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
Status: recruiting
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                                                                                      Not yet recruiting
Birmingham, Alabama, United States, 35233
Contact: Tushima Reeves    205-638-6452    Treeves@peds.uab.edu   
Principal Investigator: Hussein Abdul-Latif, MD         

United States, Arizona

Phoenix Children's Hospital                                                                                                    Not yet recruiting
Phoenix, Arizona, United States, 85006
Contact: Kiri Olsen    602-933-4112    kolsen1@phoenixchildrens.com   
Principal Investigator: Oliver Oatman, MD         

United States, California

UCLA Children's Hospital of Los Angeles                                                                               Not yet recruiting
Los Angeles, California, United States, 90027
Contact: Janelle Gonzalez       janGonzalez@chla.usc.edu   
Principal Investigator: Alaina Vidmar, MD         

Rady Children's Hospital San Diego                                                                                        Not yet recruiting
San Diego, California, United States, 92123
Contact: Rachel Winograd, RN    858-966-8453    rwinograd@rchsd.org   
Principal Investigator: Lynne Bird, MD         

United States, Colorado

Children's Hospital Colorado                                                                                                    Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Audrey Elliot    720-777-5001    Audrey.Elliott@childrenscolorado.org   
Principal Investigator: Shawn McCandless, MD         

United States, District of Columbia

Children's National                                                                                                                   Not yet recruiting
Washington, District of Columbia, United States, 20010
Contact: Shanae Henry       snhenry@childrensnational.org   
Principal Investigator: Tamanna Ratti Roshan Lal, MD         
Sub-Investigator: Kenneth Rosenbaum, MD         

United States, Florida

University of Florida                                                                                                                  Recruiting
Gainesville, Florida, United States, 32608
Contact: Beverly Giordano    352-294-5280    bgiordano@peds.ufl.edu   
Principal Investigator: Jennifer Miller, MD         

United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago                                                             Not yet recruiting
Chicago, Illinois, United States, 60611
Contact: Alice Raad    312-227-4177    araad@luriechildrens.org   
Principal Investigator: Laura Torchen, MD         

United States, Kansas

Kansas University Medical Center                                                                                           Not yet recruiting
Kansas City, Kansas, United States, 66160
Contact: Yolanda Harness    913-574-0302    yharness@kumc.edu   
Principal Investigator: Merlin Butler, MD         

United States, Massachusetts

University of Harvard Boston Children's Hospital                                                                   Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Andrea Hale, RN       Andrea.Hale@childrens.harvard.edu   
Principal Investigator: Amy Fleischman, MD         

United States, Minnesota

Children's Hospitals and Clinics of Minnesota                                                                        Not yet recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus    651-220-5730    brittany.machus@childrensmn.org   
Principal Investigator: Melinda Pierce, MD         

United States, Missouri

Cardinal Glennon Children's Medical Center                                                                          Recruiting
Saint Louis, Missouri, United States, 63104
Contact: Barbara Whitman, PhD    314-268-4027    barbara.whitman@health.slu.edu   
Principal Investigator: Susan Myers, MD         

United States, Ohio
University Hospitals Cleveland Medical Center                                                                       Not yet recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynne, PhD    216-844-7124    Audrey.Lynn@UHhospitals.org   
Principal Investigator: Ryan Farrell, MD         

Nationwide Children's Hospital                                                                                            Not yet recruiting
Columbus, Ohio, United States, 43205
Contact: Julie Holbrook       Julie.Holbrook@nationwidechildrens.org   
Principal Investigator: Kathryn Obrynba, MD         

United States, Pennsylvania

Children's Hospital of Philadelphia                                                                                          Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Kristin Wade    267-426-8724    WadeKL@email.chop.edu   
Contact: Anna DeDio    215-425-1998    dedioa@email.chop.edu   
Principal Investigator: Shana McCormack, MD         

United States, Tennessee

Vanderbilt University School of Medicine                                                                               Recruiting
Nashville, Tennessee, United States, 37212
Contact: Hailee Hunt    615-343-0915    hailee.m.hunt@vanderbilt.edu   
Principal Investigator: Ronald Cowan, MD         
Sub-Investigator: Elizabeth Roof         

United States, Texas

Texas Children's Hospital                                                                                                      Not yet recruiting
Houston, Texas, United States, 77030
Contact: Debbie Barrera, RN       dxbarrer@texaschildrens.org   
Principal Investigator: Christopher Williams, MD         

San Antonio Children's Hospital                                                                                        Recruiting
San Antonio, Texas, United States, 78207
Contact: Katrina Roberts    210-704-4755    katrina.roberts@christushealth.org   
Principal Investigator: Elizabeth Roeder, MD         

United States, Utah

University of Utah                                                                                                                  Not yet recruiting
Salt Lake City, Utah, United States, 84108
Contact: Carrie Bailey, BS, CCRC    801-587-3605    carrie.bailey@hsc.utah.edu   
Principal Investigator: David Viskochil, MD         

Canada, Alberta

University of Alberta                                                                                                              Not yet recruiting
Edmonton, Alberta, Canada, T6G 2E1
Contact: Caroline Lyster    780-248-1770    clyster@ualberta.ca   
Principal Investigator: Andrea Haqq, MD         

Canada, British Columbia

British Columbia Children's Hospital                                                                                        Not yet recruiting
Vancouver, British Columbia, Canada, V6H 3V4
Contact: Jennifer Kwan    604-875-2345 ext 5120    jkwan7@cw.bc.ca   
Principal Investigator: Carol Lam, MD         
Sub-Investigator: Laura Stewart, MD         

Canada, Ontario

Toronto Hospital for Sick Kids                                                                                                   Not yet recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Kate Hamilton    416-813-7654 ext 201931    Katharine.hamilton@sickkids.ca   
Principal Investigator: Jill Hamilton, MD         

Canada, Quebec

CHU Ste Justine                                                                                                                       Recruiting
Montréal, Quebec, Canada, H3T 1C5
Contact: Caroline Champagne, inf.    514-345-4931 poste 6788       
Caroline.champagne@recherche-ste-justine.qc.ca
Principal Investigator: Cheri Deal, MD