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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7272   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2018-002984-24
    Sponsor's Protocol Code Number:CYT-C2-001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-02
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2018-002984-24
    A.3Full title of the trial
    Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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
    A.3.2Name or abbreviated title of the trial where available
    SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years)
    A.4.1Sponsor's protocol code numberCYT-C2-001
    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 number0033147736458
    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 AuthorisationEuropean Union
    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.3.4Pharmaceutical 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 nameβ-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 typeequal
    D.3.10.3Concentration number3.8
    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 corneal cystine crystal 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 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
    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.
    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
    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
    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
    E.5.1.1Timepoint(s) of evaluation of this end point
    D90
    E.5.2Secondary end point(s)
    Change from Day 1 (Inclusion) to Day 90 (EOS) in CCCS, photophobia, and BCVA
    E.5.2.1Timepoint(s) of evaluation of this end point
    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 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 No
    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 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 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 years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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) 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
    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

    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 Decision2020-12-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-12-23
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