Testing BMB-101 for Absence Epilepsy and Related Conditions

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).

This study is testing a new treatment called BMB-101 to see if it can help reduce seizures in people with Absence Epilepsy and related conditions.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Bright Minds Biosciences Pty Ltd (Industry-sponsored)
• Locations: 5 sites (Randwick, New South Wales and 4 other locations)
• Trial ID: NCT06401538 on ClinicalTrials.gov

What this trial studies

This pilot, open-label study aims to evaluate the safety and effectiveness of BMB-101 in reducing seizure frequency in patients with Absence Epilepsy, Jeavons Syndrome, and Developmental Epileptic Encephalopathies such as Dravet and Lennox Gastaut syndromes. The study will last up to 6 months, including a 1-month screening period, followed by up to 3 months of treatment with BMB-101, and a 1-month follow-up. Participants will have six clinic visits throughout the study to monitor their progress and response to the treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with a diagnosis of Absence Epilepsy or Developmental Epileptic Encephalopathy who have not achieved adequate seizure control with existing medications.

Not a fit: Patients who have not been diagnosed with Absence Epilepsy or DEE, or those who have not responded to previous anti-seizure medications may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce seizure frequency and improve the quality of life for patients with these challenging epilepsy conditions.

How similar studies have performed: While this approach is novel for these specific conditions, similar studies targeting seizure reduction with new treatments have shown promise in the past.

Eligibility criteria

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.

Where this trial is running

Randwick, New South Wales and 4 other locations

• The Prince of Wales Hospital — Randwick, New South Wales, Australia (Recruiting)
• Royal Brisbane and Womans Hospital — Herston, Queensland, Australia (Recruiting)
• St Vincent's Hospital Melbourne — Fitzroy, Victoria, Australia (Recruiting)
• Austin Health — Heidelberg, Victoria, Australia (Recruiting)
• Alfred Health — Melbourne, Victoria, Australia (Recruiting)

Study contacts

• Principal investigator: Terence O'Brien, MD — The Alfred
• Study coordinator: Rachelle Kirk-Burnnand

Conditions Absence Epilepsy; Jeavons Syndrome; Dravet Syndrome; Lennox Gastaut Syndrome