Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
Condition: Prader-Willi Syndrome
MeSH terms: Prader-Willi Syndrome; Syndrome
Intervention: 3.2 mg intranasal carbetocin; 9.6 mg intranasal carbetocin; placebo
Intervention description: three times per day with meals; three times per day with meals; three times per day with meals
Intervention MeSH terms: Carbetocin; Oxytocin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
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
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 130.0
Sex: All
Minimum age: 7 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: PWS; Prader-Willi syndrome; carbetocin

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 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