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Neurosteroid

Ganaxolone for Fragile X Syndrome

Phase 2
Waitlist Available
Led By Berten Ceulemans, M.D.; Ph. D.
Research Sponsored by Marinus Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, weeks 6, 8, 14
Awards & highlights

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.

Eligible Conditions
  • Fragile X Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, weeks 6, 8, 14
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 6, 8, 14 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Clinical Global Impression-Improvement (CGI-I) Scale
Secondary study objectives
Aberrant Behavior Checklist (ABC)
Anxiety, Depression, and Mood Scale (ADAMS)
Pediatric Anxiety Rating Scale (PARS) Total Score
+2 more
Other study objectives
Event-related brain potentials (ERP)
KiTAP- Test of Attentional Performance for Children
Prepulse Inhibition (PPI)
+1 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Placebo then GanaxoloneExperimental Treatment2 Interventions
Participants first received placebo. They were first titrated up to the 12 mg/kg tid during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received ganaxolone for a duration of 6 weeks (from week 8 to 14).
Group II: Ganaxolone then PlaceboExperimental Treatment2 Interventions
Participants first received ganaxolone. They were first titrated up to the 12 milligram per kilogram (mg/kg) three times daily (tid) during a 2-week titration phase, then maintained on that dose for an additional 4 weeks. After a washout period of 2 weeks, participants received placebo for a duration of 6 weeks (from week 8 to 14).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxolone
FDA approved
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,714 Total Patients Enrolled
University of California, DavisOTHER
932 Previous Clinical Trials
4,721,284 Total Patients Enrolled
5 Trials studying Fragile X Syndrome
390 Patients Enrolled for Fragile X Syndrome
U.S. Army Medical Research and Development CommandFED
289 Previous Clinical Trials
246,012 Total Patients Enrolled
~5 spots leftby Sep 2025
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