A Phase 2, Double Blind, Randomized, Placebo-controlled Crossover Trial to Evaluate the Efficacy and Safety of Atomoxetine in Adults With Melanocortin Obesity Syndrome
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This is a phase 2 randomized placebo-controlled crossover trial to determine the safety and efficacy of atomoxetine for treating obesity caused by loss-of-function variants in the melanocortin-4 receptor (MC4R), the most common cause of genetic obesity disorders. Atomoxetine was selected for this pilot trial because it has been shown to increase brain-derived neurotrophic factor (BDNF) within the central nervous system and in peripheral circulation. Targeting BDNF is a specific strategy for treating MC4R abnormalities because BDNF functions as a downstream mediator of MC4R signaling.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• Age 6 years and above
• Documented MC4R variant classified as pathogenic, likely pathogenic, or variant of uncertain significance per ACMG criteria. If testing was done in a research lab, it will be confirmed by a CLIA-approved lab prior to randomization.
• Obesity defined as BMI ≥30 kg/m2 in adults or ≥95th percentile for age and sex in children
Locations
United States
New York
Mount Sinai Hospital
RECRUITING
New York
Ohio
Nationwide Children's Hospital
NOT_YET_RECRUITING
Columbus
Tennessee
Vanderbilt University Medical Center
NOT_YET_RECRUITING
Nashville
Contact Information
Primary
Joan C Han, MD
joan.han@mountsinai.org
212-241-3744
Time Frame
Start Date:2026-09-01
Estimated Completion Date:2028-06
Participants
Target number of participants:20
Treatments
Placebo_comparator: Placebo then Atomoxetine
Participants receive placebo for 16 weeks (4 weeks titration + 12 weeks at target dose), followed by washout period, then atomoxetine for 16 weeks (4 weeks titration + 12 weeks at target dose).
Active_comparator: Atomoxetine then Placebo
Participants receive atomoxetine for 16 weeks (4 weeks titration + 12 weeks at target dose), followed by washout period, then placebo for 16 weeks (4 weeks titration + 12 weeks at target dose).