Phentermine/Topiramate in Children, Adolescents, and Young Adults With Hypothalamic Obesity: a Pilot and Feasibility Study
Seattle Children's Hospital
Summary
Hypothalamic obesity (HO) refers to the substantial weight gain that often complicates hypothalamic brain tumors. Children with this treatment-recalcitrant form of obesity have excess rates of metabolic sequelae compared to otherwise healthy children with similar obesity, and later experience excess mortality related to cardiometabolic disease. In this pilot trial, our objective is to gather key preliminary data about phentermine/topiramate (Ph/T) that is FDA-approved for "common" obesity but has never been tested in HO. The subset of individuals with HO who experience hyperphagia or excess daytime sleepiness may benefit from the Ph/T-induced decrease in appetite and increase in alertness. Preliminary assessments of safety, adverse events, dosing (Aim 1), as well as of efficacy (% BMI loss, Aim 2) will be made in a 28-week parallel-arm double-blinded Phase 2 placebo-controlled clinical trial in 6-28-year-old individuals with HO.
Description
STUDY OVERVIEW This is a two-center, double-blind (participant and assessor), randomized, parallel-arm 28-week clinical phase II trial, comparing changes from pre- to post treatment in two study arms of active drug (Qsymia®) vs. placebo capsules. Twenty-four 6-28-year-old participants will be randomized 1:1 into the two intervention groups. The study will have a single central IRB (CHOP). In this pilot trial, the objective is to gather key preliminary data about phentermine/topiramate (Ph/T), a promising option containing a sympathomimetic amine (Ph) combined with an appetite-suppressive epil…
Eligibility
- Age range
- 6–28 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Males and Females; Ages 6-28 years (inclusive) 2. History of rapid weight gain related to tumor onset or treatment, as assessed by an experienced endocrinologist (for example, change in BMI z-score \> 0.2 and/or BMI +5% during the first 6 months following tumor treatment) 3. Obesity (BMI \> 95th%ile for age/sex using CDC 2000 reference for under 18; BMI \> 30 kg/m2 for 18+ years) 4. Recent evidence of hypothalamic injury by brain MRI with central review; \>6 months status-post definitive therapy (surgery, chemotherapy, or radiation); no major operations/surgeries planne…
Interventions
- DrugPhentermine / Topiramate Extended Release Oral Capsule [Qsymia]
To assess safety and maximum tolerated dose as well as efficacy on weight loss of Phentermine/Topiramate in individuals with hypothalamic obesity.
- OtherPlacebo
To assess safety and maximum tolerated dose as well as efficacy on weight loss of placebo treatment in individuals with hypothalamic obesity.
Locations (2)
- The Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Seattle Children'sSeattle, Washington