Hyperphagia in Prader-Willi SyndromePhase 3NCT06173531
Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
A Phase 3 study testing Carbetocin in people with Hyperphagia in Prader-Willi Syndrome, run by ACADIA Pharmaceuticals Inc. at 30 sites in 6 countries.
Trial summary
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS) Read more about this study
- Who is it for?
- Ages 5 to 30 years
- Conditions the study is for
- Hyperphagia in Prader-Willi Syndrome
- What is being tested?
- Carbetocin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 175 participants
- Where
- 30 sitesin 6 countries
- Study dates
- 27 Nov 2023to 19 Aug 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female participants aged 5 to 30 years
- Diagnosis of Prader-Willi syndrome with a documented disease-causing genetic mutation
- Evidence of hyperphagia, including increased appetite and decreased satiety with food-seeking behaviors
- Meeting minimum severity thresholds for hyperphagia based on validated clinical assessments at Screening and Baseline
- Residing with a caregiver who is able and willing to comply with study procedures and participate in study visits
Who cannot join
- Diagnosis of Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal disorder other than Prader-Willi syndrome
- Active upper respiratory infection at Screening or Baseline
- Clinically significant medical conditions that could interfere with study participation or interpretation of results (e.g., cardiovascular, renal, hepatic, gastrointestinal, or respiratory disease)
- History of significant neurological conditions (e.g., cerebrovascular disease, brain trauma, epilepsy, or frequent migraines), except for a history of febrile seizures
- Recent or planned nasal surgery that could affect intranasal drug administration
- Unwillingness to restrict use of intranasal medications that may interfere with study treatment
Show all 11Show fewer
- Recent clinically significant psychiatric instability, including irritability or agitation requiring changes in antipsychotic therapy
- Use of medications or treatments that may interfere with the evaluation of hyperphagia or study endpoints within a defined period prior to Screening Active psychotic disorder or history of significant psychotic symptoms
- History of suicide attempt or inpatient psychiatric hospitalization
- Recent changes in food-related behavioral or environmental interventions that may confound assessment of hyperphagia
- Additional inclusion and exclusion criteria apply. Participants will be evaluated at Screening to ensure all eligibility requirements are met.
Trial locations and contacts
30 sites in 6 countries.
30 sites in 6 countries
United States19 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Children's of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Maimonides Medical Center
Brooklyn, New York, 11219
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Cook Children's Health Care System
Fort Worth, Texas, 79104
University of Iowa
Iowa City, Iowa, 52242
Children's Mercy Hospital
Kansas City, Missouri, 64108
Vanderbilt Clinical Research Center
Nashville, Tennessee, 37232
University of California Irvine
Orange, California, 92697
Stanford University School of Medicine
Palo Alto, California, 94304
Phoenix Children's Hospital
Phoenix, Arizona, 85006
UPMC-Children's Hospital Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Utah
Salt Lake City, Utah, 84108
Christus Children's
San Antonio, Texas, 78207
Rady Children's Hospital San Diego
San Diego, California, 92123
Seattle Children's Hospital
Seattle, Washington, 98105
SSM Health/Saint Louis University
St Louis, Missouri, 63104
Spain3 sites
Parc Taulí Hospital Universitari
Barcelona, 08208
Hospital General Universitario Gregorio Marañón
Madrid, 28009
Hospital Regional Universitario de Málaga
Málaga, 29010
United Kingdom3 sites
University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
Birmingham, B9 5SS
Royal Hospital for Children Glasgow Clinical Research Facility
Glasgow, G51 4TF
Barts Health NHS Trust - The Royal London Hospital
London, E1 1BB
Canada2 sites
Alberta Diabetes Institute
Edmonton, Alberta, T6G 2E1
CHU Sainte Justine
Montreal, Quebec, H3T 1C5
Germany2 sites
KJF Klinik Josefinum gGmbH
Augsburg, 86154
Universitätsklinikum Essen
Essen, 45147
France1 site
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, 31059
About this study
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
Read the detailed description
This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Being tested
- Carbetocin, Placebo
- Keywords
- Randomized, Placebo-controlled
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarbetocinDrug
Also called ACP-101
Carbetocin nasal spray 3.2 mg administered TID
PlaceboDrug
Matching placebo nasal spray administered TID
| Study group | Role | What they receive |
|---|---|---|
| Carbetocin | Experimental | Carbetocin nasal spray 3.2 mg administered TID Receives: Drug: Carbetocin |
| Placebo | Placebo comparator | Matching placebo nasal spray administered TID Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline at Week 12 in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
The HQ-CT is a caregiver-reported, 9-item assessment of observable hyperphagia-related behaviors in participants with PWS. The questionnaire is administered to the caregiver by a trained rater using standardized prompts, and the same caregiver is expected to complete the assessment throughout the study whenever possible. Each item is scored from 0 to 4, resulting in a total score ranging from 0 to 36, with higher scores indicating greater severity of hyperphagia. A decrease from baseline reflects improvement, and a reduction of 7.7 points has been identified as a clinically meaningful within-participant improvement.
