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    The EU Clinical Trials Register currently displays   43207   clinical trials with a EudraCT protocol, of which   7151   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2018-002984-24
    Sponsor's Protocol Code Number:CYT-C2-001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-10-21
    Trial 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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-002984-24
    A.3Full title of the trial
    Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old
    Studio multicentrico, in aperto, a braccio singolo volto a valutare la sicurezza di Cystadrops® in pazienti pediatrici con cistinosi, con età compresa tra 6 mesi a meno di 2 anni
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old
    Studio per valutare la sicurezza di Cystadrops® nei pazienti con cistinosi pediatrica di eta' compresa tra 6 mesi e meno di 2 anni
    A.3.2Name or abbreviated title of the trial where available
    SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years)
    SCOB2 (Studio Cystadrops® Oftalmico per pazienti di età inferiore a 2 anni)
    A.4.1Sponsor's protocol code numberCYT-C2-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04125927
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/172/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRecordati Rare Diseases
    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 supportRecordati Rare Diseases
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRecordati Rare Diseases
    B.5.2Functional name of contact pointMedical department
    B.5.3 Address:
    B.5.3.1Street AddressImmeuble Le Wilson, 70 Avenue du General de Gaulle
    B.5.3.2Town/ cityPuteaux
    B.5.3.3Post code92800
    B.5.3.4CountryFrance
    B.5.4Telephone number+33147736458
    B.5.6E-mailplisson.c@recordati.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 Yes
    D.2.1.1.1Trade name Cystadrops
    D.2.1.1.2Name of the Marketing Authorisation holderRecordati Rare Diseases
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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
    D.3.2Product code [045251016]
    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 INNMercaptamina
    D.3.9.1CAS number 156-57-0
    D.3.9.2Current sponsor codeNot applicable
    D.3.9.3Other descriptive nameß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
    D.3.9.4EV Substance CodeSUB08770MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number4
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 corneal cystine crystal deposits
    Pazienti con cistinosi nefropatica con depositi di cristalli di cistina corneale
    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
    Malattia metabolica caratterizzata da un accumulo anormale dell'amminoacido Cistina in vari organi del corpo, uno dei quali è gli occhi
    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 20.0
    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 assess the safety profile of Cystadrops® over a 90-day period as measured by the incidence of:
    o Serious ocular Adverse Events (ocular SAEs)
    o Serious Adverse Drug Reactions (SADRs) related to Cystadrops®
    o All AEs that required temporary discontinuation/ withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visits
    o Eventi avversi oculari seri (SAE oculari)
    o Reazioni avverse al farmaco serie (SADR) correlate a Cystadrops®
    o Tutti gli EA per cui si è reso necessario interrompere/sospendere temporaneamente il trattamento (IMP) o visite oftalmiche non programmate/di emergenza
    E.2.2Secondary objectives of the trial
    To assess the efficacy of Cystadrops® by measuring ophthalmologic assessments (Corneal cystine crystal score (CCCS), photophobia and best corrected visual acuity (BCVA)) of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients.
    •Valutare l'efficacia di Cystadrops® eseguendo delle valutazioni oftalmologiche (Punteggio relativo ai cristalli di cistina nella cornea (Corneal Cystine Crystal Score, CCCS), fotofobia e Migliore acuità visiva corretta (Best Corrected Visual Acuity, BCVA)) su entrambi gli occhi dopo 90 giorni di trattamento con Cystadrops® quando possibile considerando l'età dei pazienti
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all inclusion criteria to be eligible for study participation.
    1. Patient aged from 6 months to less than 2 years old
    2. Cystinosis diagnosed patients confirmed by the physician and with presence of corneal cystine crystal deposits assessed during ophthalmic examination
    3. Evidence of a signed and dated informed consent document indicating that parents/ legally acceptable representatives had been informed of all pertinent aspects of the study (if required by regulation)
    4. Parents/ legally acceptable representatives who are willing to comply with regular visits and ophthalmic exams
    Per essere idonei alla partecipazione allo studio, i pazienti dovranno soddisfare tutti i criteri di inclusione.
