Ganaxolone Treatment in Children With Fragile X Syndrome

NCT ID: NCT01725152

Last Updated: 2023-04-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 59 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-11-30
• Study Completion Date: 2016-11-01

Brief 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.

Detailed 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.

Conditions

Fragile x Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Ganaxolone then Placebo

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).

Group Type: EXPERIMENTAL

Ganaxolone

Intervention Type: DRUG

oral suspension, given in 3 divided doses

Placebo

Intervention Type: DRUG

oral suspension, given in 3 divided doses

Placebo then Ganaxolone

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 Type: EXPERIMENTAL

Ganaxolone

Intervention Type: DRUG

oral suspension, given in 3 divided doses

Placebo

Intervention Type: DRUG

oral suspension, given in 3 divided doses

Interventions

Ganaxolone

oral suspension, given in 3 divided doses

Intervention Type: DRUG

Placebo

oral suspension, given in 3 divided doses

Intervention Type: DRUG

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of California, Davis

OTHER

Sponsor Role: collaborator

U.S. Army Medical Research and Development Command

FED

Sponsor Role: collaborator

Marinus Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

M.I.N.D. Institute at University of California at Davis Medical Center

Sacramento, California, United States

Antwerp University Hospital

Edegem, , Belgium

Countries

United States; Belgium

References

Ligsay A, Van Dijck A, Nguyen DV, Lozano R, Chen Y, Bickel ES, Hessl D, Schneider A, Angkustsiri K, Tassone F, Ceulemans B, Kooy RF, Hagerman RJ. A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome. J Neurodev Disord. 2017 Aug 2;9(1):26. doi: 10.1186/s11689-017-9207-8.

Reference Type: DERIVED

PMID: 28764646 (View on PubMed)

Other Identifiers

1042-0800

Identifier Type: -

Identifier Source: org_study_id