E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Nephropathic cystinotic patients with cystine crystals corneal deposits |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10010331 |
E.1.2 | Term | Congenital, familial and genetic disorders |
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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 compare safety of Cystadrops versus a recommended dose regimen reference treatment |
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E.2.2 | Secondary objectives of the trial |
- to compare efficacy of Cystadrops versus a recommended dose regimen reference treatment - to find the lowest efficient dose according to an empiric adaptive dose regimen algorithm - to evaluate the latest developments of measurement methods for corneal topography and cystine crystals deposit assessment.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Treated with reference Cysteamine eye drops at least for 1 month at an usual regimen dose of at least 3 instillations per day Able to comply with a regular dose regimen for 6 months
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E.4 | Principal exclusion criteria |
- Patients with uncontrolled hepatic disorder, cardiovascular disease, neurologic disease, or cancer, - Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if the investigator considers that they are not clinically significant for the conduct of study, - Patients with history or presence of alcohol abuse or drug addiction, - Pregnant or breast-feeding women, - Women of child-bearing potential without effective contraception (oral pill or IUCD), - Patients likely to be non-compliant to the study procedure or for whom a long-term follow-up seems to be difficult to achieve.
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E.5 End points |
E.5.1 | Primary end point(s) |
1-Safety The safety will be based on the assessment and the recording of the adverse events, physical examinations, laboratory parameters and ocular findings including: oTransitional ocular symptoms evaluated by patient and/or parents: - Redness - Blurring - Irritation - Itching - Burning - Discomfort - Pain o Ocular tonometry o Eye fundus examination o Anterior segment topography (Optical Coherence Tomography, slit-scanning)
2- Efficacy o Clinical o Photophobia: 0-5 points scale o Pain: Visual Analogic Scale o Visual acuity (ETDRS chart, contrast scale) o Imaging: corneal crystals deposits measurement o HRT confocal biomicroscopy (Heidelberg Retina Tomograph) o Anterior segment topography (Optical Coherence Tomography) o Slit-lamp CCCS assessment (Cystinosis Corneal Crystal Score)
3- Compliance o Vials weight o Diary card
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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 | No |
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 | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 3 |
E.8.5 | The trial involves multiple Member States | No |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |