Qsymia for Childhood Obesity

Overseen By Megan Bensignor, MD

Age: < 65

Sex: Any

Trial Phase: Phase < 1

Sponsor: University of Minnesota

Must not be taking: Anti-obesity, Sulfonylureas, Stimulants, MAOIs

Disqualifiers: Pregnancy, Glaucoma, Psychiatric disorder, others

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment option for obesity in young people, a major factor in the rapid progression of type 2 diabetes (a condition where blood sugar levels are too high) in adolescents. The treatment being tested is a medication combination called Phentermine/Topiramate (also known as Qsymia), which has proven effective in adults for weight loss. Participants will receive either the medication or a placebo (a harmless pill with no active ingredients) to evaluate its effectiveness in reducing obesity and improving diabetes-related health issues. The trial seeks participants aged 12 to 20 who have obesity and type 2 diabetes. As an Early Phase 1 trial, this research aims to understand how the treatment works in young people, offering participants a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial requires that you have not started or changed the dose of weight-altering medications in the past 6 months and excludes those currently using certain anti-obesity medications, sulfonylureas, stimulant medications, and others. If you are on these medications, you may need to stop them before participating.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that the combination of phentermine and topiramate is generally well-tolerated by adults and aids in weight loss. In studies involving adults, this combination resulted in over 9% weight loss within a year, with maintenance of this weight loss for at least two years. Common side effects included tingling sensations, dizziness, changes in taste, trouble sleeping, constipation, and dry mouth. Improvements in blood pressure and blood sugar levels were also observed.

While the FDA has approved this combination for treating obesity in adults, it has not been fully tested for safety in children. However, adult results provide some indication of expected outcomes. It is important to note that children might experience different side effects and responses than adults. Consulting a healthcare professional about potential risks and benefits is essential. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about phentermine/topiramate for childhood obesity because it combines two medications that target weight loss through different mechanisms. Phentermine is a well-known appetite suppressant, while topiramate is traditionally used to treat seizures but has shown weight loss benefits. This combination could offer a more comprehensive approach to managing obesity in children compared to standard treatments like lifestyle changes and, occasionally, medications approved for adults. By potentially addressing both appetite control and metabolic processes, phentermine/topiramate might provide a new, effective option for reducing childhood obesity.

What evidence suggests that Phentermine/Topiramate might be an effective treatment for childhood obesity?

Research has shown that Phentermine/Topiramate can aid weight loss in people with obesity. In a large study, adults taking the highest dose lost over 9% of their weight in one year and experienced improvements in blood pressure and blood sugar levels. For teenagers, the treatment significantly reduced BMI and improved triglyceride and HDL cholesterol levels. Participants in this trial will be randomized to receive either Phentermine/Topiramate or a placebo during the placebo-controlled period. A 56-week study with young people aged 12 to 17 found that Qsymia, a brand of Phentermine/Topiramate, effectively managed their weight. The treatment is generally well-tolerated, though some may experience side effects like dizziness and dry mouth. ^{1 2 5 6 7}

Who Is on the Research Team?

Megan Bensignor, MD

Principal Investigator

University of Minnesota

Are You a Good Fit for This Trial?

This trial is for adolescents aged 12-20 with type 2 diabetes and obesity (BMI ≥95th percentile). Participants must have a diagnosis of T2D with HgbA1c ≥6.5%, no diabetes auto-antibodies, and not be pregnant or planning pregnancy. They should agree to use two forms of contraception if applicable, speak English or Spanish, and not have certain medical conditions like severe kidney impairment or history of seizures.

Inclusion Criteria

For participants of child-bearing potential: when sexually active, agreement to use two forms of highly effective contraception (oral contraceptive pill, IUD, implant, and/or condoms) during study participation

My BMI is in the top 5% for my age and sex.

Negative diabetes auto-antibodies

Exclusion Criteria

I have been treated with growth hormone in the past.

I have used weight loss medication in the last 6 months.

I have a history of glaucoma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Placebo-Controlled Treatment

Participants are randomized to receive either phentermine/topiramate or placebo for 6 months

6 months

Monthly visits (in-person)

Open Label Extension

Participants receive open label phentermine/topiramate for an additional 6 months

6 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• Phentermine/Topiramate

Trial Overview The study tests Phentermine/Topiramate (Qsymia), an FDA-approved adult obesity medication, against a placebo in adolescents. The drug has shown significant weight loss in adults over one year. This trial will assess its effectiveness and safety profile in the younger population with both obesity and type 2 diabetes.

How Is the Trial Designed?

Group I: Open Label Extension Experimental Treatment 1 Intervention

Participants in this phase of the study will receive open label phentermine/topiramate.

Group II: Placebo-Controlled Period Placebo Group 2 Interventions

Participants in this phase of the study will be randomized 1:1 to receive either phentermine/topiramate or placebo.

Phentermine/Topiramate is already approved in United States for the following indications:

Approved in United States as Qsymia for:

• Obesity in adults with a BMI of 27 kg/m^2 or greater and at least one weight-related comorbidity, or with a BMI of at least 30 kg/m^2

Who Is Running the Clinical Trial?

University of Minnesota

Lead Sponsor

Trials

1,459

Recruited

1,623,000+

Published Research Related to This Trial

In a 56-week study involving adolescents aged 12 to under 17 with obesity, phentermine/topiramate (PHEN/TPM) significantly reduced body mass index (BMI) compared to placebo, with reductions of -10.44 percentage points for the top dose and -8.11 percentage points for the mid dose (both P<0.001).

