Study of PhentermineTopiramate for Treating Hypothalamic Obesity in Children, Adolescents, and Young Adults Aged 6 to 28 Years

What this Trial Is About

Researchers are evaluating the safety and effects of phenterminetopiramate PhT in children, adolescents, and young adults aged 6 to 28 years with hypothalamic obesity HO, a serious condition often caused by hypothalamic brain tumors. This form of obesity is difficult to treat and linked to higher risks of metabolic problems and early death from heart-related diseases. Phenterminetopiramate is FDA-approved for common obesity but has not been tested in HO before. This Phase 2, double-blind, placebo-controlled trial aims to gather initial data on safety, dosing, and weight loss effects in this group. Participants will be randomly assigned to one of two groups one receiving the active drug phenterminetopiramate with FDA-approved dose escalation until the highest tolerable dose is reached, and the other receiving placebo capsules that look identical. The treatment period lasts 28 weeks, followed by a one-week taper for those on the highest dose. The study is conducted at two centers and uses a double-blind design to compare changes between the active drug and placebo groups. During the trial, participants will attend a screening visit and five study visits at weeks 1, 3, 14, 16, and 28, with some visits possibly conducted remotely. There will be a remote contact after dose starts or increases to check tolerance and an additional follow-up at week 29 to assess withdrawal effects. Researchers will monitor adverse events, dosing tolerability, and changes in body mass index BMI, fat mass, and hunger. Participants will be compensated for their time and travel. The total study duration for participants is about 29 weeks.

Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity