Hypothalamic obesity is a severe, treatment-refractory obesity syndrome caused by hypothalamic injury that disrupts appetite and energy homeostasis. It is most commonly observed after tumors or treatment affecting hypothalamic structures, especially craniopharyngioma. Standard lifestyle measures are often insufficient, and management is challenging.
Rhythm Pharmaceuticals announced expanded Phase 3 TRANSCEND trial data showing an 18.8% placebo-adjusted BMI reduction at 52 weeks for setmelanotide in acquired hypothalamic obesity, with FDA PDUFA goal date set for March 20, 2026.
Amgen, Celltrion, and Palatin Technologies are advancing obesity drug candidates through clinical development, with trials planned for 2026-2028 targeting liver fat reduction, multiple biological pathways, and rare genetic obesity conditions.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07156578 |
A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO) |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT06901245 |
Tirzepatide in PWS, HO and GNSO |
RECRUITING | PHASE4 |
| NCT06760546 |
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756) |
RECRUITING | PHASE3 |
| NCT06299891 |
Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity |
RECRUITING | PHASE2 |
| NCT06239116 |
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment |
RECRUITING | PHASE1/PHASE2 |
| NCT06217848 |
The Effect of GLP-1 Agonist in Patients With Hypothalamic Obesity: Prospective, Pilot Study |
RECRUITING | EARLY_PHASE1 |
| NCT06046443 |
A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity |
COMPLETED | PHASE2 |
| NCT05774756 |
A Trial of Setmelanotide in Acquired Hypothalamic Obesity |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05319301 |
Identification and Clinical Relevance of an Oxytocin Deficient State (Melatonin Study) |
COMPLETED | NA |
| NCT05147415 |
Study of Tesomet With Open-label Extension in Subjects With Hypothalamic Obesity (HO) |
WITHDRAWN | PHASE2 |
