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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 : November 20, 2018
Sponsor:
Information provided by (Responsible Party):
Levo Therapeutics, Inc.

August 24, 2018
August 28, 2018
November 20, 2018
November 20, 2018
December 2019   (Final data collection date for primary outcome measure)
  • 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
  • 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
Same as current
Complete list of historical versions of study NCT03649477 on ClinicalTrials.gov Archive Site
  • 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
  • Global impression [ Time Frame: baseline to Week 8 ]
    Change in Clinical Global Impression of Change (CGI-C) score versus placebo
  • 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
Same as current
Not Provided
Not Provided
 
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
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)
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.

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.

Interventional
Phase 3
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
Prader-Willi Syndrome
  • 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
  • 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
    Interventions:
    • Drug: intranasal carbetocin Dose 1
    • Drug: intranasal carbetocin Dose 2
    • Drug: placebo
  • Experimental: Dose 1 of LV-101
    Intervention: Drug: intranasal carbetocin Dose 1
  • Experimental: Dose 2 of LV-101
    Intervention: Drug: intranasal carbetocin Dose 2
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
175
Same as current
December 2020
December 2019   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
7 Years to 18 Years   (Child, Adult)
No
Contact: Levo MedInfo 847-901-9260 contactus@levotx.com
United States
 
 
NCT03649477
LV-101-3-01
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Levo Therapeutics, Inc.
Levo Therapeutics, Inc.
Not Provided
Not Provided
Levo Therapeutics, Inc.
November 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP