Purpose

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

Eligibility

Eligible Ages
Between 7 Years and 18 Years
Eligible Genders
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

Study Design

Phase
Phase 3
Study Type
Interventional
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 and the optional extension period
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo
matched placebo during first 8-weeks; randomized to either one of the two doses of carbetocin during 56-week follow-up and optional extension periods
  • 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

Recruiting Locations

Children's National and nearby locations

Children's National
Washington, District of Columbia 20010
Contact:
Jin Zhang
jzhang2@childrensnational.org

More Details

NCT ID
NCT03649477
Status
Recruiting
Sponsor
Levo Therapeutics, Inc.

Study Contact

Levo MedInfo
847-901-9260
contactus@levotx.com

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 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.