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 children ages 7-18 as a treatment for hyperphagia and behavior associated with Prader-Willi syndrome.

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 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

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