Prader-Willi SyndromePhase 2NCT07266324
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
A Phase 2 study testing BMB-101 in people with Prader-Willi Syndrome, run by Bright Minds Biosciences Pty Ltd at 2 sites in Australia.
Trial summary
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS). This study is designed as a multi-centre, double-blind, randomized, placebo controlled 2-part study with a blinded main phase followed up an open label extension phase. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- BMB-101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 2 sitesAustralia
- Study dates
- Jan 2026to Mar 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 Representative who is able to provide consent.
- Moderate to severe hyperphagia as defined by a HQ-CT score ≥ 13 at time of randomization (Visit 3).
- If participant is receiving growth hormone, the subject must be on the same medication and stable dose for at least 90 days prior to Visit 1.
- Female participant of childbearing potential must have a negative urine pregnancy test at baseline. Participants 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.
- Participant and/or caregiver has the ability to be compliant with study requirements, including visit schedule, diary completion and study drug accountability.
- If a caregiver assists in completion of questionnaires, the same caregiver is available to complete the questionnaires throughout the duration of the study.
Who cannot join
- Participant has used metabolic agents known to affect appetite within 3 months of Visit 1.
- Participant use of psychotropic medications including SSRIs/SNRIs, monoamine-oxidase inhibitors, tricyclic antidepressants, other serotonergic agonists or antagonists (antipsychotics), and other agents which have known Serotonin Syndrome risk (e.g. mirtazapine) within 1 month of Visit 1.
- Participant has implementation of new food restrictions or new environmental restrictions within 1 month of Visit 1.
- Participant has participated in an interventional clinical trial of any Prader-Willi Syndrome agent within 3 months of Visit 1 or any other investigational agent within 1 month of Visit 1.
- Participant 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.
- Participant has moderate or severe hepatic impairment. Asymptomatic participants 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.
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- Participant has severe renal impairment (estimated glomerular filtration rate <30mL/min/1.73m2).
- Participant has clinically significant ECG abnormality such as QTcF >450 msec (males) or >470 msec (females).
- Participant has a history of drug or alcohol abuse within the last 12 months or a positive urine drug screen.
- A current C-SSRS score of 4 or 5 at Visit 1 or history of suicide attempt at any time during the past year.
- Participant has a clinically significant condition or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Visit 1, other than PWS, that would negatively impact study participation, collection of study data, evaluation of study endpoints or pose a risk to the participant, in the opinion of the Investigator.
- Participant is pregnant (determined by a positive urine pregnancy test) or lactating female.
- Any condition that is thought to be a degenerative neurological disease.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia2 sites
Alfred Health
Melbourne, Victoria, 3004
Daniel Fineberg, +61 411403167, D.Fineberg@alfred.org.au
Royal Prince Alfred Hospital
Sydney, New South Wales, 2050
Tania Markovic, 02 9519 7605, tania.markovic@sydney.edu.au
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Assess Efficacy, Safety and Tolerability of BMB-101 Oral Solution for the Treatment of Patients With Prader-Willi Syndrome (PWS)
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS). This study is designed as a multi-centre, double-blind, randomized, placebo controlled 2-part study with a blinded main phase followed up an open label extension phase.
Read the detailed description
This study is designed as a 2-part study:
Part 1 is designed as a randomized phase, lasting up to 16 weeks. There will be a 4-week screening period. Following the screening period, participants will be randomized in a 1:1 ratio to either BMB-101 or placebo. Participants will enter into a weekly ascending Maximum Tolerated Dose (MTD) titration phase of 4 weeks followed by a maintenance phase of 8 weeks. There will be 5 clinic visits and 4 telephone visits.
Part 2 is designed to follow after the completion of the maintenance phase in Part 1. Participants at the discretion of the Investigator may elect to continue into an unblinded, expandable open label phase to receive BMB-101.
Participants who do not elect to continue into the open label phase will be tapered from assigned study treatment over 4 weeks following completion of the maintenance phase.
- Conditions
- Prader-Willi Syndrome
- Being tested
- BMB-101, Placebo
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMB-101Drug
Participants 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.
PlaceboDrug
Matched Placebo
| Study group | Role | What they receive |
|---|---|---|
| BMB-101 | Experimental | Participants receive BMB-101 (10mg/mL liquid) orally Receives: Drug: BMB-101 |
| Placebo | Placebo comparator | Participants receiving matched placebo orally Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants.
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.
Measured: 16 weeks
Secondary outcomes (5)
Change from Baseline in hyperphagia severity score as measured by the Caregiver Global Impression of Severity 7-point scale over time.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Measured: 16 weeks
Change from Baseline in hyperphagia severity score as measured by the Clinician Global Impression of Severity 7-point scale over time.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Measured: 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.