PHEN/TPM also positively affected triglyceride and HDL cholesterol levels, although the incidence of adverse events was higher in the treatment groups (51.8% for placebo, 37.0% for mid-dose, and 52.2% for top-dose), indicating a need for careful monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phentermine/Topiramate for the Treatment of Adolescent Obesity. Kelly, AS., Bensignor, MO., Hsia, DS., et al.[2023]

Phentermine/topiramate controlled-release (PHEN/TPM CR) led to significant weight loss of 8.1-10.9% in patients, compared to only 1.4-1.8% in placebo groups, and also reduced waist circumference and improved metabolic markers like blood pressure and lipid profiles.

The treatment was generally well-tolerated, with severe adverse events being similar to those in placebo groups, although common side effects included paresthesia, dry mouth, and insomnia. Long-term safety and efficacy still need further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A review of the metabolic effects of controlled-release Phentermine/Topiramate. Kiortsis, DN.[2019]

The American Academy of Pediatrics now recognizes pediatric obesity as a chronic disease and supports the use of various treatment options, including lifestyle changes, medications, and surgery, to address this growing issue.

Phentermine/topiramate, a combination medication approved for youth aged 12 and older, has shown promise for chronic weight management in pediatric patients, although clinical trials for obesity treatments in children remain limited.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Narrative Review: Phentermine and Topiramate for the Treatment of Pediatric Obesity. Kim, A., Nguyen, J., Babaei, M., et al.[2023]

Qsymia™, a combination of phentermine and topiramate, has been shown to produce greater weight loss than either drug alone, making it an effective treatment for obesity since its approval in 2012.

While Qsymia™ is effective, it carries potential risks such as teratogenicity, increased heart rate, psychiatric and cognitive side effects, and metabolic acidosis, which are consistent with the known side effects of its individual components.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical utility of phentermine/topiramate (Qsymia™) combination for the treatment of obesity. Shin, JH., Gadde, KM.[2021]

Combination pharmacotherapy, such as using phentermine and topiramate together, may provide better weight loss results for pediatric patients with obesity compared to single-agent treatments, which often have limited efficacy.

This case report of a 10-year-old girl illustrates the potential benefits of combining medications after lifestyle modifications were insufficient, highlighting the need for more effective treatment options in pediatric obesity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Potential Role of Combination Pharmacotherapy to Improve Outcomes of Pediatric Obesity: A Case Report and Discussion. Fox, CK., Kelly, AS.[2020]

In a study involving 42 adolescents aged 12 to 17 with obesity, the fixed-dose combination of phentermine and topiramate (VI-0521) led to significant weight loss, with 13.3% of the mid-dose group and 50.0% of the top-dose group achieving at least a 5% reduction in weight by day 56.

The combination was found to be safe and well-tolerated, with pharmacokinetic profiles similar to those observed in adults, suggesting that both mid- and top-dose levels are suitable for further long-term studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, double-blind, placebo-controlled, pharmacokinetic and pharmacodynamic study of a fixed-dose combination of phentermine/topiramate in adolescents with obesity. Hsia, DS., Gosselin, NH., Williams, J., et al.[2022]

Qsymia™, a once-daily oral capsule combining phentermine and extended-release topiramate, has been approved in the US for treating obesity, leveraging phentermine's appetite-suppressing effects and topiramate's ability to increase feelings of fullness.

The formulation is also being explored for potential benefits in treating sleep apnea and type 2 diabetes, indicating its broader therapeutic applications beyond weight loss.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phentermine and topiramate extended release (Qsymia™): first global approval. Cameron, F., Whiteside, G., McKeage, K.[2021]

Phentermine/topiramate (Qsymia®) is a combination medication approved in July 2022 for chronic weight management in pediatric patients aged 12 and older with obesity, specifically those with a BMI in the 95th percentile or greater.

This medication works by decreasing appetite and increasing feelings of fullness, and it is also being studied for other conditions like sleep apnea and type 2 diabetes in obese patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phentermine/Topiramate: Pediatric First Approval. Dhillon, S.[2022]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40600300/

Real-World Use of Phentermine and Topiramate for ... Conclusions: Phentermine and/or topiramate resulted in BMI reduction in most patients exposed for 3-12 months. Safety data support ...

2. evidence.nejm.org evidence.nejm.org/doi/full/10.1056/EVIDoa2200014

Phentermine/Topiramate for the Treatment of Adolescent ... PHEN/TPM at both the mid and top doses offered a statistically significant reduction in BMI and favorably impacted triglyceride and HDL-C levels in adolescents ...

3. liebertpub.com liebertpub.com/doi/full/10.1089/chi.2025.0007?doi=10.1089/chi.2025.0007

Real-World Use of Phentermine and Topiramate for ... Conclusions: Phentermine and/or topiramate resulted in BMI reduction in most patients exposed for 3–12 months. Safety data support ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10460571/

A Narrative Review: Phentermine and Topiramate for the ... The study results suggested that phentermine monotherapy was both safe and effective in yielding clinically meaningful weight loss in patients ...

5. hcp.qsymia.com hcp.qsymia.com/efficacy-and-safety/results-in-pediatric-patients/

Weight-Management Results with Qsymia® in Pediatric Patients Qsymia was studied in a 56-week, randomized, double blind trial that involved 223 pediatric patients 12 to less than 17 years of age with obesity.

6. cdc.gov cdc.gov/mmwr/volumes/74/wr/mm7420a1.htm

Prescriptions for Obesity Medications Among Adolescents ... This report describes the proportion of adolescents with obesity who were prescribed an obesity medication during 2018–2023.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06299891

Efficacy and Safety of Phentermine/Topiramate in Youth ... 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 ...