Current Clinical Trials and Newly Released Data Offering Hope for HO
For survivors of hypothalamic-pituitary tumors, especially craniopharyngioma, hypothalamic obesity (HO) is one of the most difficult and life-altering long-term effects. It is not driven by lifestyle choices, but by damage to the part of the brain responsible for regulating hunger, energy balance, and metabolism. Conventional diet and exercise strategies are often ineffective, leaving patients with few options and a sense of hopelessness.
That’s beginning to change.
Researchers and pharmaceutical developers are now focusing on HO, developing targeted treatments that reflect the unique biology and challenges of this condition. Below is a summary of clinical trials currently recruiting participants, as well as those that have completed key phases and released data. These studies are shaping the future of care for HO and could soon bring much-needed therapies to patients and families who have long been underserved.
Actively Recruiting Trials
Phentermine/Topiramate for Hypothalamic Obesity
This Phase 2 study investigates the use of a combination drug—phentermine, an appetite suppressant, and topiramate, which impacts craving and energy use—already FDA-approved for general obesity. The trial is evaluating its safety and effectiveness in people specifically diagnosed with hypothalamic obesity, often following treatment for brain tumors. This study reflects growing recognition that HO requires a different clinical approach.
More information: Trial link
RM-718 for Hypothalamic Obesity
RM-718 is a next-generation melanocortin-4 receptor (MC4R) agonist designed to reduce hunger and support healthy weight regulation without increasing blood pressure or heart rate. This Phase 1 study is focused on determining safe dosing and initial effects in individuals with HO. This trial represents an important step forward for a community that has historically lacked evidence-based pharmacological options.
More information: Trial link
Tirzepatide for Weight Loss in Prader-Willi Syndrome and Obesity
This trial is investigating tirzepatide, a dual GIP/GLP-1 receptor agonist, in already approved for type 2 diabetes and obesity, in PWS and HO. Its mechanism may also hold promise for individuals with HO. The symptoms, including hyperphagia and metabolic dysregulation, makes this trial highly relevant for the broader hypothalamic-pituitary brain tumor survivor community.
More information: Trial link
Trials with Completed Enrollment and Data Released
Setmelanotide (IMCIVREE®)
Setmelanotide, an MC4R agonist, has already shown clinical benefit in rare genetic obesity syndromes was trialed in 120 patients, the largest randomized controlled trial in HO to-date. Data was released recently at ENDO 2025 showing significant BMI reductions across age groups. These results are encouraging for both patients and clinicians.
Press release: Read the announcement
Bivamelagon (LB54640)
Bivamelagon is an oral MC4R agonist which recently concluded Phase 2 trials. This drug offers a once-daily pill option, potentially reducing treatment burden and improving accessibility. In the 14-week, double-blind, four-arm, placebo-controlled portion of the trial, bivamelagon achieved statistically significant and clinically meaningful reductions in body mass index (BMI), consistent with BMI reductions achieved with setmelanotide therapy
Press release: Read the announcement
Why This Matters
Clinical research is a vital step toward transforming the care landscape for hypothalamic obesity. These trials don’t just represent scientific progress—they represent hope. For families navigating a condition that has been misunderstood and neglected, the availability of new therapies rooted in brain-based biology could be life-changing.
At the Raymond A. Wood Foundation, we remain committed to advocating for research that reflects the lived experiences of our community. We will continue to share updates on these and other studies as data becomes available.
For more information or guidance on clinical trials, visit our clinical trial finder or join us in November for our Pituitary Brain Tumor Family Conference in Philadelphia.