Ganaxolone Treatment in Children With Fragile X Syndrome
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Purpose
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 |
- Clinician's Global Impression-Improvement (CGI-I) [ Time Frame: Weeks 3, 6, 8, 11, 14 ] [ Designated as safety issue: No ]
- 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 ]
- 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:
|
| Placebo Comparator: Placebo |
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.
Eligibility| 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| Contact: Julia Tsai, PhD | 203-315-5820 | jtsai@marinuspharma.com |
| Contact: Gail Farfel, PhD | 201-485-5101 | gfarfel@marinuspharma.com |
| 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 erika.bickel@ucdmc.ucdavis.edu | |
| Contact: Randi J Hagerman, MD (916) 703-0247 randi.hagerman@ucdmc.ucdavis.edu | |
| Principal Investigator: | Randi J Hagerman, MD | M.I.N.D. Institute at University of California at Davis Medical Center |
More Information
No publications provided
| Responsible Party: | Marinus Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT01725152 History of Changes |
| Other Study ID Numbers: | 1042-0800 |
| Study First Received: | November 7, 2012 |
| Last Updated: | November 8, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Marinus Pharmaceuticals:
|
fragile x syndrome ganaxolone anxiety attention pediatric |
Additional relevant MeSH terms:
|
Fragile X Syndrome Mental Retardation, X-Linked Genetic Diseases, X-Linked Mental Retardation Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases Sex Chromosome Disorders Chromosome Disorders Congenital Abnormalities |
Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Pregnanolone Anesthetics Central Nervous System Depressants Physiological Effects of Drugs Pharmacologic Actions Central Nervous System Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on May 16, 2013