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

A New Clinical Trial for Prader-Willi Syndrome, Now Enrolling in Australia

If you or someone you love is living with Prader-Willi Syndrome, we'd love for you to learn about this new Phase 2 clinical trial from Bright Minds Biosciences — an investigational oral treatment now enrolling adults across Australia.

Take the Pre-Screener Find a Study Site About the Study
Study at a glance
Phase 2a  ·  Investigational oral treatment for PWS in adults
16 wk
Main study 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
Take the Pre-Screener Contact a Study Site

Now enrolling — PWFR AUS & PWSA AUS can help connect you with the study team.

About the Study

What this study is about

Three generations of a family smiling together in a kitchen

If you're caring for a family member with Prader-Willi Syndrome (PWS), you already know how much hyperphagia — that constant, hard-to-control feeling of hunger — and related behavioural symptoms can affect daily life. Bright Minds Biosciences has launched a Phase 2 clinical trial in Australia to explore whether a new investigational oral treatment could help.

Here's how the study is designed: it's randomised, double-blind, and placebo-controlled — which simply means treatment is assigned by chance, and neither you nor your doctor will know whether you're receiving the investigational treatment or a placebo (an inactive look-alike with no active medicine) until the study ends. Throughout, the study team will be watching closely to understand how well the treatment works, whether it's safe, and how well people tolerate it (i.e., any side effects).

This is an early-stage study — sometimes called a "proof-of-pharmacology" study — designed to see whether this new investigational oral treatment can help with both hyperphagia (excessive hunger) and the behavioural and psychiatric symptoms of PWS, such as anxiety, irritability, or mood changes.

This is the first time this investigational treatment is being studied in Australia for PWS, and we're glad to be able to offer it to families here.

The study runs for approximately 4 to 5 months. Afterward, participants may have the option to continue into a 9-month extension.

Everything about the study is registered publicly on clinicaltrials.gov, so you or your healthcare provider can review the full details anytime.

Study details
Phase
Phase 2a
Design
Randomised (1:1), double-blind, placebo-controlled — meaning treatment is assigned by chance, and neither you nor your doctor knows who's getting the investigational treatment vs. a placebo
Study aim
Hyperphagia (excessive hunger) and behavioural/psychiatric 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 — meaning everyone knows they're receiving the active investigational treatment (no more placebo group at this stage)
Location
Sydney & Melbourne, Australia
Registry
clinicaltrials.gov
Study Design

How the study works

Here's what to expect: an initial 16-week phase where treatment is assigned by chance and kept hidden from both you and your doctor (called "double-blind"), followed by an optional 9-month extension where everyone knows they're receiving the investigational treatment (called "open-label").

Screening
4 weeks
Dose Adjustment
4 weeks
Maintenance
8 weeks

In total, the double-blind phase lasts 16 weeks. After that, there's an optional 9-month extension — we call this "open-label" because at this point, everyone knows they're receiving the active investigational treatment.

5 clinic visits
4 phone calls
Oral liquid, twice daily
How treatment is assigned You'll be randomly assigned — like a coin flip — to receive either the active investigational treatment or a placebo (an inactive look-alike with no active medicine). Neither you nor your study doctor will know which one you're receiving during this double-blind phase. If you complete the study and choose to continue, you'll receive the active investigational treatment during the optional 9-month extension.
What is being measured
Primary endpoint
Changes in hyperphagia (hunger-related behaviour), measured using a caregiver questionnaire called the HQ-CT
Secondary endpoints
Changes in hyperphagia severity (rated by the doctor on a scale called CGI-S), body weight, and hormone levels (ghrelin and oxytocin, which affect hunger and social bonding)
Safety monitoring
Side effects (called "adverse events"), vital signs (blood pressure, heart rate), physical exams, lab tests, and a standard mood and safety questionnaire, including suicide risk (called the C-SSRS)
Pharmacokinetics (how the treatment moves through the body)
Blood samples taken to measure how much of the investigational treatment is in the body over time
Eligibility

Who can take part

A doctor talking with a patient

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.

