E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Ocular cicatricial pemphigoid (OCP) |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10010746 |
E.1.2 | Term | Conjunctivitis chronic |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
evaluate the efficacy of Cyclosporine A 0.05% eye drop treatment in controlling conjunctival inflammation in patients with OCP, in addition to conventional immunosuppressive systemic therapy (CIST). |
|
E.2.2 | Secondary objectives of the trial |
compare progression of conjunctival fibrosis (Foster Classification), visual acuity, ocular surface changes, quality of life, tear function and conjunctival epithelial cytological changes between the CyA eyedrop treated group and the placebo treated group of patients. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) diagnosis of cicatricial pemphigoid with ocular involvement observed and confirmed by direct immunofluorence at conjunctival biopsy 2) a minimum of 3 months of follow-up before the inclusion in the study 3) presence of first-line conventional immunosuppressive therapy and artificial tears at the time of enrolment 4) evidence of at least one relapse of conjunctival inflammation. |
|
E.4 | Principal exclusion criteria |
Patients with severe inflammatory disease not responding to the described first-line systemic therapy and requiring systemic Cyclophosphamide plus high doses of Prednisolone at the time of enrolment, will be not included. Patients with corneal ulceration, patients affected by infective ocular diseases, patients subjected to ocular surgery in the preceding 6 months, patients under ocular treatments or patients on systemic treatments that can influence the immunosuppressive therapy, or patients enrolled in experimental trials in the preceding 3 months shall be excluded. In addition, patients in pregnancy or in breastfeeding |
|
E.5 End points |
E.5.1 | Primary end point(s) |
evaluate the percentage of patients showing more than 2 relapses of conjunctival inflammation during the 2 years of treatment. |
|
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 | 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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
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 | 7 |
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
| |
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 | |
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 |