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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

All Individual Drugs Already Approved

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 6, 8, 14

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

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

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

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