Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
- Conditions
- Prader-Willi Syndrome
- Interventions
- Drug: placeboDrug: 3.2 mg intranasal carbetocinDrug: 9.6 mg intranasal carbetocin
- Registration Number
- NCT03649477
- Lead Sponsor
- Levo Therapeutics, Inc.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 130
- 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)
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo 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 3.2 mg of LV-101 3.2 mg intranasal carbetocin 3.2 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods 9.6 mg of LV-101 9.6 mg intranasal carbetocin 9.6 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods
- Primary Outcome Measures
Name Time Method Hyperphagia Behavior Baseline to Week 8 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.Obsessive and Compulsive Behaviors baseline to Week 8 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.
- Secondary Outcome Measures
Name Time Method Anxiety Baseline to Week 8 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.Hyperphagia Behavior (Subset) Baseline to Week 8 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.Global Impression Week 8 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.
Trial Locations
- Locations (24)
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Harvard Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Vanderbilt University School of Medicine
🇺🇸Nashville, Tennessee, United States
Children's Hospital of San Antonio
🇺🇸San Antonio, Texas, United States
Cardinal Glennon Children's Medical Center
🇺🇸Saint Louis, Missouri, United States
Children's Hospital of Los Angeles (USC)
🇺🇸Los Angeles, California, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
British Columbia Children's Hospital
🇨🇦Vancouver, British Columbia, Canada
Kansas University Medical Center
🇺🇸Kansas City, Kansas, United States
Children's Hospitals and Clinics of Minnesota
🇺🇸Saint Paul, Minnesota, United States
University of Oklahoma Health Sciences Center
🇺🇸Tulsa, Oklahoma, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Queensland Children's Hospital
🇦🇺South Brisbane, Queensland, Australia
University of Alberta
🇨🇦Edmonton, Alberta, Canada
Toronto Hospital for Sick Kids
🇨🇦Toronto, Ontario, Canada
CHU Ste Justine
🇨🇦Montréal, Quebec, Canada
Children's National
🇺🇸Washington, District of Columbia, United States
Rady Children's Hospital San Diego
🇺🇸San Diego, California, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States