Tirzepatide in PWS, HO and GNSO

NCT06901245 | Recruiting Phase 4 FDA Drug

• 2 other identifiers: STUDY00004995I8F-NS-I001
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 3

Brief Summary

This research study is comparing the effectiveness of a weight loss medication called Tirzepatide in young adults with Prader-Willi Syndrome and/or hypothalamic obesity, as compared to young adults with obesity that is unrelated to a genetic syndrome or underlying medical cause. These groups will be given medication for 1 year to see how weight and other health factors are effected by the medication.

Conditions

Prader-Willi Syndrome; Hypothalamic Obesity; Obesity/Therapy

Keywords

Tirzepatide; GLP1 Agonist; Obesity; Prader-Willi Syndrome; Hypothalamic Obesity; General Obesity

Outcome Measures

Primary Outcomes (1)

• Change in weight

  Weight will be measured in kilograms through anthropometric measurements. Change in percent weight from baseline to week 48 of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity will then be assessed.

  48 weeks

Secondary Outcomes (9)

• Change in BMI

  48 weeks

• Change in fat mass

  48 weeks

• Change in appetite - CoEQ (Control of Eating Questionnaire)

  48 weeks

• Change in appetite - Physician Rated Hyperphagia Severity Scale

  48 weeks

• Change in metabolic markers - fasting lipids

  48 weeks

Other Outcomes (5)

• Change in safety markers -CBC

  48 weeks

• Change in safety markers - CMP

  48 weeks

• Change in safety markers - coagulation factor assay PT [prothrombin time]

  48 weeks

Study Arms (3)

Prader-Willi Syndrome

ACTIVE COMPARATOR

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.

Drug: Tirzepatide

Hypothalamic Obesity

ACTIVE COMPARATOR

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.

Drug: Tirzepatide

General Non-Syndromic Obesity

ACTIVE COMPARATOR

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.

Drug: Tirzepatide

Interventions

Tirzepatide DRUG

Subjects will take Tirzepatide for 48 weeks

General Non-Syndromic Obesity; Hypothalamic Obesity; Prader-Willi Syndrome

Eligibility Criteria

• Age: 18 Years - 26 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Individuals 18-26 years with a BMI in the obesity range (BMI ≥95th percentile for age and sex or ≥30 kg/m2) with either 1) genetically confirmed diagnosis of PWS, 2) hypothalamic obesity as defined by damage to the medial hypothalamic region resulting in dysregulation of satiety and energy balance as diagnosed by a physician, 3) general obesity unrelated to a genetic syndrome or underlying medical condition
• In a stable care setting at least 6 months prior to enrollment
• Able and willing to participate in study visits including tolerating blood draws, urine samples and tolerate DXA scan.
• Ability to take weekly subcutaneous tirzepatide
• Consistent caregiver if they are 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 in order to prevent unintentional pregnancy during the study

You may not qualify if:

• Current or recent (within 3 months of start of study drug initiation) use of weight loss medications
• Current use of insulin or sulfonylurea or other medication affecting insulin secretion or GLP1 clearance
• Current or prior use of any GLP1A or DPP4 inhibitor during the 6 months before screening
• Any medications that may affect the study endpoints
• Significant weight change (\>3% weight gain or loss) in the last 2 months prior to enrollment
• Change in dose of chronic endocrine medications (testosterone, estrogen, levothyroxine, or growth hormone medications) \>10%/kg/day for at least 3 months prior to study
• Current pregnancy or desire to become pregnant within study period, current lactation
• History of recurrent pancreatitis, CKD, gastroparesis
• Chronic/acute heart, kidney, or liver disease
• Personal or family history of medullary thyroid carcinoma or MEN syndrome type 2
• Uncontrolled diabetes (A1C \>8.5%)
• DVT
• Cancer within the previous 5 years
• Current participation in an interventional clinical study
• Previous or planned surgical treatment for obesity

Sponsors & Collaborators

• Grace Kim: lead
• Vanderbilt University Medical Center: collaborator
• Children's Hospitals and Clinics of Minnesota: collaborator
• Seattle Children's Hospital: collaborator

Study Sites (3)

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37203, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Prader-Willi Syndrome; Sexual Infantilism; Obesity

Interventions

Tirzepatide

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Gonadal Disorders; Endocrine System Diseases; Hypogonadism; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Glucagon-Like Peptide-1 Receptor; Glucagon-Like Peptide Receptors; Receptors, G-Protein-Coupled; Receptors, Cell Surface; Membrane Proteins; Proteins; Amino Acids, Peptides, and Proteins; Receptors, Gastrointestinal Hormone; Receptors, Peptide

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 14, 2025
• First Posted: March 28, 2025
• Study Start: May 1, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: September 16, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)