Investigating a new target for treatment in Prader-Willi syndrome: PWS acamprosate trial

The purpose of the trial is to look at the effects of a medication, already approved in Australia (not for PWS), on the brain GABA levels in people with Prader Willi Syndrome (PWS) compared to typically developing people. This medication has been trialled in many studies, has a good safety profile, and is approved for use and regularly prescribed to treat alcohol withdrawal.

GABA is the major inhibiting neurotransmitter in the brain, meaning it helps regulate brain activity. Previously, we found that people with PWS had lower brain GABA levels compared to typically developing people. In people with PWS, lower GABA levels were associated with temper outbursts, skin-picking and autism-like behaviours. Similar studies using this medication have been conducted in people with Autism and Fragile X syndrome and have found improvements in behaviour.

Participants will be invited to complete:

• two clinic visits (2.5 hrs each)
• 10-day course of the drug
• two MRI scans
• one blood draw

You may be reimbursed for any reasonable travel, parking, meals and other expenses associated with the research project visit.

You may be eligible to participate in this study if you:

• are aged 18 to 30 years
• have no known neurological, psychiatric, neurogenetic or serious medical condition
  

You will not be able to participate in this study if you:

• Are pregnant or breastfeeding
• Have any MRI contraindications
• Have a known neurological, psychiatric, neurogenetic or serious medical condition
• Are currently receiving treatment with a GABA compound
• Have been using or have had a change in the dose of a non-GABA compound medication in the 4 weeks prior to participation.
• Have evidence of renal failure
• Have severe hepatic failure
• Have a known sulphite hypersensitivity

If you are interested in assisting with this important and exciting area of research, please contact the study facilitator:

Phone: 02 8627 6048
Email: clinicaltrials.boden@sydney.edu.au

Contacting us for additional information does not obligate you to participate in the study.