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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03649477




Registration number
NCT03649477
Ethics application status
Date submitted
24/08/2018
Date registered
28/08/2018

Titles & IDs
Public title
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
Scientific title
Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin (LV-101) in Prader-Willi Syndrome (PWS) With Long Term Follow-Up (CARE-PWS)
Secondary ID [1] 0 0
LV-101-3-01
Universal Trial Number (UTN)
Trial acronym
CARE-PWS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - 3.2 mg intranasal carbetocin
Treatment: Drugs - 9.6 mg intranasal carbetocin
Treatment: Drugs - placebo

Placebo comparator: Placebo - matched placebo during first 8-weeks; prospectively randomized 1:1 to either one of the two doses of carbetocin during 56-week follow-up and optional extension periods

Experimental: 3.2 mg of LV-101 - 3.2 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods

Experimental: 9.6 mg of LV-101 - 9.6 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods


Treatment: Drugs: 3.2 mg intranasal carbetocin
three times per day with meals

Treatment: Drugs: 9.6 mg intranasal carbetocin
three times per day with meals

Treatment: Drugs: placebo
three times per day with meals
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Hyperphagia Behavior
Timepoint [1] 0 0
Baseline to Week 8
Primary outcome [2] 0 0
Obsessive and Compulsive Behaviors
Timepoint [2] 0 0
baseline to Week 8
Secondary outcome [1] 0 0
Anxiety
Timepoint [1] 0 0
Baseline to Week 8
Secondary outcome [2] 0 0
Global Impression
Timepoint [2] 0 0
Week 8
Secondary outcome [3] 0 0
Hyperphagia Behavior (Subset)
Timepoint [3] 0 0
Baseline to Week 8

Eligibility
Key inclusion criteria
* Genetically-confirmed Prader-Willi syndrome
* Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
* PWS Nutritional Phase 3 (hyperphagic, rarely feels full)
Minimum age
7 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Living in a group home
* Genetically diagnosed Schaaf-Yang syndrome or other genetic, hormonal, or chromosomal cognitive impairment
* New food-related interventions, including environment or dietary restrictions, within 1 month of screening
* Dose of any allowed chronic concomitant medications or supplements that have not been stable for =3 months prior to the study or is not expected to remain stable while participating in the study; adjustments in growth hormone dose =10% are not exclusionary
* Presence of cardiovascular disorders, epilepsy, frequent migraines, or severe asthma
* More than 3 episodes of sinusitis in the 12 months prior to Screening Visit or presence of nasal diseases that may affect deposition of intranasal medication
* Unwilling to abstain from nasal saline, other nasal irrigation, or other intranasal medications for 2 weeks prior to the Baseline visit and during the 8-week, placebo-controlled period of the study
* Use of weight loss medication, oxytocin, carbetocin, or vasopressin in the 6 months prior to screening
* Participation in an interventional research study involving another investigational medication or device in the 6 months prior to screening or during the study
* Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to unstable medical condition, inability to comply with the protocol, or other risk to subject or to the integrity of the study

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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/11/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/07/2022
Sample size
Target
Accrual to date
Final
130
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
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
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Oklahoma
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Tennessee
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Utah
Country [18] 0 0
Canada
State/province [18] 0 0
Alberta
Country [19] 0 0
Canada
State/province [19] 0 0
British Columbia
Country [20] 0 0
Canada
State/province [20] 0 0
Ontario
Country [21] 0 0
Canada
State/province [21] 0 0
Quebec

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Levo Therapeutics, 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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03649477