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

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.

Timeline

Start

2018-11-20

Primary completion

2020-05-13

Completion

2022-07-09

Outcome

Mixed primary results

registry Reduction in score indicates improvement. NCT03649477 ↗

registry analysis (superiority test); 3.2 mg of LV-101 vs Placebo; p = 0.0162; Mean Difference (Net) -3.136 (95% CI -5.685 to -0.586); NCT03649477 ↗

registry analysis (superiority test); 9.6 mg of LV-101 vs Placebo; p = 0.3493; Mean Difference (Net) -1.202 (95% CI -3.729 to 1.324); NCT03649477 ↗

registry analysis (superiority test); 9.6 mg of LV-101 vs Placebo; p = 0.6001; Mean Difference (Net) -0.608 (95% CI -2.890 to 1.674); NCT03649477 ↗

registry analysis (superiority test); 3.2 mg of LV-101 vs Placebo; p = 0.5143; Mean Difference (Net) -0.764 (95% CI -3.068 to 1.541); NCT03649477 ↗

Publications

• Roof E, Deal CL, McCandless SE, Cowan RL, Miller JL, Hamilton JK, Roeder ER, McCormack SE, Roshan Lal TR, Abdul-Latif HD, Haqq AM, Obrynba KS, Torchen LC, Vidmar AP, Viskochil DH, Chanoine JP, Lam CKL, Pierce MJ, Williams LL, Bird LM, Butler MG, Jensen DE, Myers SE, Oatman OJ, Baskaran C, Chalmers LJ, Fu C, Alos N, McLean SD, Shah A, Whitman BY, Blumenstein BA, Leonard SF, Ernest JP, Cormier JW, Cotter SP, Ryman DC. Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial. J Clin Endocrinol Metab. 2023 Jun 16;108(7):1696-1708. doi: 10.1210/clinem/dgad015.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Carbetocin	Peptide	3.2 mg	Intranasal
Subject	Carbetocin	Peptide	9.6 mg	Intranasal

Indications