Phase 2
N=59
Ganaxolone Treatment in Children With Fragile X Syndrome
Fragile x Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01725152 ↗Enrolled (actual)
59
Serious AEs
0.0%
Results posted
Apr 2023
Primary outcome: Primary: Clinical Global Impression-Improvement (CGI-I) Scale — 3.4; 3.5 Scores on a scale — p=0.4475
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ganaxolone (Drug); Placebo (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Marinus Pharmaceuticals
- Primary completion
- Oct 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Global Impression-Improvement (CGI-I) Scale |
3.4; 3.5 | 0.4475 |
| SECONDARY Pediatric Anxiety Rating Scale (PARS) Total Score |
8.3; 9.2 | — |
| SECONDARY Visual Analogue Scale (VAS) |
5.6; 5.0; 4.4; 3.9; 4.4; 3.9 | — |
| SECONDARY Anxiety, Depression, and Mood Scale (ADAMS) |
7.4; 7.8; 3.5; 2.6; 5.8; 6.3 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) |
15.7; 16.1; 5.6; 5.7; 5.7; 6.4 | — |
| SECONDARY Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV) |
15.5; 14.6; 13.9; 12.6; 29.3; 27.1 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT01725152). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.