ANZCTR - Registration

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Trial registered on ANZCTR

Registration number

ACTRN12626001196336

Ethics application status

Approved

Date submitted

28/07/2026

Date registered

28/09/2026

Date last updated

28/09/2026

Date data sharing statement initially provided

28/09/2026

Type of registration

Retrospectively registered

Titles & IDs

Public title

A Randomized, Double-Blind, Placebo-Controlled, Phase I Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Oral Doses of BMB-105 in Fed and Fasted Adult Healthy Human Volunteers

Scientific title

Additional trial identifiers [1]

None

Universal Trial Number (UTN)

Trial acronym

BMB-105-101

Related trial records

Health condition

Health condition(s) studied:

Prader-Willi Syndrome (PWS)

Condition category

Condition code

Human Genetics and Inherited Disorders

Other human genetics and inherited disorders

Intervention/exposure

Study type

Interventional

Description of the intervention or exposure

Part 1 (SAD): Liquid suspension delivered orally
The planned dose cohorts in Part 1 are as follows:
• Cohort 1: BMB-105 0.8 mg/kg or matching placebo, once
• Cohort 2: BMB-105 1.71 mg/kg or matching placebo, once
• Cohort 3: BMB-105 2.29 mg/kg or matching placebo, once
• Cohort 4: BMB-105 6.0 mg/kg or matching placebo, once
All doses to be given in dedicated Phase I Clinic under supervision of Study nurses. Study drug / placebo will not be removed from Phase I Unit.

Part 2 (Food Effects): Liquid suspension delivered orally
• Fasted State: A single dose of BMB-105.
• Fed State: A single dose of BMB-105 after standard high-fat meal.
The two treatment levels will be selected following the Safety Review Committee (SRC) review of Part 1 data.
Participants will be discharged from the in-house clinic. After a washout period of seven days, post Day 4, participants will return to the in-house clinic for cross-over dosing period.
The high-fat breakfast will consist of the following:
Two eggs fried in butter, Two strips of bacon, Two slices of toast with butter, 125 g of hash brown potatoes, 250 ml of whole milk which results in approximately 865 kcal, 51 g fat, 66 g carbohydrate, and 35 g protein, with fat contributing approximately 53% of total energy intake.
All doses to be given in dedicated Phase I Clinic under supervision of Study nurses. Study drug / placebo will not be removed from Phase I Unit.
Part 3 (MAD): Liquid suspension delivered orally once daily for seven days.
The planned dose cohorts in Part 3 are as follows:
• Cohort 1: BMB-105 0.8 mg/kg or matching placebo × 7 days
• Cohort 2: BMB-105 1.71 mg/kg or matching placebo × 7 days
• Cohort 3: BMB-105 2.29 mg/kg or matching placebo × 7 days
• Cohort 4: BMB-105 6.0 mg/kg or matching placebo × 7 days

All doses to be given in dedicated Phase I Clinic under supervision of Study nurses. Study drug / placebo will not be removed from Phase I Unit.
Participants will be kept as Inpatients in Clinic for 11 continuous days (Day -1 until discharge on Day 10).

Intervention code [1]

Behaviour

Description of the comparator or control

Matched blinded oral placebo matching the dosing schedule of the active treatment.
0.5 mg/mL sucralose, 0.1 mg/mL strawberry flavouring, 1 mg/mL hydroxyethyl cellulose, 1.1 mg/mL methyl paraben and 0.22 mg/mL propylparaben.
Part 1 SAD: It is a matched placebo comparator delivered as a liquid suspension orally.
Part 2: Food Effects will receive blinded active drug, then cross over to blinded matched placebo comparator as above (or vice versa depending on randomisation).
Part 3 MAD: It is a matched placebo comparator delivered as a liquid suspension orally.

