Atomoxetine for MC4R-related obesity

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, placebo-controlled crossover trial in participants aged 6 years and older with pathogenic MC4R variants and obesity. The study will assess safety and potential weight- and metabolic-related benefits of atomoxetine, a medication already approved for ADHD, given its ability to raise brain-derived neurotrophic factor (BDNF) and its role in MC4R signaling. Participants receive atomoxetine and placebo in alternating periods, with outcomes focused on energy balance and weight change. The trial leverages multiple sites, including Mount Sinai, Nationwide Children's Hospital, and Vanderbilt, to evaluate the targeted genetic subset of obesity. The goal is to determine whether this repurposed drug can address the specific energy-regulation defect seen in MCOS.

Why it matters

Who can join: the full rules

Inclusion criteria:

* 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

Exclusion criteria:

* Use of atomoxetine, viloxazine (another selective norepinephrine-reuptake inhibitor), methylphenidate, amphetamine, dextroamphetamine, lisdexamfetamine, phentermine, or any other stimulant medication in the past 30 days. If on other ADHD medications, such as guanfacine and clonidine, must be on a stable dose for \>3 months.
* Weight loss \>5% in the past 3 months.
* Initiation of new weight loss program, including diet or medications. If on weight loss medications, must be on a stable dose for \>3 months.
* Inability to swallow capsules.
* History of hypersensitivity to atomoxetine.
* Narrow angle glaucoma.
* History of pheochromocytoma.
* Uncontrolled Stage 2 hypertension (≥95th percentile + 12 mmHg or \>140/90, whichever is lower) at screening. If on antihypertensive medication, must be on stable dose for \>3 months.
* Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal)
* Uncontrolled asthma requiring albuterol more than once weekly over the past 3 months
* History of a cardiac arrhythmia (not including bradycardia)
* Current use of monoamine oxidase inhibitors
* Pregnancy or intention to become pregnant during the next year
* History of Major Depressive Disorder in the past 2 years, lifetime history of suicide attempt, history of any suicidal behavior in the past month, history of other severe psychiatric disorders (e.g. schizophrenia, bipolar disorder)
* PHQ-9 score is ≥15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month
* Unable to comply with study procedures in the opinion of the investigator

Where it's running

• Mount Sinai Hospital
  New York, New York, United States Recruiting
• Nationwide Children's Hospital
  Columbus, Ohio, United States Not yet recruiting
• Vanderbilt University Medical Center
  Nashville, Tennessee, United States Not yet recruiting

Study contacts

• Principal investigator: Joan C Han, MD — Mount Sinai Kravis Children's Hospital
• Study coordinator: Joan C Han, MD
• Email: joan.han@mountsinai.org
• Phone: 212-241-3744

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.