Genetic Obesity Study
Protocol: STUDY00003292
Full Title
Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with Rare Genetic Disorders of Obesity (RM-493-014)
Description
This study enrolls adults and children ages 12+ who have obesity due to rare genetic disorders. The purpose of this study is to learn whether treatment with the study drug, setmelanotide, helps people with those disorders lose weight.

You will be in this study for about 6.5 months. You will take the study drug for about 4 months during that time. The drug is taken by a daily injection under the skin. You will have physical exams, urine tests, and blood tests during the study. It is possible to continue treatment with setmelanotide by entering into an extension study after participation in this study ends. For more information about this study, please contact ahouse@upa.chob.edu. Study takes place at UBMD Pediatrics, 1001 Main Street, 5th Floor, Buffalo, NY 14203.

Technical Description

Primary Objective: To demonstrate clinically meaningful effects of setmelanotide, after 3 months of treatment, on percent body weight change in each type of these rare genetic disorders of obesity included in this protocol.

Secondary Objective: To assess the effect of setmelanotide after 3 months of treatment on:
-Safety and tolerability of setmelanotide (including blood pressure [BP] and heart rate [HR]).
-Hunger.
-Percent change in body fat mass.
-Glucose parameters: fasting glucose, fasting insulin, glycated hemoglobin (HbA1c), oral glucose tolerate test (OGTT) with focus on parameters of insulin sensitivity.
-Waist circumference.

Secondary (For patients who continue into the long-term extension)
To assess the effect of setmelanotide after 6 and 12 months of treatment on:
-Safety and tolerability of setmelanotide (including blood pressure [BP] and heart rate [HR]).
-Hunger.
-Percent change in body fat mass.
-Glucose parameters: fasting glucose, fasting insulin, glycated hemoglobin (HbA1c), oral glucose tolerate test (OGTT) with focus on parameters of insulin sensitivity. Note: OGTT is not necessary in patients with confirmed type 1 or type 2 diabetes.
-Waist circumference.
-For consenting patients who agree to participate in a withdrawal phase: during withdrawal from drug, evaluate reversal of weight and hunger reduction.

Tertiary
To assess the effect of setmelanotide on:
-Percent change in total body mass, non-bone lean mass, and bone density.
-Fasting lipid (cholesterol and triglyceride) panel.
-Pharmacokinetics of setmelanotide.
-C-reactive protein.
-Dose response of setmelanotide through titration procedures.
-Changes in quality of life and health status.
Compensation: Yes
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Compensation may include cash, checks, gift cards, debit cards, or incentives like gift baskets, technology items, or merchandise.
Eligibility
Adults and children ages 12+.
Rare genetic disease patient with genetically confirmed diagnosis.
Obese (measured by BMI).
Age Group: Both Adults and Children
Principal Investigator: INDRAJIT MAJUMDAR
ClinicalTrials.gov: Open Study
Contact(s)
AMANDA (AYLER) HOUSE
ajayler@buffalo.edu
+1 716-323-0075
Want to Learn More?
Let us know how the study team can reach you. If you do not hear back within 2 business days, reach out to the study team directly at the contact information above or email ctsiresearch@buffalo.edu and someone will assist you.