Phase 2
Completed N=59
Ganaxolone Treatment in Children With Fragile X Syndrome
Source: ClinicalTrials.gov NCT01725152 ↗Enrolled (actual)
59
Serious AEs
0.0%
Results posted
Apr 2023
Primary outcomePrimary: Clinical Global Impression-Improvement (CGI-I) Scale — 3.4; 3.5 Scores on a scale — p=0.4475
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.
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 | — |
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. Informational only — not medical advice.