ClinicalTrials.gov record NCT07266324

A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old to 65 years old
SexAll
LocationAustralia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This phase 2, randomized, double-blind study compares oral BMB-101 with placebo in adults aged 18–65 who have genetically confirmed Prader‑Willi Syndrome and moderate-to-severe hyperphagia. The main blinded part lasts up to 16 weeks and includes a 4-week dose titration followed by 8 weeks of maintenance; there is an optional open-label extension afterward. Participants are assigned 1:1 to BMB-101 or matched placebo and have a mix of clinic and telephone visits.

What it is studying. The study tests the effects of oral BMB-101 compared with placebo on hyperphagia symptoms and on safety/tolerability in people with Prader‑Willi Syndrome.

What the researchers hope to learn. Measure change from baseline in Hyperphagia Questionnaire for Clinical Trials scores over 16 weeks. Secondary goals include caregiver and clinician global impression scales and changes in PWS-specific behavioral scores.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • BMB-101
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.
  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After a 4‑week screening period, participants are randomized 1:1 to receive either oral BMB‑101 or matched placebo twice daily. The active/placebo dosing includes a 4‑week weekly ascending dose (titration) followed by an 8‑week maintenance period (total randomized phase up to 16 weeks); there are about five clinic visits and four telephone visits during Part 1. Participants will complete caregiver- and clinician-rated questionnaires about hyperphagia and PWS behaviors; those who complete the blinded phase may, at the investigator's discretion, enter an unblinded open‑label extension to receive BMB‑101, or else will be tapered off study drug over 4 weeks.

How the study is structured. A randomized, double-blind, placebo-controlled trial with two parts. In Part 1 (blinded) participants are randomized 1:1 to receive BMB-101 or matched placebo with weekly ascending dose titration for 4 weeks then an 8-week maintenance period (total up to 16 weeks), with scheduled clinic and telephone visits. Part 2 is an optional, unblinded open-label extension where participants may receive BMB-101. Parallel groups and quadruple masking are used during the blinded phase.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in Australia. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Prader-Willi syndrome

Clinical trials19 trials listed for Prader-Willi syndromeEvery trial the registry lists for Prader-Willi syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Prader-Willi syndrome researchLatest monthly update: September 2026. Includes: Hunger symptoms. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
January 2026 (estimated)
Primary completion
November 2026 (estimated)
Study completion
March 2027 (estimated)
Target enrollment
16 participants (estimated)
Last updated on registry
Dec 5, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Bright Minds Biosciences Pty Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

Terms used on this page · explanations by MedicalUpdates.ai

double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
blinded
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
open label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Legally Authorized Representative
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
CT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
concomitant medications
Other medicines a participant takes during the study. Some must be reported; some are restricted.
estimated glomerular filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
ECG
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
1:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
Maximum Tolerated Dose
The highest dose found in a dose-escalation study that can be given without too many dose-limiting side effects.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials