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    Summary
    EudraCT Number:2014-004363-21
    Sponsor's Protocol Code Number:1042-0603
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-01-07
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2014-004363-21
    A.3Full title of the trial
    A Multicenter, Double Blind, Randomized, Placebo-Controlled Trial to
    Determine the Efficacy and Safety of Ganaxolone as Adjunctive Therapy for Adults with Drug-Resistant Partial-Onset Seizures Followed by Long-term Open-Label Treatment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Trial to evaluate efficacy and safety of ganaxolone compared to placebo as
    add-on therapy in adults with Drug-Resistant partial-onset seizures (POS).
    A.4.1Sponsor's protocol code number1042-0603
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01963208
    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 SponsorMarinus Pharmaceuticals, Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMarinus Pharmaceuticals, Inc
    B.5.2Functional name of contact pointdepartment for clinical trials
    B.5.3 Address:
    B.5.3.1Street Address142 Temple St. Suite 205
    B.5.3.2Town/ cityNew Haven, CT
    B.5.3.3Post code06510
    B.5.3.4CountryUnited States
    B.5.4Telephone number001203315-5820
    B.5.5Fax number001203315-0565
    B.5.6E-mailjtsai@marinuspharma.com
    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
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    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 INNCCD-1042
    D.3.9.1CAS number 38398-32-2
    D.3.9.2Current sponsor codeCCD 1042
    D.3.9.3Other descriptive nameGanaxolone, SPT3162, MD 9150000, CCD-1042, Mepalon, 1042,; (3α-hydroxy-3β-methyl-5α-pregnan-20-one)
    D.3.9.4EV Substance CodeSUB07880MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number225
    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 placeboCapsule
    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
    Drug-Resistant Epilepsy with Partial-Onset Seizures
    E.1.1.1Medical condition in easily understood language
    Epilepsy with Seizures which are partial and are Resistant to therapy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10065336
    E.1.2Term Partial epilepsy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate efficacy of ganaxolone compared to placebo as
    adjunctive therapy in adults with partial-onset seizures (POS), with or without secondary generalizations.
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of ganaxolone compared to placebo as adjunctive therapy in adults with POS.
    • To evaluate serum levels of ganaxolone at 1200 mg/d and 1800 mg/d after chronic dosing
    • To evaluate the safety and tolerability of ganaxolone when administered as adjunctive therapy in adults with POS over a 12-month period.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male and female subjects 18 years of age and older able to provide written informed consent with a confident diagnosis of epilepsy with POS with or without secondary generalization with a POS frequency rate of ≥3 POS per 28-day who are currently treated on a stable regimen of AEDs.
    E.4Principal exclusion criteria
    Subjects must not have previous exposure to ganaxolone, generalized epilepsy, have less than 3 POS seizures per 28-day period or ≥21 seizure-free days. Subject should not have innumerable seizures, more than 100 POS total per each 4-week baseline period. No subjects will be admitted who have an active central nervous system (CNS) infection, demyelinating disease, degenerative neurological disease, CNS disease deemed progressive, clinically unstable psychiatric disorder, suicide attempt within the last 5 years, or current significant suicidal ideation.
    E.5 End points
    E.5.1Primary end point(s)
    Significant reduced in frequency of partial onset
    seizures, with or without secondary generalizations, in patients treated with Ganaxolone compared to placebo
    E.5.1.1Timepoint(s) of evaluation of this end point
    At end of 14 weeks of double blind treatment
    E.5.2Secondary end point(s)
    • To evaluate the safety and tolerability of ganaxolone compared to placebo as adjunctive therapy in adults with POS.
    • To evaluate serum levels of ganaxolone at 1200 mg/d and 1800 mg/d after chronic dosing
    • To evaluate the safety and tolerability of ganaxolone when administered as adjunctive therapy in adults with POS over a 12-month period.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At every patient visit and at end of 68 weeks of open label treatment
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    the study has two phases: 14 weeks double blind followed by 68 open label phase
    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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Australia
    Bulgaria
    Germany
    Poland
    Russian Federation
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    the last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 300
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 42
    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 No
    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
    This study may enrol subjects who do not have the legal capacity to provide informed consent. The person providing consent for the subject may be the legally authorized representative (LAR).
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state35
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 62
    F.4.2.2In the whole clinical trial 342
    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)
    Treatment or care after the subject has ended the participation in the trial is the expected normal treatment of that condition
    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 Decision2015-02-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-03-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2016-11-30
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