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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06173531




Registration number
NCT06173531
Ethics application status
Date submitted
8/12/2023
Date registered
15/12/2023

Titles & IDs
Public title
Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Scientific 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
Secondary ID [1] 0 0
2023-506200-24
Secondary ID [2] 0 0
ACP-101-302
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hyperphagia in Prader-Willi Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Mental Health 0 0 0 0
Eating disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Carbetocin
Treatment: Drugs - Placebo

Experimental: Carbetocin - Carbetocin nasal spray 3.2 mg three times daily (TID)

Placebo comparator: Placebo - Placebo


Treatment: Drugs: Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)

Treatment: Drugs: Placebo
Placebo given TID, identical in appearance respective to carbetocin treatment
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
Timepoint [1] 0 0
Baseline to Week 12
Secondary outcome [1] 0 0
Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS
Timepoint [1] 0 0
Baseline to Week 12
Secondary outcome [2] 0 0
Clinical Global Impression-Change (CGI-C) for PWS score at Week 12
Timepoint [2] 0 0
Score at Week 12

Eligibility
Key inclusion criteria
* Male or female and 5 through 30 years of age
* Prader-Willi syndrome with a documented disease-causing mutation
* Increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)
* HQ-CT total score of =13 at Screening and Baseline
* CGI-S score for hyperphagia in PWS of =4 at Screening and Baseline
* 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
Minimum age
5 Years
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS
* An active upper respiratory infection at the Screening visit or the Baseline visit
* Any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma
* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary
* Nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study.
* Unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study
* Clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit
* Used prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary.
* Started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening.
* Used oxytocin, desmopressin (DDAVP), or tesofensine within 6 months prior to the Baseline visit
* Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
* History of suicide attempt or inpatient psychiatric hospitalization
* New food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit

Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/11/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
170
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Tennessee
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
Canada
State/province [15] 0 0
Alberta
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Birmingham
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Glasgow
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ACADIA Pharmaceuticals Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Andrew McGinn
Address 0 0
Country 0 0
Phone 0 0
610-299-6639
Fax 0 0
Email 0 0
andrew.mcginn@acadia-pharm.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06173531