Trial record 8 of 44 for:    "Fragile X syndrome"

Ganaxolone Treatment in Children With Fragile X Syndrome

This study is currently recruiting participants.
Verified January 2014 by Marinus Pharmaceuticals
Sponsor:
Collaborators:
University of California, Davis
U.S. Army Medical Research and Material Command
Information provided by (Responsible Party):
Marinus Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01725152
First received: November 7, 2012
Last updated: January 17, 2014
Last verified: January 2014
  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

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.

  Eligibility

Ages Eligible for Study:   6 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01725152

Contacts
Contact: Julia Tsai, PhD 203-315-5820 jtsai@marinuspharma.com
Contact: Gail Farfel, PhD 201-485-5101 gfarfel@marinuspharma.com

Locations
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   
Sponsors and Collaborators
Marinus Pharmaceuticals
University of California, Davis
U.S. Army Medical Research and Material Command
Investigators
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: January 17, 2014
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
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
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 April 16, 2014