The Liver Research Study
Currently Recruiting
10 May to 24 May 2026
Up to $4,370
Melbourne, Australia
The study drug is being developed for rare genetic liver diseases. These diseases happen because of a mistake in the DNA called a “nonsense mutation.” This mistake acts like a stop sign in the middle of an important gene, so the body can’t make the full protein it needs. Without these proteins, the body’s metabolism doesn’t work properly, leading to serious conditions like phenylketonuria (PKU), organic acidemias, and urea cycle disorders.
The study drug works by sending a special molecule into the liver using tiny fat-based particles. This molecule helps the body “read through” the stop signs in the gene, so it can finish making the protein correctly. In other words, it fixes the root cause of the problem instead of just treating symptoms.
Right now, there are no approved treatments that repair these mutations. Current options like strict diets or liver transplants only manage symptoms and can be very hard on patients. The study drug would be the first treatment designed to actually correct the genetic error, giving the body the ability to make the missing protein and potentially change the course of these diseases. Making this study an important trial to move medicine forward.
Join our clinical trial
This is a first-in-human (FIH) study, meaning it is 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
Trial details
Study duration
- 7 nights stay
- 1 follow-up visit
Study medication
Administered as an intravenous (IV) infusion
Additional eligibility
• Non-smokers or if current smoker must be willing to refrain from smoking during the
inpatient stay.
Ethics
This study has been reviewed and approved by an independent ethics committee.
Are you a match?
Age
18 - 65 years old
Remuneration
Up to $4,370 *with screening reimbursement. Please see T&Cs.
Gender
Male or Female
BMI
BMI 18 - 35 kg/m2
Commitment
7 nights, 1 clinic visit
Medical condition
Not taking any medications