    1. Pazienti con età compresa tra 6 mesi e meno di 2 anni.
    2. Pazienti con diagnosi di cistinosi confermata dal medico e con presenza di depositi di cristalli di cistina nella cornea valutati durante l'esame oftalmico.
    3. Sottoscrizione e datazione del Modulo di consenso informato che indichi che i genitori/rappresentanti legalmente autorizzati siano stati informati di tutti gli aspetti pertinenti dello studio (se richiesto dalla normativa).
    4. Genitori/rappresentanti legalmente autorizzati che siano disposti a rispettare le visite regolari e gli esami oftalmici.
    E.4Principal exclusion criteria
    Patients will not be eligible for study participation if they meet any of the exclusion criteria, or will be discontinued at the discretion of the Investigator in consultation with the medical monitor if they develop any of the exclusion criteria during the study.
    1. Contraindications to any of the Cystadrops® components
    2. Participation in another ophthalmic investigational study or intent to participate during the course of the study
    3. Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives
    I pazienti non potranno partecipare allo studio se soddisfano uno qualsiasi dei criteri di esclusione oppure, nel caso in cui sviluppino uno qualsiasi dei criteri di esclusione durante lo studio, la loro partecipazione allo studio verrà interrotta (a discrezione dello Sperimentatore e previa consultazione con il Monitor medico).
    1. Controindicazioni a uno qualsiasi dei componenti di Cystadrops®
    2. Partecipazione ad un altro studio oftalmico sperimentale o intenzione di parteciparvi durante il corso dello studio
    3. Qualsiasi condizione medica che, secondo lo Sperimentatore, interferirebbe con la valutazione degli obiettivi dello studio
    E.5 End points
    E.5.1Primary end point(s)
    Occurrence of the following safety criteria between signature of the informed consent form and the end of study visit (EOS, Day 90):
    o Ocular treatment emergent SAEs,
    o SADRs related to Cystadrops®
    o All AEs that required temporary discontinuation/ withdrawal of treatment (IMP), or unscheduled/emergency ophthalmic visits
    Comparsa dei criteri di sicurezza seguenti nel periodo che intercorre dalla sottoscrizione del Modulo di consenso informato (ICF) alla visita di fine dello studio (EOS, Giorno 90):
    o SAE emergenti dal trattamento oculare,
    o SADR correlati a Cystadrops®
    o Tutti gli EA per cui si è reso necessario interrompere/sospendere temporaneamente il trattamento (IMP) o visite oftalmiche non programmate/di emergenza
    E.5.1.1Timepoint(s) of evaluation of this end point
    D90
    D90
    E.5.2Secondary end point(s)
    Change from Day 1 (Inclusion) to Day 90 (EOS) in CCCS, photophobia, and BCVA
    Variazione, dal Giorno 1 (inclusione) al Giorno 90 (EOS), di CCCS, fotofobia e BCVA
    E.5.2.1Timepoint(s) of evaluation of this end point
    D90
    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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    Aperto
    Open
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 Information not present in EudraCT
    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
    LVLS
    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 months0
    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 months0
    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.1.1.1In 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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    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
    Subjects are aged from 6months to less than 2years old so by definition not able to give consent
    I soggetti hanno un'età compresa tra 6 mesi e meno di 2 anni, quindi per definizione non sono in grado di dare il consenso
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 5
    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)
    IMP can be provided by the site to parents and legally acceptable representatives of patients below 2 years of age at the time of EOS visit (Day 90) free of charge until the patient reaches 2 years of age. Decision to propose continuation of treatment with the IMP will be made by Investigators for each patient at EOS visit (Day 90) based on an individual medical benefit/risk assessment for the concerned patient. See protocol for full details

    Il farmaco in esame può essere fornito dal centro ai genitori e ai rappresentanti legali dei pazienti di età inferiore ai 2 anni al momento della visita EOS (giorno 90) gratuitamente fino a quando il paziente non raggiunge i 2 anni. La decisione di proporre la prosecuzione del trattamento con il farmaco (IMP) sarà presa dagli investigatori per ciascun paziente alla visita EOS (giorno 90) sulla base di una valutazione del beneficio / rischio medico individuale per il paziente interessato.
    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 Decision2020-06-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-17
    P. End of Trial
    P.End of Trial StatusOngoing
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