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.
Participant must be aged 18-65 years (both inclusive).
Genetically confirmed diagnosis of Prader Willi Syndrome via standard DNA testing or other commonly approved methods.
Willing and able to provide voluntary written informed consent, or have a Legally Authorized Representative who is able to provide consent.
Moderate to severe hyperphagia as defined by a HQ-CT score ≥ 13 at time of randomization (Visit 3).
Participant has used metabolic agents known to affect appetite within 3 months of Visit 1.
Participant use of psychotropic medications including SSRIs/SNRIs, monoamine-oxidase inhibitors, tricyclic antidepressants, other serotonergic agonists or antagonists (antipsychotics), and other agents which have known Serotonin Syndrome risk (e.g. mirtazapine) within 1 month of Visit 1.
Participant has implementation of new food restrictions or new environmental restrictions within 1 month of Visit 1.
Participant has participated in an interventional clinical trial of any Prader-Willi Syndrome agent within 3 months of Visit 1 or any other investigational agent within 1 month of Visit 1.
Parallel
BMB-101
DrugParticipants will receive weekly ascending oral doses of BMB-101(10 mg/mL) twice daily (BID) for 16 weeks. Doses will be based on weight (kg) and will initially start at 1.67 mg/kg. Doses may be titrated in 0.33 mg/kg increments based on tolerability up to a maximum dose of 2.0 mg/kg.
Placebo
DrugMatched Placebo
Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants.
The Hyperphagia Questionnaire for Clinical Trials consists of 9 items; each rated on a scale from 0 (no symptoms) to 4 (severe symptoms). The total score ranges from 0 to 36, with higher scores indicating worse hyperphagia symptoms. A score of approximately 13 is associated with moderate to severe hyperphagia, and a score of 22 or greater is associated with severe hyperphagia.
Change from Baseline in hyperphagia severity score as measured by the Caregiver Global Impression of Severity 7-point scale over time.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Change from Baseline in hyperphagia severity score as measured by the Clinician Global Impression of Severity 7-point scale over time.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Change from Baseline in severity of Prader-Willi Syndrome disease scores as measured by the Clinician Global Impression of Severity 7-point scale over time.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Change from Baseline in improvement of Prader-Willi Syndrome disease scores as measured by the Clinician Global Impression of Improvement 7-point scale over time.
This scale is a single-item measure from 1 to 7, where 1 indicates very much improved, and 7 indicates very much worse.
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This trial is not recruiting at the moment. You can still explore other options:
