A clinical study with an investigational drug called ganaxolone in children with fragile X syndrome

Conditions: behavioral problems in children with fragile X syndrome
MedDRA version: 17.0Level: PTClassification code 10017324Term: Fragile X syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2014-000251-89-BE

Lead Sponsor: Antwerp University Hospital

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

1.Molecular documentation of the FMR1 full mutation.
2.Ages 6 through 17 years, inclusive.
3.Willingness of parent or caretaker to participate in the protocol.
4.Sexually active women of child bearing potential must be using a medically acceptable method of birth control and have a negative qualitative serum ß-human chorionic growth hormone (ß-HCG) or urine pregnancy test collected at the initial screening visit.
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Persons who do not speak English or Dutch.
2.Concomitant medications including systemic steroids, Vigabatrin, felbamate, and ketoconazole.
3.Changes in any medications within the last two months.
4.Aspartate aminotransferase (AST), alanine aminotransferase (ALT) or total bilirubin level abnormalities.
5.History of recurrent status epilepticus.
6.Have been exposed to any other investigational drug within 30 days or less than 6 half-lives prior to randomization.
7.Subjects who are actively suicidal or have an active and compromising (CNS) disease.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with fragile X syndrome.;Secondary Objective: To evaluate clinical changes in behavior as reflected on the Pediatric Anxiety Rating Scale (PARS), Anxiety, Depression and Mood Scale (ADAMS), and visual analogue scale (VAS), changes in attention as measured by the Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV), and changes in hyperactivity/impulsivity [SNAP-IV and Aberrant Behavior Checklist- Community Edition (ABC-C)].;Primary end point(s): Clinical Global Impression Improvement scale (CGI-I) will be the primary outcome measure.;Timepoint(s) of evaluation of this end point: Week 3,Week 6,Week 8 , Week 11, Week 14

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): We will utilize a variety of other well validated assessment instruments to assess the secondary end points: the Aberrant Behavior Checklist (ABC), the Pediatric Anxiety Rating Scale (PARS), Visual Analogue Scale (VAS), Anxiety Depression and Mood Scale (ADAMS) and the Swanson, Nolan, and Pelham-IV Questionnaire (SNAP IV).;Timepoint(s) of evaluation of this end point: - CGI-S: Week 1<br>- ABC: Week 1, Week 6, Week 8, Week 14<br>- PARS: Week 1, Week 3,Week 6,Week 8 , Week 11, Week 14<br>- VAS: Week 1, Week 6, Week 8, Week 14<br>- ADAMS: Week 1, Week 6, Week 8, Week 14<br>- SNAP-IV: Week 1, Week 6, Week 8, Week 14