Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

— CARE-PWS  

Official 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03649477
• Other study ID #: LV-101-3-01
• Status: Completed
• Phase: Phase 3
• Start date: November 20, 2018
• Est. completion date: July 9, 2022

Study information

• Verified date: August 2021
• Source: Levo Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin). This study will also evaluate the safety and tolerability of LV-101.

Description:

This is a Phase 3 randomized, double-blind study with an 8-week, placebo-controlled period designed to test the effectiveness, safety, and tolerability of LV-101 in participants with PWS. Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviors, and anxiety. Safety and tolerability will be measured by adverse events, laboratory tests, and physical exams. After the 8-week placebo-controlled period, there will be a long-term follow-up period of 56 weeks and an optional extension period after study week 64 during which all participants will receive active treatment with LV-101. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 130
• Est. completion date: July 9, 2022
• Est. primary completion date: May 13, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 18 Years

Eligibility

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)

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• 3.2 mg intranasal carbetocin
three times per day with meals
• 9.6 mg intranasal carbetocin
three times per day with meals
• placebo
three times per day with meals

Locations

Country	Name	City	State
Australia	Queensland Children's Hospital	South Brisbane	Queensland
Canada	University of Alberta	Edmonton	Alberta
Canada	CHU Ste Justine	Montréal	Quebec
Canada	Toronto Hospital for Sick Kids	Toronto	Ontario
Canada	British Columbia Children's Hospital	Vancouver	British Columbia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	University of Harvard Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Florida	Gainesville	Florida
United States	Texas Children's Hospital	Houston	Texas
United States	Kansas University Medical Center	Kansas City	Kansas
United States	Children's Hospital of Los Angeles (USC)	Los Angeles	California
United States	Vanderbilt University School of Medicine	Nashville	Tennessee
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Cardinal Glennon Children's Medical Center	Saint Louis	Missouri
United States	Children's Hospitals and Clinics of Minnesota	Saint Paul	Minnesota
United States	University of Utah	Salt Lake City	Utah
United States	Children's Hospital of San Antonio	San Antonio	Texas
United States	Rady Children's Hospital San Diego	San Diego	California
United States	University of Oklahoma Health Sciences Center	Tulsa	Oklahoma
United States	Children's National	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Levo Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Hyperphagia Behavior	Change in hyperphagia (extreme hunger) as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Total Score versus placebo.; Score range: 0-36; higher scores mean a worse outcome. Reduction in score indicates improvement.	Baseline to Week 8
Primary	Obsessive and Compulsive Behaviors	Change in obsessive and compulsive behaviors as measured by the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) Total Score versus placebo.; Score range: 0-40; higher scores mean a worse outcome. Reduction in score indicates improvement.	baseline to Week 8
Secondary	Anxiety	Change in participant anxiety as measured by the PWS Anxiety and Distress Questionnaire (PADQ) Total Score versus placebo.; Score range: 0-56; higher scores mean a worse outcome. Reduction in score indicates improvement.	Baseline to Week 8
Secondary	Global Impression	Clinical Global Impression of Change (CGI-C) score versus placebo. Score range: 1-7; higher scores mean a worse outcome. Reduction in score indicates improvement.	Week 8
Secondary	Hyperphagia Behavior (Subset)	Change in hyperphagia as measured by the change in specified subsets of HQ-CT questions versus placebo.; Score range: 0-24; higher scores mean a worse outcome. Reduction in score indicates improvement.	Baseline to Week 8

External Links

•   Levo CARE-PWS Study