Actively Recruiting
Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Led by Seattle Children's Hospital · Updated on 2025-07-28
24
Participants Needed
2
Research Sites
65 weeks
Total Duration
On this page
Sponsors
S
Seattle Children's Hospital
Lead Sponsor
C
Children's Hospital of Philadelphia
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males and females aged 6 to 28 years inclusive
- History of rapid weight gain related to tumor onset or treatment, confirmed by an endocrinologist
- Obesity defined as BMI above the 95th percentile for age and sex if under 18, or BMI above 30 kg/m2 if 18 or older
- Evidence of hypothalamic injury on brain MRI with central review
- At least 6 months since definitive tumor therapy (surgery, chemotherapy, or radiation)
- No major surgeries planned during the study
- Stable on pituitary replacement or appetite-modulating medications for at least 2 months, with limited hormone dose adjustments allowed
- Post-menarchal females must use effective contraception unless hypogonadotropic hypogonadism is documented
- Ability to communicate well, comply with study requirements, and provide informed consent or assent with parental consent if under 18
You will not qualify if you...
- Contraindications to phentermine, topiramate, or Qsymia including history of glaucoma or known hyperthyroidism
- History of kidney stones
- Current diagnosis of anorexia nervosa or bulimia nervosa
- History of metabolic acidosis, low bicarbonate, or significant bone disease requiring medication
- Use of monoamine oxidase inhibitors within 14 days prior to screening
- Known hypersensitivity to sympathomimetic amines
- Significant cardiovascular conditions including abnormal blood pressure, arrhythmias, heart failure, prolonged QTc interval, or coronary artery disease
- Females who are pregnant, breastfeeding, or planning pregnancy during the trial
- "Brittle" diabetes insipidus
- Diabetes mellitus requiring insulin or secretagogues or HbA1c over 8.5% at screening
- Clinically significant hyperthyroidism
- History or current clinically significant hypokalemia
- Significant liver or kidney disease
- History of seizure in the last 12 months
- History of substance abuse, moderate or severe depression, psychiatric disorder, or suicidality
- History of abdominal surgery including gastric bypass
- Current use of supra-physiologic steroids
- Allergy or sensitivity to test agents including aspirin or FD&C Yellow No. 5
- Concurrent use of carbonic anhydrase inhibitors or non-potassium sparing diuretics
- Recent or planned use of new weight management medications, stimulants, or investigational drugs within 2 months
- Cognitive impairment preventing participation
- Considered unsuitable for participation by the investigator for other reasons
AI-Screening
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Trial Site Locations
Total: 2 locations
1
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
2
Seattle Children's
Seattle, Washington, United States, 98101
Actively Recruiting
Research Team
C
Christian L Roth, MD
CONTACT
S
Stephanie Purdy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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