Investigating a new target for treatment in Prader-Willi syndrome
Using acamprosate to investigate a new target for treatment in Prader-Willi Syndrome
The University of Sydney
30 participants
Sep 8, 2023
Interventional
Conditions
Summary
People with PWS and severe behaviour disturbance had significantly lower brain gamma-aminobutyric acid (GABA) levels than typically developing controls. GABA is the major inhibiting neurotransmitter in the brain, meaning it helps turn brain activity off. Unfortunately, there are currently no effective treatments for the core behaviours associated with PWS. We aim to address this issue by examining whether people with PWS show an increase in brain GABA levels in response to the medication acamprosate (a GABA modulator). If we find that they do, then this then this will help identify a new potential target for treatment in PWS.
Eligibility
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Interventions
This is a single-site, open-label, case-controlled trial of acamprosate in 15 individuals with Prader-Willi syndrome (PWS) and 15 typically developing controls. All participants will be exposed to the investigational product Acamprosate Calcium (Campral). Acamprosate will be delivered orally, via Campral tablets containing 333mg of acamprosate calcium as the active ingredient. Dosage will be in accordance to the TGA approved schedule, and a daily dose will be calculated according to body weight: For adults weighing 60 kg or more, the dose is 2 x 333mg tablets, taken three times daily (2 tablets in the morning, at midday and at night). For adults weighing less than 60 Kg, the dose is 2 x 333mg tablets in the morning, 1 x 333mg tablet at midday and 1 x 333mg tablet at night. Participants will take the recommended dosage for 10 days, and keep a medication log to record the time they take the medication during the day.
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ACTRN12619001779167