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    Summary
    EudraCT Number:2009-012564-13
    Sponsor's Protocol Code Number:Cystadrops®/09/choc-study
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-06-19
    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 ConcernedFrance - ANSM
    A.2EudraCT number2009-012564-13
    A.3Full title of the trial
    Cysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study
    A.3.2Name or abbreviated title of the trial where available
    CYSTADROPS CHOC study
    A.4.1Sponsor's protocol code numberCystadrops®/09/choc-study
    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 SponsorOrphan Europe SARL
    B.1.3.4CountryFrance
    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 supportOrphan Europe SARL
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOrphan Europe SARL
    B.5.2Functional name of contact pointCéline Plisson
    B.5.3 Address:
    B.5.3.1Street Address70 avenue du Général de Gaulle
    B.5.3.2Town/ cityPuteaux
    B.5.3.3Post code92800
    B.5.3.4CountryFrance
    B.5.4Telephone number330147739463
    B.5.5Fax number330149001800
    B.5.6E-mailcplisson@orphan-europe.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/578
    D.3 Description of the IMP
    D.3.1Product nameCYSTADROPS 0.55% eye drops, solution
    D.3.4Pharmaceutical form Eye drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOcular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCYSTEAMINE HYDROCHLORIDE
    D.3.9.1CAS number 156-57-0
    D.3.9.3Other descriptive nameMercaptamine hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.55
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Nephropatic cystinosis patients with cystine corneal deposits
    E.1.1.1Medical condition in easily understood language
    Metabolic disease characterized by abnormal accumulation of the amino acid Cystine in various organs of the body, one of whom is the eyes
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level LLT
    E.1.2Classification code 10071112
    E.1.2Term Nephropathic cystinosis
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare efficacy of Cystadrops® versus 0.10% cysteamine hydrochloride eye drops solution in terms of superiority in patients with nephropathic cystinosis.
    E.2.2Secondary objectives of the trial
    To evaluate safety profile of Cystadrops® in patients with nephropathic cystinosis.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Signed and dated written inform consent form in accordance with local regulations: Having freely given their written informed consent to participate in the study. For patients aged less than 18 years consent will be obtained from the two parents (or legal representatives),
    - Diagnosis of cystinosis based on a previous white blood cells cystine concentration > 1.5 nmoles half-cystine per mg protein,
    - Presence of corneal crystal deposits attested during a slit-lamp examination within 3 months prior inclusion,
    - Ability to comply with their usual eye drops treatment in order to comply with the eyewash regimen of 4 instillations,
    - Agreement to move to Ophthalmic Core Laboratory for the assessment visits,
    - Likely to be able to participate in all scheduled evaluation and complete all required study procedures,
    - In the opinion of the investigator, the patient will be compliant and have a high probability of completing the study.
    E.4Principal exclusion criteria
    - Patients with uncontrolled hepatic disorder, cardiovascular disease, neurologic disease, or cancer,
    - Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if the investigator considers that they are not clinically significant for the conduct of study,
    - Patients with history or presence of alcohol abuse or drug addiction,
    - Pregnant or breast-feeding women,
    - Women of child-bearing potential without effective contraception (oral pill or IUCD),
    - Patients likely to be non-compliant to the study procedures or for whom a long-term follow-up seems to be difficult to achieve (In the younger children, the confocal microscopy may not be feasible and therefore will not be excluded from the study but only those patients able to undergo the IVCM procedure should be included in the primary analysis).
    E.5 End points
    E.5.1Primary end point(s)
    Corneal cystine crystal density measured by IVCM (In Vivo Confocal Microscopy), composite score assessed by independent masked reader.
    E.5.1.1Timepoint(s) of evaluation of this end point
    D1-D30-D90
    E.5.2Secondary end point(s)
    - photophobia (rated by investigator and patient)
    - CCCS (corneal cystine crystal score by slit-lamp)
    E.5.2.1Timepoint(s) of evaluation of this end point
    D1-D30-D90
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 No
    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
    blinded ophtalmologist who perform an independant and masked reading of the results
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Pharmacy hospital formulation
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA5
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months8
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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: 4
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 16
    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 No
    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
    paediatrics
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    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)
    At the end of study patient will be prescribed with usual previous treatment or any other available treatment by the investigator.
    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 Decision2013-01-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-11-29
    P. End of Trial
    P.End of Trial StatusCompleted
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