Carbetocin for the Treatment of PWS

Study Purpose

Carbetocin (LV-101) is an investigational drug that was created to have effects in the body like oxytocin.  Carbetocin is not approved in the United States, but is approved in some other countries for treatment of women with excessive bleeding after giving birth via caesarean section. This Phase 3 study of intranasal carbetocin (LV-101) is a randomized, double-blind study with an 8-week, placebo controlled period, followed by a long-term follow-up period of 56 weeks 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.


A webinar sharing details on this study was recorded March, 2019. You can watch the webinar below or read a transcription of the webinar on our blog



Recruitment Criteria

Study Type: Interventional
Eligible Ages: 7-18 years
Other Criteria: PWS Nutritional Phase 3

Trial Details

Trial Id: NCT03649477
Phase: 3
Duration:  8 weeks of double blind, placebo controlled study, followed by 56 weeks of open label treatment
Lead Sponsor: Levo
Countries: United States
Additional Details: Outcome measures include changes in hyperphagia behavior, obsessive and compulsive behaviors, anxiety and  global impression 

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

This study will be available at the following sites:


United States, Alabama

University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Contact: Jacob Orr    205-638-6452   
Principal Investigator: Hussein Abdul-Latif, MD     

United States, Arizona

Phoenix Children's Hospital
Phoenix, Arizona, United States, 85006
Contact: Kiri Olsen    602-933-4112   
Principal Investigator: Oliver Oatman, MD        

United States, California

Children's Hospital of Los Angeles (USC)
Los Angeles, California, United States, 90027
Contact: Janelle Gonzalez   
Principal Investigator: Alaina Vidmar, MD         

Stanford Children's Health
Palo Alto, California, United States, 94304
Contact: Jake Urbassik 650-497-7528
Principal Investigator: Diane Stafford, MD

Rady Children's Hospital San Diego
San Diego, California, United States, 92123
Contact: Michael Farrell, RN    858-966-8453
Principal Investigator: Lynne Bird, MD        

United States, Colorado

Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Julia Smith    720-777-4030   
Principal Investigator: Shawn McCandless, MD      

United States, District of Columbia

Children's National
Washington, District of Columbia, United States, 20010
Contact: Jin Zhang   
Principal Investigator: Tamanna Ratti Roshan Lal, MD         
Sub-Investigator: Kenneth Rosenbaum, MD         

United States, Florida

University of Florida
Gainesville, Florida, United States, 32608
Contact: Beverly Giordano    352-294-5280   
Principal Investigator: Jennifer Miller, MD        

United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Contact: Amanda Barkho    312-503-7016
 Principal Investigator: Laura Torchen, MD        

United States, Kansas

Kansas University Medical Center
Kansas City, Kansas, United States, 66160
Contact: Yolanda Harness    913-574-0302   
Principal Investigator: Merlin Butler, MD        

United States, Massachusetts

University of Harvard Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Contact: Jasmina Cesko, RN 617-919-7555

Principal Investigator: Charumathi Baskaran, MD     

United States, Minnesota

Children's Hospitals and Clinics of Minnesota
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus    651-220-5730   
Principal Investigator: Melinda Pierce, MD         

United States, Missouri

Cardinal Glennon Children's Medical Center
Saint Louis, Missouri, United States, 63104
Contact: Barbara Whitman, PhD    314-268-4027   
Principal Investigator: Susan Myers, MD     

United States, Ohio

University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Contact: Ryan Ferrell   216-844-3661
Principal Investigator: Ryan Farrell, MD        

Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Contact: Emily Viall    614-722-2630   
Principal Investigator: Kathryn Obrynba, MD         

United States, Pennsylvania

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19107
Contact: Kristin Wade    267-426-8724   
Contact: Anna DeDio    215-425-1998   
Principal Investigator: Shana McCormack, MD   

United States, Tennessee

Vanderbilt University School of Medicine
Nashville, Tennessee, United States, 37212
Contact: Hailee Hunt    615-343-0915   
Principal Investigator: Ronald Cowan, MD         
Sub-Investigator: Elizabeth Roof         

United States, Texas

Texas Children's Hospital
Houston, Texas, United States, 77030
Contact: Andrew Meddaugh    832-824-1640   
Contact: Stanley Osegha   
Principal Investigator: Laurel Williams, MD      

Children's Hospital of San Antonio
San Antonio, Texas, United States, 78207
Contact: Katrina Roberts    210-704-4755   
Principal Investigator: Elizabeth Roeder, MD      

United States, Utah

University of Utah
Salt Lake City, Utah, United States, 84108
Contact: Carrie Bailey, BS, CCRC    801-587-3605   
Principal Investigator: David Viskochil, MD        

Australia, New South Wales

Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031
Contact Petrina Hetherrington +61 (0) 2 9382 5537
Principal Investigator: Ohn Nyunt, MD

The Children's Hopital at Westmead
Westmead, New South Wales, Australia, 2145
Contact: Amy Bruce +61 (0) 2 9845 1320
Principal Investigator: Yoon Hi Cho, MD

Australia Queensland

Queensland Children's Hospital
South Brisbane, Queensland, Australia 4101
Contact: Emma Brownrigg +61 (0) 7 3069 7505
Principal Investigator: Diane Jensen, MD

Canada, Alberta

University of Alberta
Edmonton, Alberta, Canada, T6G 2E1
Contact: Caroline Lyster    780-248-1770   
Principal Investigator: Andrea Haqq, MD         

Canada, British Columbia

British Columbia Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
Contact: Jennifer Kwan    604-875-2345 ext 5120   
Principal Investigator: Carol Lam, MD         
Sub-Investigator: Laura Stewart, MD      

Canada, Ontario

Toronto Hospital for Sick Kids
Toronto, Ontario, Canada, M5G 1X8
Contact: Kate Hamilton    416-813-7654 ext 201931   
Principal Investigator: Jill Hamilton, MD       

Canada, Quebec

CHU Ste Justine
Montréal, Quebec, Canada, H3T 1C5
Contact: Christine Massicotte, RN    514-345-4931 ext 3209   
Principal Investigator: Cheri Deal, MD    


Contact: Levo MedInfo 847-901-9260


New Call-to-action

Volunteer Your Time

No matter how big or how small, every effort helps us!