Control group

Placebo

Outcomes

Primary outcome [1]

Safety and tolerability - composite outcome

Timepoint [1]

Part 1: SAD Baseline, Day 1 (Treatment), Day 2, Day 3, Day 4 Day 7 (End of Study) Part 2: Baseline, Day 1 (Treatment), Day 2, Day 3, Day 4, Day 11 (Treatment), Day 12, Day 13, Day 14 , Day 15. Part 3: MAD Baseline, Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, (with Days 1-7 receiving treatment) Day 8, Day 9, Day 10, Day 15

Secondary outcome [1]

To investigate the effect of food on the PK of BMB-105 in healthy adult participants following administration of a single tolerable dose as determined from Part 1 (single ascending dose [SAD] phase of the study)

Timepoint [1]

Part 2 Visits: Baseline, Day 1 (Treatment), Day 2, Day 3, Day 4, Day 11 (Treatment), Day 12, Day 13, Day 14 , Day 15. Part 2 blood samples collected: Baseline- 0 hours / pre-dose and 0.5 hours, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours (Day 1), 24 hours (Day 2), 48 hours (Day 3), and 72 hours (Day 4) post-first dose, Day 11 (second dose), Day 12, Day 13, Day 14, Day 15.

Secondary outcome [2]

To assess the pharmacokinetic (PK) profiles in plasma and urine of BMB-105 following single and multiple oral administration of 4 ascending dose levels in healthy participants.

Timepoint [2]

Part 1: SAD Baseline, Day 1 (Treatment), Day 2, Day 3, Day 4 Day 7 (End of Study) Part 3: MAD Baseline, Day 1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, (with days 1-7 receiving treatment) Day 8, Day 9, Day 10, Day 15 Part 1 blood samples collected: SAD Baseline- 0 hours / pre-dose, and 0.5 hours, 1 hour, 1.5 hours, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, 12 hours post-first dose (Day 1), 24 hours (Day 2), 48 hours (Day 3), 72 hours (Day 4) and Day 7 (end of study) post-dose. Part 1 urine samples collected: Day 1 prior to dosing, between 1-4 hours pre-dose. After administration of study drug, all urine will be collected during the in-clinic observation period. Urine will be pooled per participant at the following planned intervals: 0–4 hours, 4–8 hours, 8-12 hours, 12–24 hours, 24–36 hours, 36–48 hours, and 48–72 hours post-dose. Part 3 blood samples collected on Day 1: 0 hours/pre-dose and 0.5 hours, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, and 12 hours post-dose; Days 2 to 6: pre-dose; Day 7: 0 hours / pre-dose, 0.5 hours, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, and 12 hours post-dose and Day 8: 24 hours post-last dose. Part 3: A spot urine sample will be collected on both Day 1 and Day 7 prior to administration of the dose of study drug, between 1-4 hours pre-dose. Additionally, all urine will be collected and pooled during the following planned intervals; Day 1 (3 samples): 0–4 hours, 4–8 hours, and 8–12 hours post-dose, Day 7 (3 samples): 0–4 hours, 4–8 hours, and 8–12 hours post-dose and Day 8 (1 sample): 24-28 hours post-last dose

Secondary outcome [3]

Part 3: Major metabolite profiles will be evaluated in the plasma samples at the dose most relevant to the targeted therapeutic level.

Timepoint [3]

Part 3: A total of 24 blood samples will be collected at the following timepoints: • Day 1 (9 blood samples): 0 hours/pre-dose and 0.5 hours, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, and 12 hours post-dose. • Days 2 to 6 (5 blood samples): pre-dose • Day 7 (9 blood samples): 0 hours / pre-dose, 0.5 hours, 1 hour, 2 hours, 3 hours, 4 hours, 6 hours, 8 hours, and 12 hours post-dose. • Day 8 (1 blood sample): 24 hours post-last dose

