E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Corneal cystine crystal deposits in pediatric patients with nephropathic cystinosis from 6 months to less than 2 years old |
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E.1.1.1 | Medical condition in easily understood language |
Corneal cystine crystal deposits in pediatric patients with nephropathic cystinosis from 6 months to less than 2 years old |
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E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
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: - Serious ocular Adverse Events (ocular SAEs) - Serious ADRs related to Cystadrops® - All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s)
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E.2.2 | Secondary objectives of the trial |
To assess the efficacy of Cystadrops® by measuring ophthalmologic assessments (CCCS, photophobia and BCVA) after 90 days of treatment with Cystadrops® when possible considering the age of the patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
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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E.4 | Principal exclusion criteria |
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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E.5 End points |
E.5.1 | Primary end point(s) |
Occurrence of the following safety criteria between signature of the ICF and the EOS visit (Day 90): • Ocular Treatment Emergent SAEs, • SADRs related to Cystadrops® • All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s) |
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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. |
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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 | Yes |
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 | No |
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.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
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 | No |
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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The end of study will be at day 90. Then, 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.(Post Trial Access). |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 30 |
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 | 1 |