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    Summary
    EudraCT Number:2011-004156-19
    Sponsor's Protocol Code Number:VX11-765-402
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-03-20
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2011-004156-19
    A.3Full title of the trial
    A Phase 2b, Randomized, Double Blind, Placebo Controlled, Parallel Group, Dose Ranging Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment Resistant Partial Epilepsy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial Epilepsy
    A.4.1Sponsor's protocol code numberVX11-765-402
    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 SponsorVertex Pharmaceuticals Incorporated
    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 supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVertex Pharmaceuticals Incorporated
    B.5.2Functional name of contact pointMedical Information Center
    B.5.3 Address:
    B.5.3.1Street Address130 Waverly Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16176348789
    B.5.6E-mailmedicalinfo@vrtx.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.3.2Product code VX-765
    D.3.4Pharmaceutical form Tablet
    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.3.9.1CAS number 273404-37-8
    D.3.9.2Current sponsor codeVX-765
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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 placeboTablet
    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
    Partial epilepsy
    E.1.1.1Medical condition in easily understood language
    Epilepsy
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10065336
    E.1.2Term Partial epilepsy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    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 the efficacy, safety, and tolerability of VX-765 to treat seizures in subjects with treatment resistant partial epilepsy
    E.2.2Secondary objectives of the trial
    To evaluate the pharmacokinetics (PK) of VX-765, VRT 043198 (active metabolite of VX-765), and concomitant antiepileptic drug (AED) levels in subjects with treatment resistant partial epilepsy
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Males or females aged 18 to 64 (inclusive) years with a BMI between 18 and 35 (kg/m2)
    • Subjects must agree to use acceptable contraceptive methods as described in Section 12.7.5. of the study protocol
    • Must have a diagnosis and hx of treatment-resistant partial-onset epilepsy (as defined in protocol) and are taking 1 to 4 stable doses of concomitant AEDs.
    • Subjects must have had at least 1 EEG consistent with partial epilepsy
    • Must have had at least 6 partial-onset seizures and a seizure free period of no more than 3 weeks during Baseline
    • Subjects with stable medical conditions as determiend by Principal investigator
    • Must understand and comply with the protocol requirements and be willing to provide written informed consent to participate
    E.4Principal exclusion criteria
    • Subjects who are pregnant or lactating or do not agree to use medically approved methods of contraception as outlined in the protocol
    • Subjects who are male and have a female partner who is pregnant, nursing, or is planning to become pregnant during the study period, or within 90 days of the last dose of study drug
    • Subjects with a history of nonepileptic, transient alterations in consciousness (e.g., metabolic, structural, and post traumatic or pseudo seizures)
    • Those who have a history of status epilepticus in the past 12 months
    • Subjects whose seizure frequency cannot be quantified
    • Subjects who have a significant medical illness including kidney, liver, pulmonary, or gastrointestinal disease; or unstable or poorly controlled conditions such as hypertension, diabetes, or angina pectoris, as judged by the investigator
    • Subjects with clinically significant psychiatric illness or had an active suicidal plan/intent, thoughts, or attempt as defined in the study protocol
    • Subjects with clinically significant laboratory abnormalities during the Screening Visit/Baseline Period, as judged by the investigator
    • Subjects with active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV)
    • Subjects with positive drug screen at screening or during the Baseline Period (excluding any allowed prescribed medications) and/or a history of alcoholism or drug addiction within past 2 years
    • Subjects on felbamate with fewer than 18 month of continuous felbamate exposure at the time of the Screening Visit or with significant adverse reactions to felbamate
    • Subjects treated with vigabatrin fewer than 2 years prior to the Screening Visit or who have a prior history of treatment with vigabatrin without a documented stable examination by an ophthalmologist or neuroophthalmologist of the visual field within the 2 years prior to the Screening Visit
    • Subjects using prohibited medications (Section 10.3 and Table 10 1) or treated with any systemic immunosuppressant as defined in the protocol
    • Subjects who experienced a symptomatic viral, fungal, or bacterial infection requiring systemic treatment within 7 days prior to the first dose of study drug
    • Subjects with a current or prior history of illness precluding them from immunomodulatory therapy
    • Subjects who have donated any blood or have had a significant loss of blood (500 mL) as defined in the protocol
    • Subjects who participated in any other clinical studies involving an investigational product or device and have received the last dose of the study drug associated with that clinical study within 30 days or 5 half lives (whichever is longer) of the Screening Visit
    • Subjects who have participated in earlier VX-765 clinical studies and received at least one dose of study drug
    • Any subject judged by the investigator or sponsor (or designee) to be inappropriate for the study
    E.5 End points
    E.5.1Primary end point(s)
    • Percent reduction in weekly seizure frequency during the Late Treatment Period compared to the Baseline Period
    • Percent of subjects with 50% or greater reduction in weekly seizure frequency (responder rate) during the Late Treatment Period compared to the Baseline Period
    • Safety and tolerability as assessed by vital signs, standard 12 lead electrocardiograms (ECGs), laboratory assessments (serum chemistry, hematology, and urinalysis), and adverse events
    E.5.1.1Timepoint(s) of evaluation of this end point
    Multiple timepoints up to Study Week 25
    E.5.2Secondary end point(s)
    • Percent of subjects who are seizure free during the Late Treatment Period
    • Percent reduction in seizure frequency during the entire Treatment Period compared to the Baseline Period
    • Percent of subjects with 50% or greater reduction in seizure frequency (responder rate) during the entire Treatment Period compared to the Baseline Period
    • Percent of subjects who are seizure-free during the entire Treatment Period
    • Maximum number of consecutive days that subjects do not have seizures at any time during the Late Treatment Period
    • Maximum number of consecutive days that subjects do not have seizures at any time during the entire Treatment Period
    • PK of VX 765, VRT 043198, and concomitant AED levels in blood
    E.5.2.1Timepoint(s) of evaluation of this end point
    Multiple timepoints up to Study Week 25
    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 Yes
    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 No
    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 trial5
    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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA74
    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
    Austria
    Belgium
    Czech Republic
    Finland
    France
    Germany
    Hungary
    Italy
    Netherlands
    Poland
    Ukraine
    United Kingdom
    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
    Last visit of the last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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 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: 500
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state44
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 500
    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 may have the opportunity to participate in an open label extension study following completion in Study VX11-765-402
    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 Decision2012-06-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-09-24
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