Investigating a new target for treatment in Prader-Willi syndrome
- Conditions
- Prader-Willi SyndromeHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12619001779167
- Lead Sponsor
- The University of Sydney
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot yet recruiting
- Sex
- All
- Target Recruitment
- 30
1. Aged between 18 to 30 years.
2. For participants with PWS, a confirmed genetic diagnosis of PWS.
3. Provide voluntary, written informed consent (all participants). For participants with PWS, parent / legal guardian also provides voluntary, written consent.
1.Aged 17 years or younger and 31 years or older.
2.MRI contraindications (e.g. pregnant, metal in the body, insulin pumps, vertigo, claustrophobia, etc)
3.For PWS participants, comorbid psychiatric or neurological disorder that is not associated with PWS
4.For controls, no known neurological, psychiatric, neurogenetic or serious medical condition
5.Currently receiving treatment with a GABA compound
6.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
7.If evidence of potential renal failure creatine levels will be measured and participants with a creatinine clearance < 30 mL/min will be excluded.
8.Known sulphite hypersensitivity
9.Pregnant or breastfeeding women
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Brain GABA levels will be assessed by E-I flux on magnetic resonance spectroscopy[at 10 days after commencing treatment]
- Secondary Outcome Measures
Name Time Method Re-established brain connectivity as measured by FMRI[at 10 days after commencing treatment]