Efficacy and Safety of Phentermine/Topiramate in Youth With Hypothalamic Obesity
Recruiting · 2 sites across 2 states
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What is this study?
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.
It is , overseen by an independent and licensed medical staff.
Read the full clinical description
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.
Who this study is looking for
In plain language, from the study's own rules. The study team confirms the full details with you — this isn't a final yes or no.
✅ You may be able to join if…
- •You are male or female and are between 6 and 28 years old (inclusive).
- •You had rapid weight gain that started around your brain tumor onset or treatment, as judged by an endocrinologist.
- •You have obesity based on BMI rules for your age/sex (CDC 2000 for under 18; BMI > 30 for 18+).
- •Your MRI shows hypothalamic injury (checked with central review), and you are more than 6 months past definitive tumor treatment (surgery/chemo/radiation).
- •You are on stable pituitary replacement and/or appetite-related medications for at least 2 months (with specific limits on how much some hormones can be adjusted).
🚫 You may not be able to join if…
- •You have a condition or allergy that makes phentermine, topiramate, or Qsymia unsafe, including history of glaucoma or known hyperthyroidism.
- •You currently have kidney stones (nephrolithiasis).
- •You currently have anorexia nervosa or bulimia nervosa.
- •You have certain blood chemistry or bone problems, like metabolic acidosis (low bicarbonate) or clinically significant bone disease that needs medication beyond calcium/vitamin D.
- •You have important heart or rhythm problems, or blood pressure that is too high, or a long QT problem (QTc > 460 ms), or heart failure/cardiomyopathy, or coronary artery disease.
Are you a good fit?
Simplified highlights. The study team always confirms the full details with you.
- ✓Adults roughly 6–28
- ✓Have Obesity / overweight
- !Some conditions may not be a fit: Heart / cardiovascular disease
- !Not for people who are pregnant or breastfeeding
What to expect, step by step
- 1
Usually a few weeks
The study team checks whether the study is a good fit for you, with a visit and sometimes lab tests. You can ask any questions before deciding.
- 2
Treatment
If you join and choose to continue, you receive the study treatment and are watched closely by medical staff.
- 3
Follow-up
After treatment, the team checks on your health and confirms the visit schedule with you. You can leave the study at any point.
Has this treatment been tested before?
Yes. This treatment has already been through earlier human studies for safety before reaching this stage.
What you need to know before you apply
What is this study testing?+
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.
Is it safe? Who makes sure of that?+
This is a mid-stage study (Phase 2), looking at safety and whether the treatment works. Every study is reviewed and monitored by an independent ethics board (called an IRB) whose job is to protect participants, and care is overseen by licensed medical staff. You'll be told the known risks before you agree to anything, and you can stop at any time.
Will I get a placebo instead of the real treatment?+
Some studies compare a treatment against a placebo (an inactive version), and some don't. If this one does, the study team will explain your chances of receiving the active treatment before you decide. Nothing is hidden from you.
Do I have to stop taking my current medications?+
It depends on the study. Some let you stay on your current medications and some ask you to adjust them. Never stop a medication on your own — the study team will review everything with you first.
Does it cost anything? Will I be paid?+
The study treatment and study-related visits are provided at no cost to you. Some studies also pay for your time; the coordinator can tell you if this one does. You should never be asked to pay to take part.
Do I need insurance? Will anyone ask about my immigration status?+
No. You do not need health insurance to take part in a research study, and you will not be asked about your immigration status to join. Taking part is about whether you're a medical fit for the study.
What if English isn't my first language?+
You have the right to understand everything before you agree. Study sites can often provide materials or an interpreter in your language — you can ask the coordinator for one.
Is my information private?+
Yes. Your health information is only shared with the study sites you choose to be contacted by, and only to help match and enroll you. It is never sold, and you can ask us to delete it at any time.
Some requirements (like specific lab values or timing) are confirmed directly by the study team, not by us.
Source: ClinicalTrials.gov · NCT06299891 · Locations: Pennsylvania · Washington