Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

Last updated: July 18, 2022
Sponsor: Levo Therapeutics, Inc.
Overall Status: Completed

Phase

3

Condition

Hypogonadism

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT03649477
LV-101-3-01
  • Ages 7-18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Genetically-confirmed Prader-Willi syndrome
  • Provide voluntary, written informed consent (parent(s) / legal guardian(s) ofparticipant); provide voluntary, written assent (participants, as appropriate)
  • PWS Nutritional Phase 3 (hyperphagic, rarely feels full)

Exclusion

Exclusion Criteria:

  • Living in a group home
  • Genetically diagnosed Schaaf-Yang syndrome or other genetic, hormonal, or chromosomalcognitive 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 beenstable for ≥3 months prior to the study or is not expected to remain stable whileparticipating in the study; adjustments in growth hormone dose ≤10% are notexclusionary
  • Presence of cardiovascular disorders, epilepsy, frequent migraines, or severe asthma
  • More than 3 episodes of sinusitis in the 12 months prior to Screening Visit orpresence of nasal diseases that may affect deposition of intranasal medication
  • Unwilling to abstain from nasal saline, other nasal irrigation, or other intranasalmedications 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 monthsprior to screening
  • Participation in an interventional research study involving another investigationalmedication 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 theprotocol, or other risk to subject or to the integrity of the study

Study Design

Total Participants: 130
Study Start date:
November 20, 2018
Estimated Completion Date:
July 09, 2022

Study 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.

Connect with a study center

  • Sydney Children's Hospital

    Randwick, New South Wales 2031
    Australia

    Site Not Available

  • The Children's Hospital at Westmead

    Westmead, New South Wales 2145
    Australia

    Site Not Available

  • Queensland Children's Hospital

    South Brisbane, Queensland 4101
    Australia

    Site Not Available

  • University of Alberta

    Edmonton, Alberta T6G 2E1
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver, British Columbia V6H 3V4
    Canada

    Site Not Available

  • Toronto Hospital for Sick Kids

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • CHU Ste Justine

    Montréal, Quebec H3T 1C5
    Canada

    Site Not Available

  • University of Alabama at Birmingham

    Birmingham, Alabama 35233
    United States

    Site Not Available

  • Phoenix Children's Hospital

    Phoenix, Arizona 85006
    United States

    Site Not Available

  • Children's Hospital of Los Angeles (USC)

    Los Angeles, California 90027
    United States

    Site Not Available

  • Stanford Children's Health

    Palo Alto, California 94304
    United States

    Site Not Available

  • Rady Children's Hospital San Diego

    San Diego, California 92123
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's National

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32608
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Kansas University Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Harvard Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Children's Hospitals and Clinics of Minnesota

    Saint Paul, Minnesota 55102
    United States

    Site Not Available

  • Cardinal Glennon Children's Medical Center

    Saint Louis, Missouri 63104
    United States

    Site Not Available

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio 44106
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • University of Oklahoma Health Sciences Center

    Tulsa, Oklahoma 74135
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19107
    United States

    Site Not Available

  • Vanderbilt University School of Medicine

    Nashville, Tennessee 37212
    United States

    Site Not Available

  • Texas Children's Hospital

    Houston, Texas 77030
    United States

    Site Not Available

  • Children's Hospital of San Antonio

    San Antonio, Texas 78207
    United States

    Site Not Available

  • University of Utah

    Salt Lake City, Utah 84108
    United States

    Site Not Available

  • MultiCare Health System

    Tacoma, Washington 98405
    United States

    Site Not Available

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