Referral Opportunity – Phase 2 Clinical Trial in Adults with Prader-Willi Syndrome (PWS)

Study Overview

This is a randomized, double-blind, placebo-controlled Phase 2a study assessing the efficacy, safety, and tolerability of BMB-101 oral solution in adults with genetically confirmed PWS.

The study aims to generate early clinical evidence on the potential of BMB-101 to improve hyperphagia and associated behavioral symptoms in this population. The study will enroll approximately 16–24 participants.

Participants will undergo a 1-month baseline, followed by a treatment period consisting of dose titration and a maintenance phase for a study duration of 4 months. Following the maintenance phase, participants will be offered enrollment into an optional open-label extension, where all participants will receive BMB-101.

STUDY AT A GLANCE

Protocol

BMB-101-201

Phase

Phase 2a

Study Design

Randomised (1:1), double-blind, placebo-controlled

Study Duration 

16 weeks

Participants

16–24

Age Range

18–65 years

Open Label Extension

9 + months


The study will be conducted at two sites in Australia: Sydney (Royal Prince Alfred Hospital, led by A/Prof Tania Markovic) and Melbourne (The Alfred, led by Principal Investigator Dr Daniel Fineberg). The Sponsor will cover travel and related expenses for patients and caregivers attending these locations


Key Eligibility Criteria

Inclusion Criteria:

  • Adults aged 18–65 years

  • Genetically confirmed Prader-Willi Syndrome

  • Moderate to severe hyperphagia (HQ-CT score ≥13)

  • Ability to comply with study procedures (with caregiver support if needed)

Key Exclusion Criteria:

  • Recent use of medications affecting appetite or serotonergic agents

  • Recent participation in another interventional clinical trial

  • Significant cardiovascular, hepatic, or renal disease

  • Clinically significant ECG abnormalities or recent substance abuse

Why Refer?

Patients with PWS often have limited treatment options for hyperphagia and associated behavioral challenges. This study offers:

  • Access to a novel investigational therapy

  • Close clinical monitoring and comprehensive assessments

  • Contribution to advancing therapeutic options for PWS

Referral Process

If you have patients who may be suitable for this study, we would greatly appreciate your referral. Our study team will conduct full eligibility screening and manage all study-related procedures.

To refer a patient or learn more, please contact:

We sincerely appreciate your support in advancing research for individuals with Prader-Willi Syndrome. Please feel free to reach out if you would like additional information or to discuss potential referrals.

This study is conducted in accordance with Good Clinical Practice and has received appropriate ethics approval.