Overview

This trial is active, not recruiting.

Condition fragile x syndrome
Treatments ganaxolone, placebo
Phase phase 2
Sponsor Marinus Pharmaceuticals
Collaborator University of California, Davis
Start date November 2012
End date June 2016
Trial size 60 participants
Trial identifier NCT01725152, 1042-0800

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
3 mg/kg up to 12 mg/kg, with maximum of 1500 mg/day
ganaxolone GNX
oral suspension, given in 3 divided doses
(Placebo Comparator)
non active
placebo PBO
oral suspension, given in 3 divided doses

Primary Outcomes

Measure
Clinician's Global Impression-Improvement (CGI-I)
time frame: Weeks 3, 6, 8, 11, 14

Secondary Outcomes

Measure
Pediatric Anxiety Rating Scale (PARS)
time frame: Baseline, Weeks 3, 6, 8, 11, 14
Visual Analog Scale
time frame: Baseline, Weeks 6, 8, 14
Anxiety, Depression, and Mood Scale (ADAMS)
time frame: Baseline, Weeks 6, 8, 14
Aberrant Behavior Checklist
time frame: Baseline, Weeks 6, 8, 14
Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
time frame: Baseline, Weeks 6, 8, 14

Eligibility Criteria

Male or female participants from 6 years up to 17 years old.

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

Additional Information

Official title A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome
Principal investigator Randi J Hagerman, MD
Description This is a single center study at UC Davis MIND Institute. Children with fragile x syndrome between the ages of 6-17yrs, 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. The primary aim of the study is to assess efficacy of ganaxolone treatment compared to placebo on clinical behaviors such as anxiety and attention as measured by Clinician's Global Impression-Improvement (CGI-I). The key secondary efficacy measure is the Pediatric Anxiety Scale (PARS). Other secondary efficacy measures include the visual analog scale (VAS), Anxiety, Depression, Attention, and Mood Scale (ADAMS), Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV), and Aberrant Behavior Checklist- Community Edition (ABC-C). Tolerability and safety will be monitored by routine vital signs, physical/neurological exams, ECGs, clinical laboratory and adverse event assessments.
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by Marinus Pharmaceuticals.