Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6-28
SponsorSeattle Children's Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Males and Females; Ages 6-28 years (inclusive)
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)
Obesity (BMI > 95th%ile for age/sex using CDC 2000 reference for under 18; BMI > 30 kg/m2 for 18+ years)
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 planned during the study period.
Disqualifiers
Contraindication to Phentermine, Topiramate, or Qsymia as assessed using current package inserts. Including: History of glaucoma and known hyperthyroidism.
Known history of nephrolithiasis (kidney stones).
Current clinical diagnosis of anorexia nervosa or bulimia nervosa in the medical record.
Known history of metabolic acidosis, low bicarbonate on screening laboratory assessment (below lower limit of normal), or clinically significant bone disease requiring medication (beyond calcium and/or vitamin D).
Trial design
Design model
Parallel
Treatments tested in this trial
Phentermine / Topiramate Extended Release Oral Capsule [Qsymia]
DrugTo assess safety and maximum tolerated dose as well as efficacy on weight loss of Phentermine/Topiramate in individuals with hypothalamic obesity.
Placebo
Other interventionTo assess safety and maximum tolerated dose as well as efficacy on weight loss of placebo treatment in individuals with hypothalamic obesity.
Treatment groups
24 Participants
are divided into 2 treatment groupsGroup A: Drug InterventionExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Treatment-emergent adverse events
Incidence of treatment-emergent adverse events including any during withdrawal in study drug vs. placebo.
Time frame
From baseline to completion of week 28Secondary outcomes
1
Maximum tolerated dose
Maximum tolerated dose achieved by the end of the study in study drug vs. placebo.
Time frame
Week 0 to 282
% change in BMI
% change in BMI in response to study drug vs. placebo.
Time frame
Week 0 to 283
Proportion of individuals who experience 5% decrease in BMI
Proportion of individuals who experience 5% decrease in BMI in response to study drug vs. placebo.
Time frame
Week 0 to 284
Proportion of individuals who experience 2.5% decrease in BMI
Proportion of individuals who experience 2.5% decrease in BMI in response to study drug vs. placebo
Time frame
Week 0 to 28Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Seattle Children's Hospital
Lead sponsor
Children's Hospital of Philadelphia
Collaborator
Source ClinicalTrials.gov