Measured: Baseline to Week 12
Secondary outcomes (4)
Change From Baseline at Week 12 in Clinical Global Impression of Severity (CGI-S) for PWS Score
The CGI-S for PWS is a clinician-rated assessment of the overall severity of PWS symptoms at the time of evaluation. The CGI-S is rated on a 7-point scale ranging from 1 (normal, not at all ill) to 7 (among the most severely ill participants), with higher scores indicating greater illness severity. The assessment is performed by the Investigator or a trained, qualified designee, and the same rater is expected to evaluate a given participant throughout the study whenever possible to maintain rating consistency.
Measured: Baseline to Week 12
Clinical Global Impression of Change (CGI-C) for PWS Score at Week 12
The CGI-C for PWS is a clinician-rated assessment of change in the overall severity of PWS symptoms relative to Baseline. The CGI-C is rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse), with lower scores indicating greater improvement from Baseline. The assessment is performed by the Investigator or a trained, qualified designee, and the same rater is expected to evaluate a given participant throughout the study whenever possible to maintain rating consistency.
Measured: Week 12
Percentage of Subjects With Treatment Response at Week 12 (Defined as Improvement From Baseline ≥8 Points) Based on the HQ-CT Score at Week 12
Treatment response was defined as an improvement from Baseline of at least 8 points in the HQ-CT total score at Week 12. The HQ-CT is a caregiver-reported, 9-item assessment of observable hyperphagia-related behaviors in participants with PWS. Total scores range from 0 to 36, with higher scores indicating greater severity of hyperphagia. Participants meeting the ≥8-point improvement threshold were classified as responders.
Measured: Week 12
Change From Baseline at Week 12 in CGI-S for Hyperphagia in PWS Score
The CGI-S for PWS is a clinician-rated assessment of the overall severity of PWS symptoms at the time of evaluation. The CGI-S is rated on a 7-point scale ranging from 1 (normal, not at all ill) to 7 (among the most severely ill participants), with higher scores indicating greater illness severity. The assessment is performed by the Investigator or a trained, qualified designee, and the same rater is expected to evaluate a given participant throughout the study whenever possible to maintain rating consistency.
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2023
- Study start27 Nov 2023
- Primary completion19 Aug 2025
- Study completion19 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hyperphagia in 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 175 participants (actual)
Sponsor and investigators
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06173531
- Sponsor's protocol ID
- ACP-101-302
- Other IDs
- 2023-506200-24-00, 2023
- Status last verified
- Sep 2026
- First submitted
- 8 Dec 2023
- Last updated
- 15 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Roof E, Strong TV, Stafford DEJ, Singh D, Roy SM, Doshi D, Zhang Y, Salvucci A, Youakim JM, McCandless SE; COMPASS PWS Investigators. Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome: Results From the Randomized, Placebo-Controlled, Phase 3 Compass PWS Study. Clin Ther. 2026 Jul 22:S0149-2918(26)00234-1. doi: 10.1016/j.clinthera.2026.06.019. Online ahead of print.
