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    Summary
    EudraCT Number:2014-000251-89
    Sponsor's Protocol Code Number:1042-0800
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-08-18
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2014-000251-89
    A.3Full title of the trial
    A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study with an investigational drug called ganaxolone in children with fragile X syndrome
    A.3.2Name or abbreviated title of the trial where available
    GNX study
    A.4.1Sponsor's protocol code number1042-0800
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01725152
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAntwerp University Hospital
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMarinus Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportUniversity of California Davis
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportJérôme Lejeune foundation
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportMarguerite Marie Delacroix foundation
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportFRAXA Research Foundation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAntwerp University Hospital
    B.5.2Functional name of contact pointCognitive Genetics Group
    B.5.3 Address:
    B.5.3.1Street AddressPrins Boudewijnlaan 43
    B.5.3.2Town/ cityEdegem
    B.5.3.3Post code2650
    B.5.3.4CountryBelgium
    B.5.4Telephone number+3232759756
    B.5.5Fax number+3232759723
    B.5.6E-mailmedische.genetica@uza.be
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameGanaxolone
    D.3.2Product code CCD 1042, MD 9150000, Mepalon 1042, SPT316
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNGANAXOLONE
    D.3.9.1CAS number 38398-32-2
    D.3.9.2Current sponsor codeCCD 1042
    D.3.9.4EV Substance CodeSUB07880MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    behavioral problems in children with fragile X syndrome
    E.1.1.1Medical condition in easily understood language
    behavioral problems in children ages 6-17 years with fragile X syndrome
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10017324
    E.1.2Term Fragile X syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with fragile X syndrome.
    E.2.2Secondary objectives of the trial
    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)].
    E.2.3Trial contains a sub-study No
    E.3Principal 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 β-human chorionic growth hormone (β-HCG) or urine pregnancy test collected at the initial screening visit.
    E.4Principal 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.
    E.5 End points
    E.5.1Primary end point(s)
    Clinical Global Impression Improvement scale (CGI-I) will be the primary outcome measure.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 3, Week 6, Week 8 , Week 11, Week 14
    E.5.2Secondary 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).
    E.5.2.1Timepoint(s) of evaluation of this end point
    - CGI-S: Week 1
    - ABC: Week 1, Week 6, Week 8, Week 14
    - PARS: Week 1, Week 3, Week 6, Week 8 , Week 11, Week 14
    - VAS: Week 1, Week 6, Week 8, Week 14
    - ADAMS: Week 1, Week 6, Week 8, Week 14
    - SNAP-IV: Week 1, Week 6, Week 8, Week 14
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 60
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Fragile X syndrome is a genetic condition that causes intellectual disability.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who have completed participation or have discontinued early from the trial will continue care by their primary care physician and/or regular neurologist.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-10-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-15
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