The PKU Study
Recruitment Closed
3 May to 19 May 2026
Up to $5,100
Melbourne, Australia
Phenylketonuria (PKU) is a rare condition that people are born with. It happens when the body cannot properly break down a part of protein called phenylalanine.
When phenylalanine builds up in the blood, it can damage the brain and cause learning problems, nerve problems, and mental health issues if it is not treated.
People with PKU are treated by eating very low‑protein diets or using special medicines, but these treatments can be hard to follow and do not work perfectly. The study drug is being developed to potentially treat PKU.
This study is being conducted with healthy volunteers as part of early research to help researchers understand the study drug, before it is tested in people living with PKU.
Join our clinical trial
This is not a first-in-human (FIH) study, meaning it is not the first time the potential treatment is being tested in people. The study has been carefully designed with strict safety protocols and has been approved by an independent ethics committee. It is not known whether the study drug may cause unexpected, serious, or life-threatening side effects. While these are considered unlikely, you will be carefully monitored throughout the study and provided with prompt treatment if any such effects occur.
Before joining, you’ll receive detailed information about the study, meet with a doctor, and have the chance to ask any questions.
What to expect
- Participation in a monitored clinical environment
- Study visits scheduled
- Support from a professional medical team
- Reimbursement for your time and commitment, which may be up to $5,100 if you are eligible.
Trial details
Study duration
- 10 nights stay
- 1 phone call
Study medication
Administered via oral tablets
Additional eligibility
- Non-smokers for at least 2 weeks prior to screening.
Ethics
An independent ethics committee has approved this clinical trial.
Are you a match?
Age
18 - 55 years old
Remuneration
Up to $5,100 *with screening reimbursement. Please see T&Cs.
Gender
Male or Female (Post-menopausal)
BMI
BMI 18 - 32 kg/m2
Commitment
10 nights
Medical condition
Not taking any medications