PubMed 42486751
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT06173531
- Acronym / sponsor ID
- ACP-101-302
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Keywords
- Randomized, Placebo-controlled
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 175 participants (actual)
- Lead sponsor
- ACADIA Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Nov 2023 (actual)
- Primary completion
- 19 Aug 2025 (actual)
- Study completion
- 19 Aug 2025 (actual)
- First posted
- 15 Dec 2023 (actual)
- Results
- Posted 15 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
Detailed description
This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Carbetocin | Experimental | Drug: CarbetocinCarbetocin nasal spray 3.2 mg administered TID |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo nasal spray administered TID |
Who may qualify
- Ages
- 5 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Male or female participants aged 5 to 30 years
- Diagnosis of Prader-Willi syndrome with a documented disease-causing genetic mutation
- Evidence of hyperphagia, including increased appetite and decreased satiety with food-seeking behaviors
- Meeting minimum severity thresholds for hyperphagia based on validated clinical assessments at Screening and Baseline
- Residing with a caregiver who is able and willing to comply with study procedures and participate in study visits
Key exclusion criteria (11 listed)
- Diagnosis of Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal disorder other than Prader-Willi syndrome
- Active upper respiratory infection at Screening or Baseline
- Clinically significant medical conditions that could interfere with study participation or interpretation of results (e.g., cardiovascular, renal, hepatic, gastrointestinal, or respiratory disease)
- History of significant neurological conditions (e.g., cerebrovascular disease, brain trauma, epilepsy, or frequent migraines), except for a history of febrile seizures
- Recent or planned nasal surgery that could affect intranasal drug administration
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 |
|---|---|---|
| CarbetocinAlso: ACP-101Carbetocin nasal spray 3.2 mg administered TID | Drug | Carbetocin |
| PlaceboMatching placebo nasal spray administered TID | Drug | Placebo |
Primary outcome
Change From Baseline at Week 12 in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score
Time frame: Baseline to Week 12
The HQ-CT is a caregiver-reported, 9-item assessment of observable hyperphagia-related behaviors in participants with PWS. The questionnaire is administered to the caregiver by a trained rater using standardized prompts, and the same caregiver is expected to complete the assessment throughout the study whenever possible. Each item is scored from 0 to 4, resulting in a total score ranging from 0 to 36, with higher scores indicating greater severity of hyperphagia. A decrease from baseline reflects improvement, and a reduction of 7.7 points has been identified as a clinically meaningful within-participant improvement.
4 secondary outcomes
Change From Baseline at Week 12 in Clinical Global Impression of Severity (CGI-S) for PWS Score
Time frame: Baseline to Week 12
Clinical Global Impression of Change (CGI-C) for PWS Score at Week 12
Time frame: Week 12
Percentage of Subjects With Treatment Response at Week 12 (Defined as Improvement From Baseline ≥8 Points) Based on the HQ-CT Score at Week 12
Time frame: Week 12
Change From Baseline at Week 12 in CGI-S for Hyperphagia in PWS Score
Time frame: Baseline to Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42486751Roof E, Strong TV, Stafford DEJ, Singh D, Roy SM, Doshi D, Zhang Y, Salvucci A, Youakim JM, McCandless SE; COMPASS PWS Investigators. Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome: Results From the Randomized, Placebo-Controlled, Phase 3 Compass PWS Study. Clin Ther. 2026 Jul 22:S0149-2918(26)00234-1. doi: 10.1016/j.clinthera.2026.06.019. Online ahead of print.
- Individual participant dataSharing planned: No
Related trials for Hyperphagia in Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness
Recruiting
Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation
Prader-Willi Syndrome
Active, not recruitingPhase 2
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome, Obesity
Recruiting
Effects of Transcutaneous Vagus Nerve Stimulation on Emotion Regulation and Executive Functioning in Prader-Willi Syndrome
Prader-Willi Syndrome
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
30 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Carbetocin in people with Hyperphagia in Prader-Willi Syndrome, run by ACADIA Pharmaceuticals Inc. at 30 sites in 6 countries.
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 5 inclusion and 11 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: finished, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Spain, United Kingdom, Canada, Germany and 1 more country. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline at Week 12 in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06173531, which also lists 1 linked publication.
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 15 Oct 2026.