NCT03649477

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)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 130 (actual)
• Sponsor: Levo Therapeutics, Inc. · Industry
• Sex: All
• Age: 7 Years – 18 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Prader-Willi Syndrome

Interventions

Type	Name	Description
DRUG	3.2 mg intranasal carbetocin	three times per day with meals
DRUG	9.6 mg intranasal carbetocin	three times per day with meals
DRUG	placebo	three times per day with meals

Timeline

• Start date: 2018-11-20
• Primary completion: 2020-05-13
• Completion: 2022-07-09
• First posted: 2018-08-28
• Last updated: 2022-07-26
• Results posted: 2021-11-17

Locations

24 sites across 3 countries: United States, Australia, Canada

Regulatory

• FDA-regulated drug study