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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06401538




Registration number
NCT06401538
Ethics application status
Date submitted
2/05/2024
Date registered
6/05/2024
Date last updated
17/07/2024

Titles & IDs
Public title
BMB-101 in Absence Epilepsy and DEE
Scientific title
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).
Secondary ID [1] 0 0
BMB-101-103
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Absence Epilepsy 0 0
Jeavons Syndrome 0 0
Dravet Syndrome 0 0
Lennox Gastaut Syndrome 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BMB-101

Experimental: BMB-101 - BMB-101 10 mg/ml liquid


Treatment: Drugs: BMB-101
BMB-101 liquid administered orally twice a day for 3 months
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline in seizure frequency in DEE subjects
Timepoint [1] 0 0
10 weeks
Primary outcome [2] 0 0
Change from baseline in the number of generalized spike-wave discharges (GSWD) on the 24 hr EEG in Absence subjects.
Timepoint [2] 0 0
6 weeks
Secondary outcome [1] 0 0
Change from baseline in quality of life
Timepoint [1] 0 0
6-10 weeks

Eligibility
Key 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. They should have at least 4 electrographic seizure discharges lasting at least 5 seconds on a 24 hr EEG during baseline.
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.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
15/09/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/10/2025
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Alfred Health - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bright Minds Biosciences Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06401538
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Terence O'Brien, MD
Address 0 0
The Alfred
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Suzy Cake
Address 0 0
Country 0 0
Phone 0 0
+61 408 771 971
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06401538