BMB-101 in Absence Epilepsy and DEE

An Open-Label Phase 2 Study to Evaluate the Efficacy, Safety and Tolerability of BMB-101 in Adults With Either Classic Absence Epilepsy (With or Without Eyelid Myoclonia (EEM; Jeavons Syndrome), OR Developmental Epileptic Encephalopathy (DEE).

The study is a pilot, open-label, study to test whether BMB-101 is safe and effective in reducing the frequency of seizures in subjects with Absence Epilepsy including Epilepsy with Eyelid Myoclonia (also called Jeavons Syndrome) as well as Developmental Epileptic Encephalopathies such as Dravet and Lennox Gastaut. The study will last up to 6 months. There will be a 1 month screening period, then up to 3 months on open-label BMB-101 including titration and tapering/washout periods, and then a 1 month follow-up period. There will be 6 clinic visits.

Study Overview

• Status: Recruiting
• Conditions: Dravet Syndrome; Absence Epilepsy; Lennox Gastaut Syndrome; Jeavons Syndrome
• Intervention / Treatment: Drug: BMB-101

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Rachelle Kirk-Burnnand; Phone Number: +61 439615368; Email: rachelle@basebio.com.au

Study Locations

• Australia
  • New South Wales
    • Randwick, New South Wales, Australia, 2031
      • Recruiting
      • The Prince of Wales Hospital
      • Contact: Christian Zentner; Phone Number: +612 9382 2437; Email: Christian.Zentner@health.nsw.gov.au
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Recruiting
      • Royal Brisbane and Womans Hospital
      • Contact: David Reutens; Phone Number: +61 7 3646 2523; Email: d.reutens@uq.edu.au
  • Victoria
    • Fitzroy, Victoria, Australia, 3065
      • Recruiting
      • St Vincent's Hospital Melbourne
      • Contact: Wendyl D'Souza; Phone Number: +61 3 9288 3069; Email: wendyl.dsouza@svhm.org.au
    • Heidelberg, Victoria, Australia, 3084
      • Recruiting
      • Austin Health
      • Contact: Ingrid Scheffer; Phone Number: +61 3 9035 7116; Email: i.scheffer@unimelb.edu.au
    • Melbourne, Victoria, Australia, 3004
      • Recruiting
      • Alfred Health
      • Contact: Terrence O'Brien, MD; Phone Number: +61 3 9903 0855; Email: te.obrien@alfred.org.au

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects must have a diagnosis of Absence Epilepsy with or without eyelid myoclonia (Jeavons Syndrome) or a diagnosis of Developmental and Epileptic Encephalopathy (DEE) such as Dravet syndrome or Lennox-Gastaut syndrome or other DEE.
2. Subjects with Absence must experience at least 4 episodes of 3-4/second SWD lasting at least 3 seconds each in a 24 hour EEG during the baseline period. Those with DEE must have a typical EEG pattern for DEE on routine EEG and experience at least 4 seizures during the 4 week baseline period prior to BMB-101 administration.
3. Subjects can be male or female ages 18-65 inclusive at time of baseline.
4. Subject must have tried at least one anti-seizure medication at a recommended dose and duration and must be on a stable dose on their current anti-seizure medications for at least 4 weeks prior to baseline and remain stable throughout the study.
5. Subjectis willing and able to be compliant with diary completion, visit schedule, and study drug accountability.
6. Female subjects of childbearing potential must have a negative urine pregnancy test at baseline. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while in this study and for 90 days after the last dose of study drug.

Exclusion Criteria:

1. Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, pulmonary hypertension, myocardial infarction or stroke, or clinically significant structural cardiac abnormality.
2. Subject has moderate or severe hepatic impairment. Asymptomatic subjects with mild hepatic impairment (elevated liver enzymes < 3x upper limit of normal (ULN) and/or elevated bilirubin <2x ULN) may be entered into the study after review and approval by the Medical Monitor in conjunction with the sponsor, in consideration of comorbidities and concomitant medications.
3. Subject has severe renal impairment (estimated glomerular filtration rate <30mL/min/1.73m2)
4. Clinically significant ECG abnormality such as QTcF >450 msec (males) or >470 msec (females)
5. Subject is receiving concomitant therapy with: fenfluramine, lorcaserin, monoamine-oxidase inhibitors, SSRIs, SNRIs, tricyclic antidepressants or other serotonergic agonists or antagonists (antipsychotics).
6. Subject is currently receiving an investigational medicinal product.
7. Subject has participated in another clinical trial within the past 30 days (calculated from that study's last scheduled visit). Participation in non-treatment trials will be reviewed by the medical monitor.
8. Subject has a history of drug or alcohol abuse within the last 12 months or a positive urine drug screen (with the exception of cannabinoids).
9. A current C-SSRS score of 4 or 5 at baseline or history of suicide attempt at any time during the past year
10. Subject has a clinically significant condition or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Baseline Visit, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BMB-101; BMB-101 10 mg/ml liquid	Drug: BMB-101; BMB-101 liquid administered orally twice a day for 3 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in seizure frequency in DEE subjects	Seizure diary	10 weeks
Change from baseline in the number of generalized spike-wave discharges (GSWD) on the 24 hr EEG in Absence subjects.	24 hour EEG	6 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in quality of life	Quality of Life in Epilepsy (QOLIE-31) for adults	6-10 weeks

Collaborators and Investigators

• Sponsor: Bright Minds Biosciences Pty Ltd
• Investigators: Principal Investigator: Terence O'Brien, MD, The Alfred

Study record dates

Study Major Dates

• Study Start (Actual): December 5, 2024
• Primary Completion (Estimated): September 15, 2025
• Study Completion (Estimated): November 30, 2025

Study Registration Dates

• First Submitted: May 2, 2024
• First Submitted That Met QC Criteria: May 3, 2024
• First Posted (Actual): May 6, 2024

Study Record Updates

• Last Update Posted (Actual): August 12, 2025
• Last Update Submitted That Met QC Criteria: August 7, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Epileptic Syndromes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Genetic Diseases, Inborn; Disease; Epilepsy, Generalized; Syndrome; Epilepsy; Epilepsies, Myoclonic; Epilepsy, Absence; Lennox Gastaut Syndrome
• Other Study ID Numbers: BMB-101-103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No