Hyperphagia in Prader-Willi SyndromePhase 3NCT06420297
OLE 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
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with 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?
- No, everyone receives an active treatment
- How many people
- 168 participants
- Where
- 30 sitesin 6 countries
- Study dates
- 11 Mar 2024to 19 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302
- Met all entry criteria for the antecedent study
- May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator.
- Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits.
Who cannot join
- History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
- Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
- History of suicide attempt or inpatient psychiatric hospitalization
- Has a clinically significant abnormality in vital signs at Baseline
- Has an average QTcF interval of >450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
- Has developed a clinically significant ECG finding during the antecedent study
- Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
- Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation 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 Kansas City
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, 28007
Hospital Regional Universitario de Málaga
Málaga, 29009
United Kingdom3 sites
University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
Birmingham, B9 5SS
Royal Hospital for Children Glasgow Clinical
Glasgow, G51 4TF
Barts Health NHS Trust - The Royal London Hospital
London, E1 1B6
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 - Hopital des Enfants
Toulouse, Cedex 9 Occitanie, 31059
About this study
Official title: A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS
Read the detailed description
This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Being tested
- Carbetocin
- Keywords
- Opel-label extension, safety, tolerability
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 three times daily (TID)
| Study group | Role | What they receive |
|---|---|---|
| Drug: Carbetocin | Experimental | Carbetocin nasal spray 3.2 mg three times daily (TID) Receives: Drug: Carbetocin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions
Treatment-emergent adverse events (TEAEs) and device incidents or device malfunctions, TEAEs leading to discontinuation, TEAEs related to study drug, TEAEs by maximum severity, fatal TEAEs, treatment-emergent SAEs, and treatment-emergent SAEs related to study drug will all be summarized for all subjects as well as by previous treatment group.
Measured: Baseline to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2024
- Study start11 Mar 2024
- Primary completion19 Dec 2025
- Study completion19 Dec 2025
Why it stopped: Primary efficacy endpoint was not met in the parent study (ACP-101-302). Based on review of available data, continued administration of investigational product in ACP-101-303 was not considered likely to provide significant clinical benefit.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 168 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
- NCT06420297
- Sponsor's protocol ID
- ACP-101-303
- Other IDs
- 2023-506201-19-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 7 May 2024
- Last updated
- 17 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 17 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT06420297
- Acronym / sponsor ID
- ACP-101-303
- Conditions
- Hyperphagia in Prader-Willi Syndrome
- Keywords
- Opel-label extension, safety, tolerability
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Primary efficacy endpoint was not met in the parent study (ACP-101-302). Based on review of available data, continued administration of investigational product in ACP-101-303 was not considered likely to provide significant clinical benefit.
- Enrolment
- 168 participants (actual)
- Lead sponsor
- ACADIA Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Mar 2024 (actual)
- Primary completion
- 19 Dec 2025 (actual)
- Study completion
- 19 Dec 2025 (actual)
- First posted
- 20 May 2024 (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.
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS
Detailed description
This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Drug: Carbetocin | Experimental | Drug: CarbetocinCarbetocin nasal spray 3.2 mg three times daily (TID) |
Who may qualify
- Ages
- 5 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302
- Met all entry criteria for the antecedent study
- May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator.
- Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits.
Key exclusion criteria (8 listed)
- History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
- Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
- History of suicide attempt or inpatient psychiatric hospitalization
- Has a clinically significant abnormality in vital signs at Baseline
- Has an average QTcF interval of >450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
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 three times daily (TID) | Drug | Drug: Carbetocin |
Primary outcome
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions
Time frame: Baseline to 36 months
Treatment-emergent adverse events (TEAEs) and device incidents or device malfunctions, TEAEs leading to discontinuation, TEAEs related to study drug, TEAEs by maximum severity, fatal TEAEs, treatment-emergent SAEs, and treatment-emergent SAEs related to study drug will all be summarized for all subjects as well as by previous treatment group.
Results and safety
The study was stopped early (reason given: Primary efficacy endpoint was not met in the parent study (ACP-101-302). Based on review of available data, continued administration of investigational product in ACP-101-303 was not considered likely to provide significant clinical benefit.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2026.
- 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. 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 4 inclusion and 8 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: stopped early, as of the sponsor's last update on 17 Aug 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. 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?
The study was stopped early (reason given: Primary efficacy endpoint was not met in the parent study (ACP-101-302). Based on review of available data, continued administration of investigational product in ACP-101-303 was not considered likely to provide significant clinical benefit.). Results are not available in the source record.
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?
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions (time frame: Baseline to 36 months). 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 17 Aug 2026; AzTrials last refreshed its copy on 8 Nov 2026.