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

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

Plain Language Summary

Simplified for easier understanding

Prader-Willi syndrome (PWS) is a rare genetic condition that causes a range of challenges including insatiable hunger, behavioural difficulties, and developmental delays. One of the most difficult aspects of PWS is that there are currently no effective medications to address the core behavioural symptoms. This study looks at whether a medication called acamprosate — normally used to help people with alcohol dependence — could boost levels of a brain chemical called GABA, which helps 'calm' brain activity. Researchers believe that low GABA levels in the brain may contribute to the challenging behaviours seen in PWS. Participants will take acamprosate for a period and then have brain scans (using a specialised MRI technique called MRS) to measure whether GABA levels have increased. A healthy control group without PWS will also participate for comparison. This study is open to adults aged 18 to 30, either with a confirmed genetic diagnosis of PWS or as a healthy control. Participants must not have MRI contraindications (such as metal implants or severe claustrophobia), must not currently be taking GABA-based medications, and must have adequate kidney function. Women who are pregnant or breastfeeding are not eligible.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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.


Locations(1)

ACT,NSW,NT,QLD,SA,TAS,WA,VIC, Australia

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ACTRN12619001779167


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