Tirzepatide for Weight Loss in Prader-Willi Syndrome and Obesity

Recruiting at 3 trial locations

Overseen By Parisa Salehi, MD

Age: 18 - 65

Sex: Any

Trial Phase: Phase 4

Sponsor: Grace Kim

Must not be taking: Weight loss drugs, Insulin, GLP1A, DPP4

Disqualifiers: Pregnancy, Pancreatitis, Diabetes, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 48 weeks

36 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a weight loss medication called Tirzepatide in young adults with various types of obesity. It aims to evaluate the medication's effectiveness for individuals with Prader-Willi Syndrome, hypothalamic obesity (a condition where the brain struggles to control hunger and energy use), and general obesity without a specific medical cause. Participants will take the medication for a year to observe changes in weight and health. Those who have had obesity for at least two years and have not recently used weight loss medication might be suitable candidates. As a Phase 4 trial, this research seeks to understand how this FDA-approved and effective treatment benefits a broader range of patients.

Will I have to stop taking my current medications?

The trial requires that you stop taking any weight loss medications, insulin, sulfonylureas, or medications affecting insulin secretion or GLP1 clearance at least 3 months before starting the study. You also need to avoid any medications that might affect the study's results.

What is the safety track record for Tirzepatide?

Research has shown that tirzepatide is generally safe and effective for individuals with various types of obesity. Studies have found that it aids significant weight loss, including in those with general obesity, hypothalamic obesity, and Prader-Willi Syndrome.

For instance, one study reported significant and lasting weight loss in participants taking tirzepatide, with doses ranging from 5 mg to 15 mg per week. In another case, a person with hypothalamic obesity lost 9 kg over four months by gradually increasing their dose. Additionally, a review of several studies found that tirzepatide significantly reduces body weight in individuals with both diabetes and obesity.

While these results are encouraging, the effects of tirzepatide on individuals with Prader-Willi Syndrome have not been thoroughly studied. However, tirzepatide has shown positive results for weight management in other conditions, indicating it is generally safe to use. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Tirzepatide is unique because it combines the actions of two hormones that regulate blood sugar and appetite, GLP-1 and GIP, offering a dual approach to weight management. Unlike most treatments for obesity, which often focus solely on appetite suppression or calorie burning, tirzepatide targets both appetite and energy balance more comprehensively. Researchers are particularly excited about tirzepatide's potential for addressing obesity in conditions like Prader-Willi Syndrome and hypothalamic obesity, where traditional treatments often fall short. This dual-acting mechanism could lead to more effective weight loss and improved metabolic outcomes, offering new hope for individuals who haven't had success with current options.

What evidence suggests that Tirzepatide might be an effective treatment for Prader-Willi Syndrome and hypothalamic obesity?

This trial will evaluate Tirzepatide for weight loss in various types of obesity, including Prader-Willi Syndrome, hypothalamic obesity, and general non-syndromic obesity. Research has shown that Tirzepatide can aid weight loss in different contexts. For general obesity, studies found that certain doses led to a weight loss of about 19.5% to 20.9% of body weight. In one case of hypothalamic obesity, a patient lost 9 kg while using Tirzepatide. Although not extensively studied in Prader-Willi Syndrome, similar treatments have resulted in significant weight loss, such as one patient losing 125 lbs. Tirzepatide works by affecting hormones that control hunger and energy use, supporting its potential in various types of obesity. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for young adults with Prader-Willi Syndrome, hypothalamic obesity, or general obesity without a genetic syndrome. Participants will be given Tirzepatide for weight loss over a year to see its effects on weight and health.

Inclusion Criteria

Consistent caregiver if not independent

Stable diet and exercise regimen for at least 6 months prior to enrollment

Able to use contraceptive methods if able to conceive offspring to prevent unintentional pregnancy during the study

Exclusion Criteria

I am currently using or have recently used weight loss medications.

Significant weight change (>3% weight gain or loss) in the last 2 months prior to enrollment

Current pregnancy, desire to become pregnant within study period, or current lactation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tirzepatide for once-weekly dosing over 48 weeks, with regular monitoring and assessments

48 weeks

6 visits (in-person), 4 visits (virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tirzepatide

Trial Overview

The study tests the effectiveness of Tirzepatide, a weight loss medication, in different groups: those with Prader-Willi Syndrome/hypothalamic obesity versus those with non-syndromic obesity.

How Is the Trial Designed?

Group I: General Non-Syndromic Obesity Active Control 1 Intervention

Individuals 18-26 years with a BMI in the obesity range (BMI ≥95th percentile for age and sex or ≥30 kg/m2) and general obesity unrelated to a genetic syndrome or underlying medical condition.

