Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 12:37 AM
Ignite Modification Date:
2025-12-25 @ 12:37 AM
NCT ID:
NCT02019667
Eligibility Criteria:
* INCLUSION CRITERIA
* Aged 4 years or older
* 4-hydroxybutyric aciduria (gamma-hydroxybutyric aciduria) on two separate tests
* Documented succinic semialdehyde dehydrogenase enzyme deficiency
* Patients must have clinical features consistent with SSADH deficiency including developmental delay especially deficit in expressive language, hypotonia, ataxia, seizures, and other neuropsychiatric symptoms including sleep disturbances , attention deficit, anxiety, obsessivecompulsive disorder, and autistic traits
* During the study, women of child-bearing potential must use a reliable method of birth control until one month after the final drug taper is complete.
EXCLUSION CRITERIA
* Current alcohol use (\>14 drinks/wk in men and \>7 drinks/wk in women or or recreational drug use
* Contraindications to MRI: metal in the body including pacemakers, medication pumps, aneurysm clips, metallic prostheses (including metal pins and rods, heart valves or cochlear implants), shrapnel fragments, permanent eye liner or small metal fragments in the eye that welders and other metal workers may have
* Claustrophobia
* Cannot lie comfortably flat on the back for up to 2h in the MRI scanner
* Patients with a history of other major medical disorders with clinical fluctuations, or requiring therapy that might affect study participation or drug response such as severe depression or psychoses, renal or hepatic disease.
* Patients requiring treatment with drugs known to affect the GABAergic system, including vigabatrin and benzodiazepines.
* Pregnant and lactating women
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
4 Years
Study:
NCT02019667
Study Brief:
Protocol Section:
NCT02019667