Eligibility

Key inclusion criteria

1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
2. Participant must be aged between 18 and 55 years (both inclusive) at the time of signing the informed consent.
3. Healthy participants with no clinically significant screening results in the opinion of the Investigator.
4. Body mass index (BMI) 18.0 to 32.0 kg/m2, inclusive.
5. Male and non-pregnant female
Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
a. Male participants:
i. Practicing true abstinence from at least 28 days prior to Day 1 and willing to continue until 3 months following the last dose of study drug, or
ii. Surgically sterilized (vasectomy – verbal confirmation acceptable), or
iii. Willing to use a condom during sexual activity throughout the study and for 3 months after last dose of study drug, plus appropriate contraceptive measures for your female partner. and
iv. Willing to not donate sperm for 3 months after last dose of study drug (non vasectomized participants).
b. Non-pregnant female participants:
i. Documented to be surgically sterile (verbal confirmation acceptable) or postmenopausal (amenorrhea greater than 1 year and follicle-stimulating hormone equal to 40 mU/mL); or
ii. Practicing true abstinence from at least 28 days prior to Day 1 and willing to continue until 1 month following the last dose of study drug, and must have a negative serum pregnancy test (women of childbearing potential [WOCBP] only) at screening and a negative urine pregnancy test (WOCBP only) on Day -1; or
iii. Practicing an effective method of contraception during treatment and for 1 month following the last dose of study drug and having a negative serum pregnancy test (WOCBP only) at Screening and a negative urine pregnancy test (WOCBP only) on Day -1.
iv. Willing to not donate eggs for 3 months after last dose of study drug.
6. Agree to frequent blood and urine sampling during the course of the study.
7. Agree to be confined in the study unit and follow study procedures.
8. Agree to comply with study-specified diet while confined in the Phase I unit.

