ClinicalTrials.gg
TerminatedNCT06420297Updated Aug 17, 2026

OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

A Phase 3 interventional study of Carbetocin in Hyperphagia in Prader-Willi Syndrome, sponsored by ACADIA Pharmaceuticals Inc.. Terminated at 30 sites in 6 countries. Open to participants aged 5 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-08-17.

Sponsored by ACADIA Pharmaceuticals Inc. · Phase 3, Interventional, and Treatment

Why this study was terminated
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.
Phase
Phase 3
Study type
Interventional
Enrollment
168
Allocation
Not applicable
Ages
5 Years to 30 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Hyperphagia in Prader-Willi Syndrome

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Keywords

  • Opel-label extension
  • safety
  • tolerability
03

In context

Prader-Willi Syndrome

144 studies on the registry are indexed under Prader-Willi Syndrome; 23 are open to participants now.

This study's enrollment of 168 is above the median of 30 across 100 interventional studies indexed under Prader-Willi Syndrome.

Browse Prader-Willi Syndrome studies →

Lead sponsor

ACADIA Pharmaceuticals Inc. is the lead sponsor of 41 studies on the registry; 5 are open to participants now.

Of its 26 completed or terminated interventional studies of FDA-regulated products, 24 (92%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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.

Exclusion criteria

Exclusion Criteria:

  • 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 abnormality in vital signs at Baseline
  • Has an average interval of >450 ms on the Baseline 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 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.

Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
168 participants (actual)

Study arms

  • Experimental
    Drug: Carbetocin

    Carbetocin nasal spray 3.2 mg three times daily (TID)

    Drug: Carbetocin

Interventions

  • Carbetocin nasal spray 3.2 mg three times daily (TID)

    Also known as: ACP-101

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline to 36 months

07

Study locations

30 sites
  • Children's of Alabama
    Birmingham, Alabama 35233, United States
  • Phoenix Children's Hospital
    Phoenix, Arizona 85006, United States
  • University of California Irvine
    Orange, California 92697, United States
  • Stanford University School of Medicine
    Palo Alto, California 94304, United States
  • Rady Children's Hospital San Diego
    San Diego, California 92123, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • University of Iowa
    Iowa City, Iowa 52242, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Children's Mercy Kansas City
    Kansas City, Missouri 64108, United States
  • SSM Health/Saint Louis University
    St Louis, Missouri 63104, United States
  • Maimonides Medical Center
    Brooklyn, New York 11219, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • UPMC-Children's Hospital Pittsburgh
    Pittsburgh, Pennsylvania 15224, United States
  • Vanderbilt Clinical Research Center
    Nashville, Tennessee 37232, United States
  • Cook Children's Health Care System
    Fort Worth, Texas 79104, United States
  • Christus Children's
    San Antonio, Texas 78207, United States
  • University of Utah
    Salt Lake City, Utah 84108, United States
  • Seattle Children's Hospital
    Seattle, Washington 98105, United States
  • Alberta Diabetes Institute
    Edmonton, Alberta T6G 2E1, Canada
  • CHU Sainte-Justine
    Montreal, Quebec H3T 1C5, Canada
  • Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital des Enfants
    Toulouse, Cedex 9 Occitanie 31059, France
  • KJF Klinik Josefinum gGmbH
    Augsburg, 86154, Germany
  • Universitätsklinikum Essen
    Essen, 45147, Germany
  • Parc Taulí Hospital Universitari
    Barcelona, 08208, Spain
  • Hospital General Universitario Gregorio Marañón
    Madrid, 28007, Spain
  • Hospital Regional Universitario de Málaga
    Málaga, 29009, Spain
  • University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
    Birmingham, B9 5SS, United Kingdom
  • Royal Hospital for Children Glasgow Clinical
    Glasgow, G51 4TF, United Kingdom
  • Barts Health NHS Trust - The Royal London Hospital
    London, E1 1B6, United Kingdom
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Mar 2024 to primary completion in Dec 2025, 21 months.

How these figures are worked out, and which studies are left out of them.

Registered
May 7, 2024
First posted
May 20, 2024
StartActual
Mar 11, 2024
Primary completionActual
Dec 19, 2025
CompletionActual
Dec 19, 2025
Last update
Aug 17, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06420297
Responsible party
Sponsor
First posted
May 20, 2024
Start date
Mar 11, 2024
Primary completion
Dec 19, 2025
Completion
Dec 19, 2025
Last update
Aug 17, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Carbetocin

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