Clinical trial NCT01725152: Ganaxolone Treatment in Children With Fragile X Syndrome
Official title: A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile x Syndrome
Keywords: fragile x syndrome
ganaxolone
anxiety
attention
pediatric
Lead sponsor: Marinus Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: University of California, Davis
name: U.S. Army Medical Research and Development Command
First posted: 2012-11-12
Enrollment: 59
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.

Eligibility 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

Primary outcomes:
measure: Clinical Global Impression-Improvement (CGI-I) Scale
timeFrame: Week 14 (End of Treatment)
Secondary outcomes:
measure: Pediatric Anxiety Rating Scale (PARS) Total Score
timeFrame: Week 14 (End of Treatment)
measure: Visual Analogue Scale (VAS)
timeFrame: Week 14 (End of Treatment)
measure: Anxiety, Depression, and Mood Scale (ADAMS)
timeFrame: Week 14 (End of Treatment)
measure: Aberrant Behavior Checklist (ABC)
timeFrame: Week 14 (End of Treatment)
measure: Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
timeFrame: Week 14 (End of Treatment)
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: M.I.N.D. Institute at University of California at Davis Medical Center
state: California
city: Edegem
country: Belgium
facility: Antwerp University Hospital
Study officials:
References:
citation: Ligsay A, Van Dijck A, Nguyen DV, Lozano R, Chen Y, Bickel ES, Hessl D, Schneider A, Angkustsiri K, Tassone F, Ceulemans B, Kooy RF, Hagerman RJ. A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome. J Neurodev Disord. 2017 Aug 2;9(1):26. doi: 10.1186/s11689-017-9207-8.