E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Nephropatic Cystinosis patients with corneal cystine crystal deposits |
Pazienti con cistinosi nefropatica con depositi di cristalli di cistina corneale |
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E.1.1.1 | Medical 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 |
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E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10071112 |
E.1.2 | Term | Nephropathic cystinosis |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main 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
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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 |
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E.2.2 | Secondary 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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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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. |
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E.4 | Principal 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
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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 |
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E.5 End points |
E.5.1 | Primary 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
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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 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary 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 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 7 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial 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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |