RecruitingPhase 2ACTRN12619001779167

Investigating a new target for treatment in Prader-Willi syndrome

Using acamprosate to investigate a new target for treatment in Prader-Willi Syndrome

Sponsor

The University of Sydney

Enrollment

30 participants

Start Date

Sep 8, 2023

Study Type

Interventional

Conditions

Prader-Willi Syndrome

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

Sex: Both males and femalesMin Age: 18 YearssMax Age: 30 Yearss

Inclusion Criteria3

Aged between 18 to 30 years.
For participants with PWS, a confirmed genetic diagnosis of PWS.
Provide voluntary, written informed consent (all participants). For participants with PWS, parent / legal guardian also provides voluntary, written consent.

Exclusion Criteria9

Aged 17 years or younger and 31 years or older.
MRI contraindications (e.g. pregnant, metal in the body, insulin pumps, vertigo, claustrophobia, etc)
For PWS participants, comorbid psychiatric or neurological disorder that is not associated with PWS
For controls, no known neurological, psychiatric, neurogenetic or serious medical condition
Currently receiving treatment with a GABA compound
For non-GABA compounds, concomitant drug use will be allowed as long as there has been stable dosing for at least 4 weeks prior to participation
If evidence of potential renal failure creatine levels will be measured and participants with a creatinine clearance < 30 mL/min will be excluded.
Known sulphite hypersensitivity
Pregnant or breastfeeding women

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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 th

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.