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BMB-101

Phase 2

Prader-Willi Syndrome | Small molecule | Rare Disease |Bright Minds Biosciences Inc.|Last Updated: Dec 5, 2025

Success Probability
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment16
FDA Designations
No designations recorded
Clinical trial landscape

BMB-101 · 3 trials · 6 indications

Phase 2 2Phase 1 1
NCT07266324A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.Prader-Willi Syndrome
NOT YET_RECRUITING16 Analytics
NCT06401538BMB-101 in Absence Epilepsy and DEEAbsence Epilepsy
RECRUITING20 Analytics
PHASE2NOT YET_RECRUITING
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
Prader-Willi Syndrome
NCT0726632416
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PHASE2RECRUITING
BMB-101 in Absence Epilepsy and DEE
Absence Epilepsy
NCT0640153820
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Study Endpoints
Primary Endpoints
Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants.
16 weeks

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 seizure frequency in DEE subjects
10 weeks

Seizure diary

Change from baseline in the number of generalized spike-wave discharges (GSWD) on the 24 hr EEG in Absence subjects.
6 weeks

24 hour EEG

Number of Treatment-emergent Adverse Events
Baseline up to Follow Up/End of Treatment visit, an average of 8 months.

Incidence and severity of adverse events, including serious adverse events and adverse events, clinically significant changes in laboratory testing, vital signs, Holter monitoring, physical examination, and ECGs

Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Administered at each of the following visits in Part 3: Screening, Clinic Discharge, and Follow-up/Early Withdrawal.

Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior

Secondary Endpoints
Change from Baseline in hyperphagia severity score as measured by the Caregiver Global Impression of Severity 7-point scale over time.
16 weeks
Change from Baseline in hyperphagia severity score as measured by the Clinician Global Impression of Severity 7-point scale over time.
16 weeks
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.
16 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BMB-101EXPERIMENTALParticipants receive BMB-101 (10mg/mL liquid) orally
PlaceboPLACEBO_COMPARATORParticipants receiving matched placebo orally
Interventions
NameTypeDescription
BMB-101DRUGParticipants 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.
PlaceboDRUGMatched Placebo
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Eligibility Criteria
Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * 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 Represe...

Countries:Australia
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06401538primaryCompletionDate: changed
LOWMay 26, 2026NCT07266324primaryCompletionDate: changed
LOWMay 24, 2026NCT06401538studyFirstPostDate: changed
LOWMay 24, 2026NCT07266324studyFirstPostDate: changed