Clinical trial NCT03649477: Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Keywords: PWS
Prader-Willi syndrome
carbetocin
Lead sponsor: Levo Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2018-08-28
Enrollment: 130
Sex: ALL
Minimum age (years): 7
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Three parallel groups (two different doses of carbetocin and placebo) for the first 8 weeks; two parallel groups (two different doses of carbetocin) during 56 weeks of follow-up and the optional extension period
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Hyperphagia Behavior
timeFrame: Baseline to Week 8
measure: Obsessive and Compulsive Behaviors
timeFrame: baseline to Week 8
Secondary outcomes:
measure: Anxiety
timeFrame: Baseline to Week 8
measure: Global Impression
timeFrame: Week 8
measure: Hyperphagia Behavior (Subset)
timeFrame: Baseline to Week 8
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles (USC)
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Boston
country: United States
facility: University of Harvard Boston Children's Hospital
state: Massachusetts
city: Saint Paul
country: United States
facility: Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Cardinal Glennon Children's Medical Center
state: Missouri
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Tulsa
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University School of Medicine
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: San Antonio
country: United States
facility: Children's Hospital of San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: Toronto
country: Canada
facility: Toronto Hospital for Sick Kids
state: Ontario
city: Montréal
country: Canada
facility: CHU Ste Justine
state: Quebec
Study officials:
References: