NCT01725152

Ganaxolone Treatment in Children With Fragile X Syndrome

A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Marinus Pharmaceuticals · Industry
• Sex: All
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

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). 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.

Detailed description

This is a single center study conducted at University of California Davis (UCD) MIND Institute. Children with FXS between the ages of 6-17 years, inclusive will be randomized at a 1:1 ratio to receive ganaxolone or placebo treatment for 6 weeks, discontinue treatment and washout for 2 weeks, and then cross over to the opposite treatment for another 6 weeks.

Conditions

• Fragile x Syndrome

Interventions

Type	Name	Description
DRUG	Ganaxolone	oral suspension, given in 3 divided doses
DRUG	Placebo	oral suspension, given in 3 divided doses

Timeline

• Start date: 2012-11-01
• Primary completion: 2015-10-16
• Completion: 2016-11-01
• First posted: 2012-11-12
• Last updated: 2023-04-10
• Results posted: 2023-04-10

Locations

2 sites across 2 countries: United States, Belgium