Ethics approved: Human Research Ethics Committee (RPAH Zone), Sydney Local Health District  ·  Approval No. X25-0286
Phase 2 Clinical Trial  ·  Now Recruiting  ·  Australia

Bright Minds Biosciences Announces Australian Trial for Prader-Willi Syndrome Treatment

Bright Minds Biosciences is offering a new clinical trial opportunity for the PWS community with the launch of their Phase 2 clinical trial in Australia, set to begin in Q1 2026.

Study at a glance
Phase 2a  ·  Oral medication for PWS in adults
16 wk
Double-blind phase
18–65
Age range (years)
16–24
Approx. participants
+9 mo
Possible extension study
Australian study sites
Royal Prince Alfred Hospital Sydney  ·  Dr. Tania Markovic, PI
The Alfred Hospital Melbourne  ·  Dr. Daniel Fineberg, PI
Contact a Study Site

PWFR AUS & PWSA AUS will keep families updated as enrolment opens.

About the Study

A new oral medication as a possible treatment for PWS

Bright Minds Biosciences has launched a Phase 2 clinical trial in Australia, exploring a new oral treatment aimed at reducing hyperphagia and behavioural symptoms in Prader-Willi Syndrome in adults.

The trial will evaluate the new oral medication as a possible treatment for Prader-Willi Syndrome. This randomised, double-blind, placebo-controlled study will assess the drug's efficacy, safety, and tolerability in adult PWS patients.

This proof-of-pharmacology clinical study is designed to evaluate a new potential oral treatment's utility in addressing both hyperphagia (excessive hunger) and the behavioural and neuropsychiatric symptoms of PWS.

This is the first Australian clinical study of this investigational treatment for PWS.

The study will run for approximately 4 to 5 months. At the conclusion of this initial study period, participants may have the opportunity to enrol in a 9-month extension study.

The study is registered on clinicaltrials.gov, where full details are publicly available for review.

Study details
Phase
Phase 2a
Design
Randomised (1:1), double-blind, placebo-controlled
Study aim
Hyperphagia and behavioural/neuropsychiatric symptoms of PWS
Participants
Approximately 16–24
Who can join
Adults with PWS, males and females aged 18–65
Dosing
Oral liquid, twice daily (0.67–2.0 mg/kg based on tolerance)
Duration
~4 to 5 months (16-week double-blind phase)
Visits
5 clinic visits + 4 phone calls
Extension
Optional 9-month open-label extension (everyone receives the active study treatment)
Location
Sydney & Melbourne, Australia
Study Design

How the study works

A randomised, double-blind, placebo-controlled 16-week study followed by an optional open-label extension.

Screening
4 weeks
Dose Titration
4 weeks
Maintenance
8 weeks

Total double-blind phase: 16 weeks. An optional 9-month open-label extension follows, where all participants receive the active study treatment.

5 clinic visits
4 phone calls
Oral liquid, twice daily
Randomised 1:1 Participants are randomly assigned to receive either the active study treatment or placebo. Neither the participant nor the study doctor will know which is received during the double-blind phase. All participants who complete the study and choose to continue will receive the active study treatment in the optional 9-month extension.
What is being measured
Primary endpoint
Changes in hyperphagia (behaviour) questionnaire measured by HQ-CT
Secondary endpoints
Changes in hyperphagia severity (CGI-S), weight, hormone levels (ghrelin, oxytocin)
Safety monitoring
Adverse events, vital signs, physical exam, lab tests, suicide rating (C-SSRS)
Pharmacokinetics
Serum samples to measure concentration of the study treatment
Eligibility

Who can take part

👥

Not sure if you or your family member is eligible?

Contact your local study site or PWS healthcare provider for more information. The criteria below are a guide — a member of the study team will be able to answer your specific questions.

Must have
  • Males and females aged 18–65 years old
  • Genetically confirmed PWS via standard DNA testing
  • Moderate to severe hyperphagia (HQ-CT score ≥13)
  • Consistent caregiver available throughout the study
  • Signed informed consent, or a legally authorised representative who can sign
  • If on growth hormone, must be at a stable dose for 90+ days
  • Willing to use medically acceptable birth control during the study and for 90+ days after
Cannot have
  • Recent use of metabolic agents for appetite, or psychotropic medications including SSRIs/SNRIs, monoamine-oxidase inhibitors, or tricyclic antidepressants
  • Cardiovascular disease, significant ECG abnormality, or severe liver or kidney disease
  • Pregnancy or breastfeeding
  • Recent participation in another PWS clinical trial
  • Degenerative neurologic disease or history of alcohol or drug abuse in the last 12 months
  • History of suicide attempt in the last 12 months or C-SSRS score of 4 or 5

Contact your local study site or PWS healthcare provider for more information about eligibility. Full inclusion and exclusion criteria are available on clinicaltrials.gov.

Trial Locations

Where the study is taking place

The NOVA trial is being conducted at two specialist clinical sites in Sydney and Melbourne.

Sydney, New South Wales
Royal Prince Alfred Hospital
Primary Investigator: Dr. Tania Markovic
Enquire about this site
clinicaltrials.boden@sydney.edu.au
Melbourne, Victoria
The Alfred Hospital
Primary Investigator: Dr. Daniel Fineberg
Enquire about this site
NeurologyClinicaltrials@alfred.org.au
Enrolment opening soon. PWFR AUS and PWSA AUS will keep patients and families interested in participating updated as enrolment approaches. You can also reach out directly to either study site above for more information.
Advocacy Partners

Working with the Australian PWS community

Bright Minds Biosciences looks forward to working with PWS clinicians and advocacy groups to bring this important research to families across Australia.

PWRF
AUS
Prader-Willi Research Foundation Australia
PWRF AUS will keep patients and families updated as enrolment in the NOVA trial approaches, and can connect interested families with the study team.
PWSA
AUS
Prader-Willi Syndrome Association Australia
PWSA AUS is a key partner in bringing this research to the Australian PWS community and will keep members informed as enrolment opens.
Ethics & Approval

Ethics approval & complaints

Human Research Ethics Committee Approval
This study has been approved by the Human Research Ethics Committee (RPAH Zone) of the Sydney Local Health District (SLHD).

Any person with concerns or complaints about the conduct of this study should contact the Executive Officer:

  📞  02 9515 6766
  ✉  SLHD-RPAEthics@health.nsw.gov.au

Please quote ethics approval number: X25-0286
Ethics No. X25-0286 SLHD HREC (RPAH Zone) Phase 2a Registered on ClinicalTrials.gov ↗
Get Involved

Interested in participating?

Reach out directly to a study site in Sydney or Melbourne, or contact PWFR AUS or PWSA AUS to register your interest and be kept updated as enrolment opens.

Sydney Site
Royal Prince Alfred Hospital
Dr. Tania Markovic, PI
clinicaltrials.boden@sydney.edu.au
Melbourne Site
The Alfred Hospital
Dr. Daniel Fineberg, PI
NeurologyClinicaltrials@alfred.org.au
Study Sponsor
Bright Minds Biosciences Inc.
brightmindsbio.com
clinicaltrials.gov