Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency

Completed · Phase 2 · Has a placebo group

Conditions studied: Metabolic Disease, Seizures

In brief

Objective: To perform a clinical trial assessing the safety, tolerability and efficacy of the GABA(B) receptor antagonist SGS-742 in patients with SSADH deficiency. Study Population: Twenty-two children and adults with SSADH deficiency. Design: Double-blind, cross-over, phase II clinical trial. Outcome Measures: The primary outcome measures for drug efficacy will be performance on neuropsychological testing and responses to parent questionnaire. The secondary outcome measure will be TMS parameters of cortical excitation and inhibition. The outcome measures for safety will include clinical examination and neuropsychological tests.

Key facts

Study ID

NCT02019667

Run by

National Institute of Neurological Disorders and Stroke (NINDS)

People needed

19

Starts

2014-03-31

Expected to finish

2019-04-03

Last updated by the study team

2020-02-24

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• 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

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
• Washington State University — Pullman, Washington, United States

Full record on ClinicalTrials.gov

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