You may qualify if you have
  • Males and females aged 18–65 years old
  • Genetically confirmed PWS via standard DNA testing
  • Moderate to severe hyperphagia, based on a caregiver questionnaire score (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
This might not be the right fit if you have
  • Recent use of metabolic agents for appetite, or psychiatric medications such as antidepressants (including SSRIs/SNRIs, MAOIs, or tricyclic antidepressants)
  • Cardiovascular disease, a significant ECG (heart tracing) abnormality, or severe liver or kidney disease
  • Pregnancy or breastfeeding
  • Recent participation in another PWS clinical trial
  • Degenerative neurologic disease, or alcohol or substance use disorder within the last 12 months
  • A suicide attempt within the last 12 months, or a current elevated score (4 or 5) on a standard suicide risk questionnaire (C-SSRS) used to help keep participants safe

Some of the items above — such as those relating to mental health history or substance use — are standard safety measures used across many clinical trials, not judgments about you or your family member. If you have questions about how any of these might apply to your situation, we encourage you to discuss them directly and confidentially with the study team rather than trying to self-screen based on this list alone.

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

Pre-Screener

See if You Qualify

Answer a few quick, friendly questions to find out if you or your family member may be eligible for the NOVA Trial. There's no obligation to move forward — think of it as a first step, not a commitment.

The pre-screener takes about 2 minutes and opens in a new tab.

Start the Pre-Screener  →
Trial Locations

Where the study is taking place

We're running the NOVA Trial at two specialist clinical sites — one in Sydney, one in Melbourne — so wherever you're located, our team is ready to help.

A doctor consulting with a patient in a clinical setting
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
We're now enrolling. Reach out directly to either study site above to learn more, or take the pre-screener to see if you or your family member may be eligible. PWFR AUS and PWSA AUS are also wonderful partners who can help connect you with the study team.
Advocacy Partners

Working with the Australian PWS community

We're proud to be working alongside PWS clinicians and advocacy groups to bring this important research — and real opportunities — to families across Australia.

PWRF
AUS
Prader-Willi Research Foundation Australia
PWRF AUS can connect interested families with the study team as the NOVA trial enrols participants across Australia.
PWSA
AUS
Prader-Willi Syndrome Association Australia
PWSA AUS is a key partner in bringing this research to the Australian PWS community and can help members connect with the study team now that enrolment is open.
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 ↗
FAQs

Common questions from caregivers

Once you're considering taking part, these are usually the next things families want to know.

What does a typical study visit look like?+

Visits generally include things like a physical exam, vital signs, blood tests, and a caregiver questionnaire about hyperphagia (the HQ-CT). The oral liquid investigational treatment is taken at home twice daily. Over the 16-week double-blind phase, that means 5 in-person clinic visits plus 4 shorter phone check-ins in between — your study coordinator will walk you through exactly what happens at each one before you commit to anything.

Will it cost us anything to take part?+

The investigational treatment itself and all study-related visits, tests, and procedures are provided at no cost to you. Every site handles things like travel a little differently, so it's worth asking your study site directly about what support may be available for your situation.

What if we don't live near Sydney or Melbourne?+

The NOVA Trial is currently only running at Royal Prince Alfred Hospital (Sydney) and The Alfred Hospital (Melbourne), so some travel may be involved if you're further away. Many families do make this work around the visit schedule above — the study site can talk through logistics, timing, and options with you before you decide.

Can we stop taking part if we change our mind?+

Yes. Taking part is entirely voluntary, and you or your family member can withdraw at any time and for any reason, without it affecting your usual medical care.

What if my family member doesn't qualify?+

That's alright — the criteria exist to help keep participants safe, not to judge anyone. If the NOVA Trial isn't the right fit, your local study site or PWFR AUS/PWSA AUS can point you toward other options, and may keep you in mind for future research opportunities.

Is this study safe?+

Yes. The NOVA Trial has been reviewed and approved by an independent Human Research Ethics Committee (see above), and the study team monitors participants closely throughout, including regular safety check-ins and lab tests. As with any investigational treatment, there are risks involved, and the study team will explain these in detail so you can make an informed decision before taking part.

Get Involved

Interested in participating?

The NOVA Trial is now enrolling, and we'd love to hear from you. Reach out directly to a study site in Sydney or Melbourne, take the pre-screener, or get in touch with PWFR AUS or PWSA AUS to register your interest.

Take the Pre-Screener Contact a Study Site View on ClinicalTrials.gov
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