This scale is a single-item measure from 1 to 7, where higher scores indicate greater severity.
Measured: 16 weeks
Change from Baseline in improvement of Prader-Willi Syndrome disease scores as measured by the Clinician Global Impression of Improvement 7-point scale over time.
This scale is a single-item measure from 1 to 7, where 1 indicates very much improved, and 7 indicates very much worse.
Measured: 16 weeks
Change from Baseline in Prader-Willi Syndrome correlated behavioral issues such as symptoms measured by the Prader-Willi Syndrome Profile on a 3-point scale over time.
The Prader-Willi Syndrome Profile is a 57-item caregiver rated questionnaire that measures 52 Prader-Willi Syndrome specific behaviors on a 3-point scale (0-2), where 0=Not True, and 2=Often True. Higher scores indicate greater severity.
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2025
- Study startJan 2026 (estimated)
- Primary completionNov 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bright Minds Biosciences Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07266324
- Sponsor's protocol ID
- BMB-101-201
- Status last verified
- Nov 2025
- First submitted
- 17 Nov 2025
- Last updated
- 5 Dec 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Assess Efficacy, Safety and Tolerability of BMB-101 Oral Solution for the Treatment of Patients With Prader-Willi Syndrome (PWS)
- ClinicalTrials.gov ID
- NCT07266324
- Acronym / sponsor ID
- BMB-101-201
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Bright Minds Biosciences Pty LtdIndustry (pharma, biotech, devices)
- Study start
- Jan 2026 (estimated)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 5 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS). This study is designed as a multi-centre, double-blind, randomized, placebo controlled 2-part study with a blinded main phase followed up an open label extension phase.
Detailed description
This study is designed as a 2-part study:
Part 1 is designed as a randomized phase, lasting up to 16 weeks. There will be a 4-week screening period. Following the screening period, participants will be randomized in a 1:1 ratio to either BMB-101 or placebo. Participants will enter into a weekly ascending Maximum Tolerated Dose (MTD) titration phase of 4 weeks followed by a maintenance phase of 8 weeks. There will be 5 clinic visits and 4 telephone visits.
Part 2 is designed to follow after the completion of the maintenance phase in Part 1. Participants at the discretion of the Investigator may elect to continue into an unblinded, expandable open label phase to receive BMB-101.
Participants who do not elect to continue into the open label phase will be tapered from assigned study treatment over 4 weeks following completion of the maintenance phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BMB-101 | Experimental | Drug: BMB-101Participants receive BMB-101 (10mg/mL liquid) orally |
| Placebo | Placebo comparator | Drug: PlaceboParticipants receiving matched placebo orally |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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 Representative who is able to provide consent.
- Moderate to severe hyperphagia as defined by a HQ-CT score ≥ 13 at time of randomization (Visit 3).
- If participant is receiving growth hormone, the subject must be on the same medication and stable dose for at least 90 days prior to Visit 1.
Key exclusion criteria (13 listed)
- Participant has used metabolic agents known to affect appetite within 3 months of Visit 1.
- Participant use of psychotropic medications including SSRIs/SNRIs, monoamine-oxidase inhibitors, tricyclic antidepressants, other serotonergic agonists or antagonists (antipsychotics), and other agents which have known Serotonin Syndrome risk (e.g. mirtazapine) within 1 month of Visit 1.
- Participant has implementation of new food restrictions or new environmental restrictions within 1 month of Visit 1.
- Participant has participated in an interventional clinical trial of any Prader-Willi Syndrome agent within 3 months of Visit 1 or any other investigational agent within 1 month of Visit 1.
- Participant 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMB-101Participants 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. | Drug | BMB-101 |
| PlaceboMatched Placebo | Drug | Placebo |
Primary outcome
Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants.
Time frame: 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.
5 secondary outcomes
Change from Baseline in hyperphagia severity score as measured by the Caregiver Global Impression of Severity 7-point scale over time.
Time frame: 16 weeks
Change from Baseline in hyperphagia severity score as measured by the Clinician Global Impression of Severity 7-point scale over time.
Time frame: 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.
Time frame: 16 weeks
Change from Baseline in improvement of Prader-Willi Syndrome disease scores as measured by the Clinician Global Impression of Improvement 7-point scale over time.
Time frame: 16 weeks
Change from Baseline in Prader-Willi Syndrome correlated behavioral issues such as symptoms measured by the Prader-Willi Syndrome Profile on a 3-point scale over time.
Time frame: 16 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BMB-101 in people with Prader-Willi Syndrome, run by Bright Minds Biosciences Pty Ltd at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rachelle Kirk-Burnnand (+61 439615368, rachelle@basebio.com.au), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Hyperphagia Questionnaire for Clinical Trials scores over time in Prader-Willi Syndrome participants. (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2025.