|
|Nashville, Tennessee, United States, 37203|
|Study Director:||Fred Fiedorek, MD||Rhythm Pharmaceuticals, Inc.|