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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03649477
Recruitment Status : Recruiting
First Posted : August 28, 2018
Last Update Posted : November 20, 2018
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.

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

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 identifier (NCT number): NCT03649477

Contact: Levo MedInfo 847-901-9260

United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32608
Contact: Beverly Giordano    352-294-5280   
Principal Investigator: Jennifer Miller, MD         
United States, Tennessee
Vanderbilt University School of Medicine Recruiting
Nashville, Tennessee, United States, 37212
Contact: Hailee Hunt    615-343-0915   
Principal Investigator: Ronald Cowan, MD         
Sub-Investigator: Elizabeth Roof         
Sponsors and Collaborators
Levo Therapeutics, Inc.

Responsible Party: Levo Therapeutics, Inc. Identifier: NCT03649477     History of Changes
Other Study ID Numbers: LV-101-3-01
First Posted: August 28, 2018    Key Record Dates
Last Update Posted: November 20, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

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

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