CARbetocin Efficacy and Safety Study in PWS

Research is uncovering new approaches to treating the hallmark symptoms of Prader-Willi syndrome.  Levo Therapeutics is currently enrolling patients with PWS in our Phase 3 clinical trial investigating intranasal carbetocin (LV-101) in 7-18 year olds as a treatment for hyperphagia and behavior associated with Prader-Willi syndrome.

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About intranasal carbetocin (LV-101)

Intranasal carbetocin is similar to the naturally-occurring hormone oxytocin, but with greater selectivity for the effect of interest – stimulation of oxytocin receptors.  These receptors are involved in the regulation of both social-emotional and feeding behaviors.

About the study

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 investigational 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.

All participants will receive active treatment with LV-101 after the 8-week placebo-controlled period, during a long-term follow-up period of 56 weeks. 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.

Study endpoints

In this study, we are studying the effect of LV-101 on various hallmark features of PWS, specifically hyperphagia, obsessive and compulsive symptoms, and anxiety.  To measure hyperphagia, caregivers will be asked to complete the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).  To measure obsessive and compulsive symptoms, study site professionals will ask caregivers about how these symptoms show up in their child with PWS.  To measure anxiety and distress in patients with PWS, caregivers will be asked to complete the PWS Anxiety and Distress Questionnaire (PADQ).

For more information about the CARE-PWS study, please view the trial listing at: NCT03649477

Study sites

Below is a listing of clinical study sites that are currently open and enrolling participants in the CARE-PWS study.  Please continue to come back to this website or to the trial listing on www.clinicaltrials.gov (here) for up-to-date information regarding open study sites.

United States

California
Rady Children’s Hospital San Diego
San Diego, California, United States, 92123
Contact: Rachel Winograd, RN   858-966-8453    rwinograd@rchsd.org
Principal Investigator: Lynne Bird, MD

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

Illinois
Ann & Robert H. Lurie Children’s Hospital of Chicago
Chicago, Illinois, United States, 60611
Contact: Sarayu Ratna  312-227-6617   sratnam@luriechildrens.org
Principal Investigator: Laura Torchen, MD

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

Minnesota
Children’s Hospitals and Clinics of Minnesota
Saint Paul, Minnesota, United States, 55102
Contact: Brittany Machus   651-220-5730   brittany.machus@childrensmn.org
Principal Investigator: Melinda Pierce, MD

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

Ohio
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn, PhD    216-844-7124    Audrey.Lynn@UHhospitals.org
Principal Investigator: Ryan Farrell, MD

Pennsylvania
Children’s Hospital of Philadelphia
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

Tennessee
Vanderbilt University School of Medicine
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

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

Texas Children’s Hospital
Houston, Texas, United States, 77030
Contact: Andrew Meddaugh   832-824-1640   axmeddau@texaschildrens.org
Contact: Stanley Osegha     sxosegha@texaschildrens.org
Principal Investigator: Laurel Williams, MD

Canada

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

British Columbia
British Columbia Children’s Hospital
Vancouver, BC, 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

Ontario
Toronto Hospital for Sick Kids
Toronto, Ontario, Canada, M5G 1X8
Contact: Kate Hamilton   416-813-7654 Ext. 201931   katharine.hamilton@sickkids.ca
Principal Investigator: Jill Hamilton, MD

Quebec
CHU Ste Justin
Montréal, Quebec, Canada, H3T 1C5
Contact: Christine Massicotte, RN   514-345-4931 Ext. 3209   christine.massicotte@recherche-ste-justine.qc.ca
Principal Investigator: Cheri Deal, MD

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