Group II: Hypothalamic Obesity Active Control 1 Intervention

Individuals 18-26 years with a BMI in the obesity range (BMI ≥95th percentile for age and sex or ≥30 kg/m2) and hypothalamic obesity as defined by damage to the medial hypothalamic region resulting in dysregulation of satiety and energy balance as diagnosed by a physician.

Group III: Prader-Willi Syndrome Active Control 1 Intervention

Individuals 18-26 years with a BMI in the obesity range (BMI ≥95th percentile for age and sex or ≥30 kg/m2) with genetically confirmed Prader-Willi Syndrome between 18-26 years old.

Who Is Running the Clinical Trial?

Grace Kim

Lead Sponsor

Trials

1

Recruited

40+

Vanderbilt University Medical Center

Collaborator

Trials

922

Recruited

939,000+

Seattle Children's Hospital

Collaborator

Trials

319

Recruited

5,232,000+

Children's Hospitals and Clinics of Minnesota

Collaborator

Trials

67

Recruited

5,022,000+

Published Research Related to This Trial

A review of 14 studies and a case series of 10 adolescents with Prader-Willi syndrome (PWS) indicated that anti-obesity medications (AOMs) can help manage hyperphagia, with 60% of participants showing decreased or stable BMI z-scores after treatment.

No significant side effects were reported in the case series, suggesting that AOMs may be safe for use in youth with PWS, but further research is needed to establish comprehensive treatment guidelines.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-Obesity Medication Use in Children and Adolescents with Prader-Willi Syndrome: Case Review and Literature Search. Goldman, VE., Naguib, MN., Vidmar, AP.[2023]

In a 72-week phase 3 trial involving 938 adults with obesity and type 2 diabetes, tirzepatide (10 mg and 15 mg) led to significant weight loss, with reductions of -12.8% and -14.7% respectively, compared to only -3.2% with placebo.

The safety profile of tirzepatide was generally favorable, with most adverse events being mild to moderate gastrointestinal issues, and serious adverse events were rare, indicating it is a safe option for weight management in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tirzepatide once weekly for the treatment of obesity in people with type 2 diabetes (SURMOUNT-2): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial. Garvey, WT., Frias, JP., Jastreboff, AM., et al.[2023]

Tirzepatide, a new GLP-1 receptor agonist, was found to be more effective for weight loss compared to other GLP-1RAs like semaglutide and liraglutide, with significant weight loss observed in over 12,300 patients in the analysis.

While all GLP-1RAs were associated with gastrointestinal side effects, tirzepatide showed better efficacy outcomes for weight loss while maintaining a similar safety profile to other treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative effectiveness of glucagon-like peptide-1 receptor agonists for the management of obesity in adults without diabetes: A network meta-analysis of randomized clinical trials. Alkhezi, OS., Alahmed, AA., Alfayez, OM., et al.[2023]

Exenatide, a GLP-1 agonist, was effectively used to manage diabetes in a 19-year-old female with Prader-Willi syndrome, highlighting its potential in this specific population.

The drug demonstrated beneficial effects in reducing weight and suppressing appetite, suggesting it could be a promising treatment strategy for obesity and diabetes associated with Prader-Willi syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treating diabetes mellitus in Prader-Willi syndrome with Exenatide. Seetho, IW., Jones, G., Thomson, GA., et al.[2018]

In a systematic review of randomized controlled trials involving 5800 patients, tirzepatide was found to be highly effective for weight loss, with 78.22% achieving at least 5% weight loss and 32.28% achieving at least 15% weight loss.

Tirzepatide demonstrated superior weight loss compared to placebo and semaglutide, with a significant mean weight loss of -12.47 kg at the 5 mg dose, while showing a manageable safety profile with only a slight increase in gastrointestinal adverse events compared to placebo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of tirzepatide for treatment of overweight or obesity. A systematic review and meta-analysis. Tan, B., Pan, XH., Chew, HSJ., et al.[2023]

Tirzepatide, administered once weekly, was found to be effective in reducing obesity in individuals with type 2 diabetes, as demonstrated in a large phase 3 trial involving multiple centers.

The study was double-blind and placebo-controlled, ensuring robust results that support tirzepatide as a promising treatment option for weight management in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In adults with BMI ≥27 kg/m2 and type 2 diabetes, adding tirzepatide to a lifestyle intervention increased weight loss at 72 wk. Davidson, MB.[2023]

Tirzepatide, a novel GLP-1 receptor agonist, has been shown to significantly reduce A1C levels and body weight in patients with type 2 diabetes compared to placebo and other active treatments.

The safety profile of tirzepatide is similar to other GLP-1 receptor agonists, with the most common side effects being mild to moderate gastrointestinal issues, and it does not increase the risk of hypoglycemia, making it a promising treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Tirzepatide in Adults With Type 2 Diabetes: A Perspective for Primary Care Providers. Kushner, P., Anderson, JE., Simon, J., et al.[2023]

Citations

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