About the study

This study is being conducted to investigate a potential new treatment for a rare muscle disease, called GNE

The study will evaluate the amount of the study treatment absorbed into the body after oral administration
compared to when it is administered via intravenous injection.

It is anticipated that this information will provide key data to further develop this potential new treatment for
patients with GNE myopathy.

What it involves

All volunteers are required to attend a screening evaluation (approximately 1 hour) at the PARC Clinical
Research Unit (Royal Adelaide Hospital) to determine suitability for study participation.

If you are selected for study participation, you will be required to attend the PARC Clinical Research Unit for 2
full days + 4 brief follow-up visits. During these visits you will receive the study treatment and will undergo
regular blood sampling and safety assessments.

Study participants will be paid for their time.


If you are male, aged 18-59 years and generally in good health, you may be eligible to participate in this trial.

Getting involved

For more information and to register your interest, please click on the button below.
Register Your Interest

Alternatively, please email researchclinic@adelaide.edu.au or phone (08) 7074 4404 to request information
about the ManNAc study.

This project has been approved by the Central Adelaide Local Health Network Human Research Ethics
Committee (ID 2022/HRE00117) and the Human Research Ethics Committee of the University of South Australia
(HREC Protocol Number 204476).