Ganaxolone Treatment in Children With Fragile X Syndrome

• Status: Completed
• Trial end date: 2016-11-01
• Target enrollment: 0
• Participant gender: All

Summary

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). Up to 60 subjects (ages 6-17 yrs) will be randomized to receive either ganaxolone or placebo for 6 weeks and then cross over to the opposite treatment for another 6 weeks. The aim of the study is assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with FXS. The hypothesis is that ganaxolone treatment compared to placebo will improve anxiety and attention as measured by the several neuropsychological and psychometric tests.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Marinus Pharmaceuticals
• Collaborators: U.S. Army Medical Research and Development Command; U.S. Army Medical Research and Materiel Command; University of California, Davis
• Treatments: Ganaxolone; Pregnanolone

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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