Ganaxolone Treatment in Children With Fragile X Syndrome

Completed · Phase 2 · Has a placebo group

Conditions studied: Fragile x Syndrome

In brief

This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS). The objective of the study is to assess the safety, tolerability and efficacy of ganaxolone in the treatment of anxiety and attention in subjects with FXS.

Key facts

Study ID

NCT01725152

Run by

Marinus Pharmaceuticals

People needed

59

Starts

2012-11-01

Expected to finish

2016-11-01

Last updated by the study team

2023-04-10

Who can join

Age: 6 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• molecular documentation of FMR1 full mutation
• ages 6-17 yrs, inclusive
• sexually active subjects are required to use a medically acceptable form of birth control

You may not qualify if…

• non-English or Spanish speaking subjects
• concomitant systemic steroid, vigabatrin, felbamate and ketoconazole
• changes in medications within last 2 months
• clinically unstable medical disease, progressive CNS disease/disorder
• history of recurrent status epilepticus
• unwilling to withhold grapefruit or grapefruit juice for the duration of the study
• actively suicidal

Where it is running

• M.I.N.D. Institute at University of California at Davis Medical Center — Sacramento, California, United States
• Antwerp University Hospital — Edegem, Belgium

Full record on ClinicalTrials.gov

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