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

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ClinicalTrials.gov Identifier: NCT03649477
Recruitment Status : Recruiting
First Posted : August 28, 2018
Last Update Posted : May 24, 2019
Sponsor:
Information provided by (Responsible Party):
Levo Therapeutics, Inc.

Brief 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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: intranasal carbetocin Dose 1 Drug: intranasal carbetocin Dose 2 Drug: placebo Phase 3

Detailed 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 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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 175 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Three parallel groups (two different doses of carbetocin and placebo) for the first 8 weeks; two parallel groups (two different doses of carbetocin) during 56 weeks of follow-up period
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin (LV-101) in Prader-Willi Syndrome (PWS) With Long Term Follow-Up (CARE-PWS)
Actual Study Start Date : November 20, 2018
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Placebo Comparator: Placebo
matched placebo during first 8-weeks; randomized to either one of the two doses of carbetocin during 56-week follow-up period
Drug: intranasal carbetocin Dose 1
three times per day before meals

Drug: intranasal carbetocin Dose 2
three times per day before meals

Drug: placebo
three times per day before meals

Experimental: Dose 1 of LV-101 Drug: intranasal carbetocin Dose 1
three times per day before meals

Experimental: Dose 2 of LV-101 Drug: intranasal carbetocin Dose 2
three times per day before meals




Primary Outcome Measures :
  1. Hyperphagia behavior [ Time Frame: baseline to Week 8 ]
    Change in hyperphagia (extreme hunger) as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Total Score versus placebo

  2. Obsessive and compulsive behaviors [ Time Frame: baseline to Week 8 ]
    Change in obsessive and compulsive behaviors as measured by the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) Total Score versus placebo


Secondary Outcome Measures :
  1. Anxiety [ Time Frame: baseline to Week 8 ]
    Change in participant anxiety as measured by the PWS Anxiety and Distress Questionnaire (PADQ) Total Score versus placebo

  2. Global impression [ Time Frame: baseline to Week 8 ]
    Change in Clinical Global Impression of Change (CGI-C) score versus placebo

  3. Hyperphagia behavior [ Time Frame: baseline to Week 8 ]
    Change in hyperphagia as measured by the change in specified subsets of HQ-CT questions versus placebo



Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03649477


Contacts
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Contact: Levo MedInfo 847-901-9260 contactus@levotx.com

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Locations
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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
Children's Hospital of Los Angeles (USC) 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 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: Julia Smith    720-777-4030    julia.smith@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: Jin Zhang       jzhang2@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 Recruiting
Chicago, Illinois, United States, 60611
Contact: Sarayu Ratnam    312-227-6617    sratnam@luriechildrens.org   
Principal Investigator: Laura Torchen, MD         
United States, Kansas
Kansas University Medical Center 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 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 Recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn, 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: Emily Viall    614-722-2630    Emily.Viall@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 Recruiting
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         
Children's Hospital of San Antonio 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 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 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 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: Christine Massicotte, RN    514-345-4931 ext 3209    christine.massicotte@recherche-ste-justine.qc.ca   
Principal Investigator: Cheri Deal, MD         
Sponsors and Collaborators
Levo Therapeutics, Inc.

Additional Information:
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Responsible Party: Levo Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03649477     History of Changes
Other Study ID Numbers: LV-101-3-01
First Posted: August 28, 2018    Key Record Dates
Last Update Posted: May 24, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Levo Therapeutics, Inc.:
PWS
Prader-Willi syndrome
carbetocin

Additional relevant MeSH terms:
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Syndrome
Prader-Willi Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Carbetocin
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs