Prader-Willi SyndromePhase 4NCT06901245
Tirzepatide in PWS, HO and GNSO
A Phase 4 study testing Tirzepatide in people with Prader-Willi Syndrome and Hypothalamic Obesity, run by Grace Kim at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 26 years
- Conditions the study is for
- Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
- What is being tested?
- Tirzepatide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 May 2025to Dec 2026
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 26 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 20Show fewer
- 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
- Individuals with current substance abuse equivalnt to moderate or severe based on DSM 5 criteria (Hasin DS, 2013)
- Any suicidal ideation in the past year
- Unable to perform any of the procedures for the study
- Have a body weight, height, and/or width that that prohibits the ability to obtain accurate measurements according to the DXA manufacturer's specification
- Any condition that would prevent successful participation in the study.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37203
Martha Upchurch, 6158754274, martha.upchurch@vumc.org
Ashley Shoemaker, MD
- Recruiting
Children's Minnesota
Saint Paul, Minnesota, 55102
Diabetes/Endocrinology Research Team, 651-220-5730, endocrineresearch@childrensmn.org
Jennifer Abuzzahab, MD
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Endocrine Research Team, 206-987-2540, EndocrineResearch@seattlechildrens.org
Grace Kim, MD
About this study
Official title: The Effects of Tirzepatide in Young Adults With Prader-Willi Syndrome, Hypothalamic Obesity and General Non-Syndromic Obesity
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.
Read the detailed description
This study is for the following individuals:
- Age 18-26.
- Have Prader-Willi Syndrome, Hypothalamic Obesity, or Obesity unrelated to a genetic syndrome or medical condition.
- Have a BMI in the obesity range
If you/your child decide to take part in the research study, you/your child would participate in the study for approximately 56-60 weeks. During this time the following can be expected:
- Receive tirzepatide for once-weekly dosing.
- Complete 6 in-person SCH study visits
- Complete 4 telehealth visits.
- Complete the following research procedures:
- Medical Record Review
- Vital Signs
- Anthropometry (e.g., height, weight, waist measurements)
- Physical Exams
- Laboratory Tests (e.g., fasting blood draws, urine test)
- Dual-Energy Absorptiometry (DXA) scans
- Questionnaires
- Conditions
- Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
- Being tested
- Tirzepatide
- Keywords
- Tirzepatide, GLP1 Agonist, Obesity, Prader-Willi Syndrome, Hypothalamic Obesity, General Obesity
- Medical subject headings
- Prader-Willi Syndrome, Sexual Infantilism, Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TirzepatideDrug
Subjects will take Tirzepatide for 48 weeks
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: Tirzepatide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 48 weeks
Secondary outcomes (9)
Change in BMI
Change in BMI from baseline to 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity through anthropometric measurements. BMI will be calculated using weight (kg) and height (m) which will be combined to report BMI in kg/m^2. The percent change in this BMI value from baseline to 48 weeks will be calculated.
Measured: 48 weeks
Change in fat mass
Change in fat mass (measured in kg by DXA scan) from baseline to 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity.
Measured: 48 weeks
Change in appetite - CoEQ (Control of Eating Questionnaire)
Change in appetite during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity using an appetite questionnaire (CoEQ - Control of Eating Questionnaire). CoEQ is a 21-item scale to assess the severity and type of food cravings an individual experiences using a visual analog scale. Higher scores indicate higher severity of hyperphagia.
Measured: 48 weeks
Change in appetite - Physician Rated Hyperphagia Severity Scale
Change in appetite during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity assed with a physician rated hyperphagia severity scale. This is a 5 point scale (0 to 5) to rate clinical imppression of hyperphagia severity. Higher scores mean more severity.
