Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

Phase 3 Completed 115 enrolled Soleno Therapeutics, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.

Timeline

Start: 2018-10-01
Primary completion: 2023-08-17
Completion: 2023-08-17

Outcome

Met primary endpoint

release “Primary endpoint demonstrated a statistically significant change from baseline in hyperphagia” soleno.life ↗

release “The study met its primary endpoint, demonstrating a highly statistically significant difference in change from baseline in HQ-CT total score” soleno.life ↗

Publications

Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.
Miller JL, Gevers E, Bridges N, Yanovski JA, Salehi P, Obrynba KS, Felner EI, Bird LM, Shoemaker AH, Angulo M, Butler MG, Stevenson D, Goldstone AP, Wilding J, Lah M, Shaikh MG, Littlejohn E, Abuzzahab MJ, Fleischman A, Hirano P, Yen K, Cowen NM, Bhatnagar A; C601/C602 Investigators. Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study. Obesity (Silver Spring). 2024 Feb;32(2):252-261. doi: 10.1002/oby.23928. Epub 2023 Nov 2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	DCCR	Unknown	—	Oral