Ganaxolone Treatment in Children With Fragile X Syndrome

Marinus Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Fragile x Syndrome

Treatments: Drug: Placebo; Drug: Ganaxolone

Study type: Interventional

Funder types: Other; Industry; Other U.S. Federal agency

Identifiers

NCT01725152

1042-0800

Details and patient eligibility

About

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.

Full description

This is a single center study conducted at University of California Davis (UCD) MIND Institute. Children with FXS between the ages of 6-17 years, 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.

Enrollment: 59 patients

Sex: All

Ages: 6 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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59 participants in 2 patient groups

Ganaxolone then Placebo

Experimental group

Description:

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:

Drug: Placebo

Drug: Ganaxolone

Placebo then Ganaxolone

Experimental group

Description:

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).

Treatment:

Drug: Placebo

Drug: Ganaxolone