A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
brief summary
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS). This study is designed as a multi-centre, double-blind, randomized, placebo controlled 2-part study with a blinded main phase followed up an open label extension phase.
detailed description
This study is designed as a 2-part study:
Part 1 is designed as a randomized phase, lasting up to 16 weeks. There will be a 4-week screening period. Following the screening period, participants will be randomized in a 1:1 ratio to either BMB-101 or placebo. Participants will enter into a weekly ascending Maximum Tolerated Dose (MTD) titration phase of 4 weeks followed by a maintenance phase of 8 weeks. There will be 5 clinic visits and 4 telephone visits.
Part 2 is designed to follow after the completion of the maintenance phase in Part 1. Participants at the discretion of the Investigator may elect to continue into an unblinded, expandable open label phase to receive BMB-101.
Participants who do not elect to continue into the open label phase will be tapered from assigned study treatment over 4 weeks following completion of the maintenance phase.
official title
A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Assess Efficacy, Safety and Tolerability of BMB-101 Oral Solution for the Treatment of Patients With Prader-Willi Syndrome (PWS)