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    Summary
    EudraCT Number:2013-003228-35
    Sponsor's Protocol Code Number:18072013
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-11-12
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-003228-35
    A.3Full title of the trial
    Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients
    Estudio de la eficacia preliminar y seguridad de la cisteamina tópica formulada en solución viscosa en pacientes con cistinosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the preliminary efficacy and safety of topical cysteamine formulated in viscous solution in cystinosis patients
    Estudio de la eficacia preliminar y seguridad de la cisteamina tópica formulada en solución viscosa en pacientes con cistinosis
    A.3.2Name or abbreviated title of the trial where available
    Viscou solution cysteamina
    Cisteamina en solución viscosa
    A.4.1Sponsor's protocol code number18072013
    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 SponsorFundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
    B.1.3.4CountrySpain
    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 supportVHIR
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDra. Nieves Martin
    B.5.2Functional name of contact pointStudy coordinator
    B.5.3 Address:
    B.5.3.1Street AddressPasseig Vall d'Hebron, 119-129
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08035
    B.5.3.4CountrySpain
    B.5.4Telephone number+34934893166
    B.5.5Fax number+34934894180
    B.5.6E-mailnmartin@vhebron.net
    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 namecysteamine (mercaptamine) viscous solution
    D.3.4Pharmaceutical form Eye drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOphthalmic use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMercaptamine
    D.3.9.1CAS number 156-57-0
    D.3.9.3Other descriptive nameMERCAPTAMINE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB25229
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    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
    Cystinosis
    Cistinosis
    E.1.1.1Medical condition in easily understood language
    Cystinosis
    Cistinosis
    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 16.1
    E.1.2Level PT
    E.1.2Classification code 10011777
    E.1.2Term Cystinosis
    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
    The main objective of the
    study is to assess the efficacy of 0.55% cysteamine formulated in viscous solution compared with saline solution at the same dose, in corneal crystal deposition.
    El objetivo principal del estudio es valorar la eficacia de la cisteamina 0,55% formulada en solución viscosa comparada con la formulación en solución salina, con la misma posología, en el depósito de cristales corneales.
    E.2.2Secondary objectives of the trial
    -The secondary objective of the study is to assess whether the efficacy of 0.55% cysteamine formulated viscous solution administered four times a day is comparable to more frequent administration.
    -To evaluate the tolerance and compliance with the new formulation.
    -El objetivo secundario del estudio es valorar si la eficacia de la cisteamina 0,55% formulada en solución viscosa administrada cuatro veces al día es comparable a su administración más frecuente.
    -Valorar la tolerancia y el cumplimiento de la nueva formulación.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet each of the following criteria to be eligible for admission to the study:
    -Cystinosis patients treated with cysteamine 0.55% eye drops (saline solution), made ??by the Pharmacy Department of Hospital Universitari Vall d'Hebron.
    -Age ? 5 years
    -Cooperative and compliant patient
    -Parents who sign the consent or acquiescence in patients over 12 years old.
    Los pacientes deben cumplir cada uno de los siguientes criterios para ser elegibles para su admisión en el estudio:
    -Pacientes afectos de cistinosis en tratamiento con colirio de cisteamina al 0,55%, fórmula magistral en colirio, formulada por el Servicio de Farmacia del Hospital Universitari Vall d?Hebron.
    -Edad ?5 años
    -Paciente colaborador y cumplidor
    -Padres que firmen el consentimiento o asentimiento en pacientes de más de 12 años de edad.
    E.4Principal exclusion criteria
    -Using other eye topical treatments.
    -Withdrawal of consent or acquiescence.
    -Utilización concomitante de otros tratamientos tópicos oculares.
    -Retirada del consentimiento o asentimiento.
    E.5 End points
    E.5.1Primary end point(s)
    Number of corneal cystine crystals
    Número de cristales de cistina corneales
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months
    3 meses
    E.5.2Secondary end point(s)
    Number of corneal cystine crystals (4 times a day)
    Number of prescription instillations/ number of instillations performed
    -Número de cristales de cistina en córnea (4 veces al día)
    -Número de instilaciones pautadas/número de instilaciones reales
    E.5.2.1Timepoint(s) of evaluation of this end point
    3 months
    3 meses
    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 No
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Se comparan los resultados de cada paciente con los del tratamiento realizado previamente
    Results of each patient will be compared with results with its previous treatment
    E.8.2.4Number of treatment arms in the trial1
    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 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
    At 6 months of initiation of treatment of the last patient included in the study.
    A los 6 meses del inicio del tratamiento del último paciente incluido en el estudio.
    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 months0
    E.8.9.1In the Member State concerned 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: 5
    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: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    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 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
    Patients are between 5 and 16 years, parents signed consent for children under 12 years and between 12 and 16 will sign the consent and assent.
    Dados que los pacientes son entre 5 y 16 años, los padres firmaran consentimiento en los menores de 12 años y entre 12 y 16 se firmará el consentimiento y el asentimiento.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    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)
    Patients with good response during the test will continue with the formulation (0.55% cysteamine in viscous solution).
    Los pacientes con buena respuesta durante el ensayo, continuarán con la formulación (cisteamina 0,55% en solución viscosa).
    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 Decision2014-07-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-10-02
    P. End of Trial
    P.End of Trial StatusCompleted
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