Minimum age

18 Years

Maximum age

55 Years

Sex

Both males and females

Healthy volunteers

Yes

Key exclusion criteria

1. Participants with unstable or severe illness as indicated on medical history, physical examination, or clinical laboratory, vital signs, and ECGs evaluations, or in the opinion of the Investigator. Participants with a medical history of asthma or current medical condition of seasonal allergies may be included if treatment with concomitant antihistamine medications is not required.
2. Participants with reported history within past 6 months of, or current treatment for, any gastrointestinal (GI) disease that may impact the absorption of an oral drug for example gastroesophageal reflux disorder, peptic ulcer disease, inflammatory bowel disease., Gilbert’s syndrome. Participants who have had a cholecystectomy or appendectomy may be included if there is no clinical evidence of impaired GI absorption.
3. Participants with a history of seizures other than febrile seizures as a child.
4. Participants with a history of migraines.
5. Participants with any report of acute illness or febrile event that has not resolved within 72 hours of first dosing.
6. Participants with history of or current glucose intolerance; or with history of gestational diabetes.
7. Part 3 only: Participants with lifetime history of suicidal behavior or with lifetime history of suicidal ideation as indicated by the Columbia-Suicide Severity Rating Scale (C-SSRS). (Non-suicidal depression will not be exclusionary.)
8. Participants with any use of or intent to use any medications, including prescription, over-the-counter (OTC), herbal preparations, or vitamin/mineral supplementation, other than study medications, from 7 days prior or 5 half-lives (whichever is longer) (unless specified below) to first dose through follow-up visit (except for participants on a stable hormone replacement therapy (HRT) for at least 2 months, contraceptives as described in inclusion 5b and occasional paracetamol use, up to 2 g/day, prior to Day -1).
a. Recent (within 5 half-lives) or concomitant use of medications known to chronically alter drug absorption or elimination processes.
b. Recent (within 5 half-lives) or concomitant use of serotonergic medications or monoamine oxidase inhibitors.
i. These include but are not limited to, selective serotonin-norepinephrine reuptake inhibitors, selective serotonin reuptake inhibitors, tricyclic antidepressants, bupropion, triptans, dietary supplements (e.g., St. John’s Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors).
9. Participants who have received any vaccine within the 2 weeks prior to administration of first dose of study drug.
10. Participants who have participated (taken investigative drug and/or device) in another clinical trial within 30 days or 5 half-lives, whichever is longer, prior to first dose.
Diagnostic Assessments
11. Participants with creatinine clearance < 60 mL/min/1.73 m² (estimated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)) at Screening or Day -1.
12. Participants with any elevation of liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], gamma-glutamyl transferase [GGT], bilirubin, or alkaline phosphatase greater than 1.5 times the upper limit of normal range [ULN]) at Screening or Day -1.
13. Participants with a creatine kinase (CK) value of greater than 1.5 times the ULN (not clinically significant abnormality is acceptable) at Screening or Day -1, that is not explainable by exercise and that does not come back to reference range upon retest.
14. Participants with leucocytes or lymphocytes less than 60% of the lower limit of normal, eosinophils above the ULN or hemoglobin < 115 g/L for females, 125 g/L for males at Screening or Day -1.
15. Participants with proteinuria (more than trace) at Screening or Day -1. If the Investigator suspects a false positive result, one repeat is allowed.
16. Female participants with a positive pregnancy test at Screening or Day -1 or who are breastfeeding.
17. Participants with positive hepatitis B surface antigen, (except due to recent hepatitis B vaccination), hepatitis C virus (HCV), or human immunodeficiency syndrome (HIV).
18. Participants with a personal history of congenital long QT syndrome or a family history of congenital long QT or sudden cardiac arrest. Male participants with a resting Fridericia’s corrected QT interval (QTcF) value > 450 msec, and female participants with a resting QTcF value > 470 msec, as measured at Screening or Day -1.
19. Participants who have a heart rate < 45 or > 100 bpm after lying semi-supine or supine for 5 minutes at Screening or Day -1.
20. Participants who have an average blood pressure higher than 160/90 mm Hg at Screening or Day -1, as indicated in the Schedule of Assessments
21. Participants with a history of donation of blood or blood products, or significant blood loss (Investigator’s discretion) within 30 days prior to the first study dose, or if a participant has a diagnosed bleeding disorder (e.g., factor deficiency, coagulopathy, or platelet disorder requiring special precautions) or significant bruising or bleeding difficulties with blood draws or is unable to provide a blood sample without undue trauma or distress or has poor peripheral venous access and is unsuitable for cannulation or repeated venipuncture.
22. Participants who self-report having used more than 5 cigarettes, cigars, or nicotine-containing products per month within 6 months prior to first study dose, or plan to use them through completion of the follow-up visit.
23. History of drug or alcohol abuse or dependence (within the 1 years prior to Screening) as reported by the participant or known to the Investigator. Definition of alcohol abuse includes regularly consuming more than 10 standard drinks in a week or regularly consuming more than 4 standard drinks on any one day. One standard drink is 10 g of pure alcohol and is equivalent to 285 mL beer [4.9% Alc./Vol], 100 mL wine [12% Alc./Vol], 30 mL spirit [40% Alc./Vol] https://www.nhmrc.gov.au/health-advice/alcohol.
24. Participants with a positive qualitative drug screen for illegal drugs including cannabis at Screening or Day -1. If the Investigator suspects a false positive result, one repeat drug screen is allowed.
25. Participants who have not abstained from alcoholic beverages/alcohol-containing products at least 24 hours prior to first dose, or plan to consume them through completion of the follow-up visit.
26. Participants who have not abstained from red wine and/or grapefruit/Seville oranges at least 7 days prior to first dose, or plan to consume them through completion of the follow-up visit.
27. Participants who have consumed food containing poppy seeds during the study starting 72 hours before Screening or 72 hours before admission to study site (to avoid false positive urine drug test for opiates).
28. Participants who have not abstained from caffeinated beverages/caffeine or xanthine-containing products at least 24 hours prior to first study dose.
29. Participants with dietary restrictions who may not tolerate the house diet including the high fat diet in Part 2, in the in-patient unit as determined by the Investigator during the 28 days prior to the first study dose.
30. Participants who cannot refrain from strenuous exercise from 72 hours prior to first study dose through completion of the follow-up visit.
31. Participants who are employees of the study unit or their family members, students who are working in the study unit, or family members of the Investigator or Sponsor.
32. Hypersensitivity or idiosyncratic reaction to the study drug, naproxen, related compounds, or aspartame.

