Ganaxolone Treatment in Children With Fragile X Syndrome

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Marinus Pharmaceuticals
University of California, Davis
U.S. Army Medical Research and Materiel Command
Information provided by (Responsible Party):
Marinus Pharmaceuticals Identifier:
First received: November 7, 2012
Last updated: February 25, 2015
Last verified: January 2014

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). Up to 60 subjects (ages 6-17 yrs) will be randomized to receive either ganaxolone or placebo for 6 weeks and then cross over to the opposite treatment for another 6 weeks. The aim of the study is assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with FXS. The hypothesis is that ganaxolone treatment compared to placebo will improve anxiety and attention as measured by the several neuropsychological and psychometric tests.

Condition Intervention Phase
Fragile x Syndrome
Drug: Ganaxolone
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome

Resource links provided by NLM:

Further study details as provided by Marinus Pharmaceuticals:

Primary Outcome Measures:
  • Clinician's Global Impression-Improvement (CGI-I) [ Time Frame: Weeks 3, 6, 8, 11, 14 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Pediatric Anxiety Rating Scale (PARS) [ Time Frame: Baseline, Weeks 3, 6, 8, 11, 14 ] [ Designated as safety issue: No ]
  • Visual Analog Scale [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Aberrant Behavior Checklist [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV) [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • KiTAP- Test of Attentional Performance for Children [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Prepulse Inhibition (PPI) [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Social Gaze (eye tracking) [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]
  • Event-related brain potentials (ERP) [ Time Frame: Baseline, Weeks 6, 8, 14 ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: November 2012
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ganaxolone
3 mg/kg up to 12 mg/kg, with maximum of 1500 mg/day
Drug: Ganaxolone
oral suspension, given in 3 divided doses
Other Names:
  • GNX
  • GNX OS
Placebo Comparator: Placebo
non active
Drug: Placebo
oral suspension, given in 3 divided doses
Other Name: PBO

Detailed Description:

This is a single center study at UC Davis MIND Institute. Children with fragile x syndrome between the ages of 6-17yrs, 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. The primary aim of the study is to assess efficacy of ganaxolone treatment compared to placebo on clinical behaviors such as anxiety and attention as measured by Clinician's Global Impression-Improvement (CGI-I). The key secondary efficacy measure is the Pediatric Anxiety Scale (PARS). Other secondary efficacy measures include the visual analog scale (VAS), Anxiety, Depression, Attention, and Mood Scale (ADAMS), Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV), and Aberrant Behavior Checklist- Community Edition (ABC-C). Tolerability and safety will be monitored by routine vital signs, physical/neurological exams, ECGs, clinical laboratory and adverse event assessments.


Ages Eligible for Study:   6 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01725152

Contact: Julia Tsai, PhD 203-315-5820
Contact: Gail Farfel, PhD 201-485-5101

United States, California
M.I.N.D. Institute at University of California at Davis Medical Center Recruiting
Sacramento, California, United States, 95817
Contact: Erika Bickel, BS    916-703-0281   
Contact: Randi J Hagerman, MD    (916) 703-0247   
Antwerp University Hospital Recruiting
Edegem, Belgium, 2650
Contact: Anke Van Dijck, MD    +32 3 275 97 56      
Sponsors and Collaborators
Marinus Pharmaceuticals
University of California, Davis
U.S. Army Medical Research and Materiel Command
Principal Investigator: Randi J Hagerman, MD M.I.N.D. Institute at University of California at Davis Medical Center
Principal Investigator: Berten Ceulemans, M.D.; Ph. D. University Hospital, Antwerp
  More Information

No publications provided

Responsible Party: Marinus Pharmaceuticals Identifier: NCT01725152     History of Changes
Other Study ID Numbers: 1042-0800
Study First Received: November 7, 2012
Last Updated: February 25, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Marinus Pharmaceuticals:
fragile x syndrome

Additional relevant MeSH terms:
Fragile X Syndrome
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Intellectual Disability
Mental Retardation, X-Linked
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Pathologic Processes
Sex Chromosome Disorders processed this record on August 27, 2015