Measured: 48 weeks
Change in metabolic markers - fasting lipids
Change in metabolic markers during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity including: fasting lipids via lipid profile assay via blood sample
Measured: 48 weeks
Change in metabolic markers - fasting plasma glucose
Change in metabolic markers during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity including: fasting plasma glucose, via chemistry panel via blood sample
Measured: 48 weeks
Change in metabolic markers- fasting insulin
Change in metabolic markers during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity including: fasting insulin via insulin assay via blood sample
Measured: 48 weeks
Change in metabolic markers - hemoglobin A1C
Change in metabolic markers during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity including: hemoglobin A1C via hemoglobin A1C assay via blood sample
Measured: 48 weeks
Change in metabolic markers- Homeostatic Model Assessment of Insulin Resistance (HOMA-IR)
Change in metabolic markers during 48 weeks of treatment with tirzepatide in young adults with PWS, HO, GNSO and obesity including: Homeostatic Model Assessment of Insulin Resistance (HOMA-IR). This is product measuring insulin resistance calculated using fasting insulin and fasting glucose values. Higher numbers indicate more insulins resistance.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2025
- Study start1 May 2025
- Primary completionJun 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Grace Kim
- Sponsor type
- University, hospital or other
- Collaborators
- Vanderbilt University Medical Center, Children's Hospitals and Clinics of Minnesota, Seattle Children's Hospital
- Responsible party
- Sponsor-investigator, Grace Kim, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06901245
- Sponsor's protocol ID
- STUDY00004995
- Other IDs
- I8F-NS-I001
- Status last verified
- Jan 2025
- First submitted
- 14 Mar 2025
- Last updated
- 16 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Tirzepatide in Young Adults With Prader-Willi Syndrome, Hypothalamic Obesity and General Non-Syndromic Obesity
- ClinicalTrials.gov ID
- NCT06901245
- Acronym / sponsor ID
- STUDY00004995
- Conditions
- Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
- Keywords
- Tirzepatide, GLP1 Agonist, Obesity, Prader-Willi Syndrome, Hypothalamic Obesity, General Obesity
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Grace KimUniversity, hospital or other
- Collaborators
- Vanderbilt University Medical Center, Children's Hospitals and Clinics of Minnesota, Seattle Children's Hospital
- Study start
- 1 May 2025 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 28 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study is for the following individuals:
- Age 18-26.
- Have Prader-Willi Syndrome, Hypothalamic Obesity, or Obesity unrelated to a genetic syndrome or medical condition.
- Have a BMI in the obesity range
If you/your child decide to take part in the research study, you/your child would participate in the study for approximately 56-60 weeks. During this time the following can be expected:
- Receive tirzepatide for once-weekly dosing.
- Complete 6 in-person SCH study visits
- Complete 4 telehealth visits.
- Complete the following research procedures:
- Medical Record Review
- Vital Signs
- Anthropometry (e.g., height, weight, waist measurements)
- Physical Exams
- Laboratory Tests (e.g., fasting blood draws, urine test)
- Dual-Energy Absorptiometry (DXA) scans
- Questionnaires
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Prader-Willi Syndrome | Active comparator | Drug: TirzepatideIndividuals 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. |
| Hypothalamic Obesity | Active comparator | Drug: TirzepatideIndividuals 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. |
| General Non-Syndromic Obesity | Active comparator | Drug: TirzepatideIndividuals 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. |
Who may qualify
- Ages
- 18 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (20 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TirzepatideSubjects will take Tirzepatide for 48 weeks | Drug | General Non-Syndromic Obesity, Hypothalamic Obesity, Prader-Willi Syndrome |
Primary outcome
Change in weight
Time frame: 48 weeks
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.
9 secondary outcomes
Change in BMI
Time frame: 48 weeks
Change in fat mass
Time frame: 48 weeks
Change in appetite - CoEQ (Control of Eating Questionnaire)
Time frame: 48 weeks
Change in appetite - Physician Rated Hyperphagia Severity Scale
Time frame: 48 weeks
Change in metabolic markers - fasting lipids
Time frame: 48 weeks
Change in metabolic markers - fasting plasma glucose
Time frame: 48 weeks
Change in metabolic markers- fasting insulin
Time frame: 48 weeks
Change in metabolic markers - hemoglobin A1C
Time frame: 48 weeks
Change in metabolic markers- Homeostatic Model Assessment of Insulin Resistance (HOMA-IR)
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tirzepatide in people with Prader-Willi Syndrome and Hypothalamic Obesity, run by Grace Kim at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in weight (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.