Study design

Purpose of the study

Treatment

Allocation to intervention

Randomised controlled trial

Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)

A randomisation schedule will be provided from the unblinded biostatistician to the unblinded pharmacy staff in a password protected file which will be used solely by the unblinded pharmacy staff or treatment assignment. Based on the randomisation schedule the unblinded pharmacy staff will prepare code break envelopes containing the treatment assignment information. If needed for intentional unblinding for safety reasons, the code break envelope will be used in the event of an emergency if the pharmacy staff are not available.

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

Part 1: Treatment is randomised 1:1 (BMB-105: Placebo) in the Sentinel group, and 4:1 (BMB-105: Placebo) for each of the SAD cohorts.
Part 2: Treatment is randomised 1:1 (BMB-105 in fed / fasted state) crossed over.
Part 3: Treatment is randomised 4:1 (BMB-105: Placebo) for each cohort.
The randomisation is a computer generated random sequence.

Masking / blinding

Blinded (masking used)

Who is / are masked / blinded?

Intervention assignment

Parallel

Other design features

Part 1 and 3: Parallel
Part 2: Crossover

Phase

Phase 1

Type of endpoint/s

Statistical methods / analysis

Recruitment

Recruitment status

Recruiting

Date of first participant enrolment

Anticipated

Actual

10/08/2026

Date of last participant enrolment

Anticipated

16/11/2026

Actual

Date of last data collection

Anticipated

26/12/2026

Actual

Sample size

Target

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

Recruitment hospital [1]

CMAX Clinical Research Pty Ltd - Adelaide

Recruitment postcode(s) [1]

5000 - Adelaide

Funding & Sponsors

Funding source category [1]

Commercial sector/Industry

Name [1]

Bright Minds Biosciences Pty Ltd

Address [1]

Country [1]

Australia

Primary sponsor type

Commercial sector/Industry

Name

Bright Minds Biosciences Pty Ltd

Address

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Bellberry Human Research Ethics Committee L

Ethics committee address [1]

https://bellberry.com.au/

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

20/05/2026

Approval date [1]

06/07/2026

Ethics approval number [1]

Summary

Brief summary

BMB-105 is a novel, potent, and selective 5-HT2C receptor agonist being developed for the treatment of hyperphagia and associated neurobehavioral symptoms in PWS. Preclinical pharmacokinetic data indicate good oral bioavailability.  
Part 1 SAD: will be 4 single dose cohorts, consisting of 8 participants each.  Dosing will be initiated at 0.8mg/kg (or matching placebo) once.
Part 2 Food Effect: 12 participants will have a single dose of BMB-105 with and without a meal 7 days apart (crossover). The dose will be determined following the SRC of Part 1 data.
Part 3 MAD: will be 4 cohorts of 8 participants each, using different doses.  Dosing will be initiated at 0.8mg/kg (or matching placebo) daily for 7 days.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Prof Sepehr Shakib

Address

CMAX Clinical Research, 21-24 North Terrace, Adelaide, SA, 5000

Country

Australia

Phone

+61 411100278

Fax

[email protected]

Contact person for public queries

Name

Sepehr Shakib

Address

CMAX Clinical Research 21-24 North Terrace, Adelaide, SA, 5000

Country

Australia

Phone

+61 8 7088 7900

Fax

[email protected]

Contact person for scientific queries

Name

Alex Vasilkevich

Address

Bright Minds Biosciences, 19 Vestry St, New York, NY, 10013-1975

Country

United States of America

Phone

+1 571 7224501

Fax

[email protected]

Data sharing statement

Will the study consider sharing individual participant data?

No
No IPD sharing reason/comment: There will be no de-identified, raw line-by-line individual participant data made publicly available for this trial.

What supporting documents are/will be available?

No Supporting Document Provided

Results publications and other study-related documents

Documents added manually

No documents have been uploaded by study researchers.

Documents added automatically

No additional documents have been identified.

Trial Review

Documents added manually

Documents added automatically