E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Dry Eye Disease |
Enfermedad de Ojo Seco |
|
E.1.1.1 | Medical condition in easily understood language |
Ocular dryness |
Sequedad ocular |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013774 |
E.1.2 | Term | Dry eye |
E.1.2 | System Organ Class | 10015919 - Eye disorders |
|
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 tivanisiran sodium ophthalmic solution (1.125%) administered as 1 drop OU once a day in treating dry eye disease signs and symptoms in subjects with a documented diagnosis of dry eye associated with Sjögren’s Syndrome. |
Evaluar la eficacia del colirio en solución de tivanisirán sódico (11.25 mg/m) administrado en 1 gota OU una vez al día para el tratamiento de los signos y síntomas de la Enfermedad de Ojo Seco (EOS) en sujetos con un diagnóstico documentado de Ojo Seco asociado al Síndrome de Sjögren. |
|
E.2.2 | Secondary objectives of the trial |
Evaluate the safety of tivanisiran sodium ophthalmic solution (1.125%) administered as 1 drop OU QD in treating DED signs and symptoms in subjects with a documented diagnosis of dry eye associated with Sjögren’s Syndrome. |
Evaluar la seguridad del colirio en solución de tivanisiran sódico 11.25 mg/ml administrado 1 gota OU una vez al día para el tratamiento de los signos y síntomas de la EOS en sujetos con un diagnóstico documentado de Ojo Seco asociado al Síndrome de Sjögren. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject is a male or a female aged ≥ 18 years - Have given their written consent to participate in the study - Use of artificial tears, autologous serum or specific dry eye medications during the last 6 months. - Willing to not use AT or autologous serum for the study duration. - VAS scale for Dry Eye Symptom Score ≥40. - Total CFS using ≥ 5 - Schirmer’s test with anesthesia < 10 mm/5min - Diagnosed with Sjögren’s Syndrome |
- Ambos sexos con edad ≥ 18 años - Que hayan firmado el consentimiento informado. - Uso de lagrimas artificiales, suero autólogo o medicación específica para el ojo seco durante los últimos 6 meses. - Dispuesto a no utilizar lagrimas artificiales o suero autólogo durante todo el estudio. - Escala VAS para la puntuación de los síntomas del ojo seco ≥40. - Tinción corneal total con fluoresceína (CFS) ≥ 5. - Diagnóstico de Síndrome de Sjögren |
|
E.4 | Principal exclusion criteria |
- Any concomitant treatment or prior ocular procedure or surgery, or alteration of the dose of systemic medications at the time of entry into the study - Use of contact lenses during the study - Significant Eye diseases according to investigator's opinion |
- Cualquier medicación concomitante o procedimiento o cirugía ocular previo o alteración de la dosis de medicamentos sistémicos en el momento de la entrada en el estudio - Uso de lentillas durante el estudio. - Cualquier otra enfermedad ocular que sea significativa a juicio del Investigador. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. Mean change from Baseline to Day 85 in Corneal Fluorescein Staining (CFS) total score [0-3 scale] 2. Mean change from Baseline to Day 85 in Dry Eye Symptom Score (0-100 scale). |
1. Cambio medio desde el inicio hasta el día 85 en la puntuación total de la tinción corneal con fluoresceína (CFS). 2. Cambio medio desde el inicio hasta el día 85 en la puntuación de síntomas de ojo seco (escala de 0 a 100). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- Mean change from Baseline to Day 29, Day 57 and Day 85 in Corneal Fluorescein Staining (CFS) central region score - Mean change from Baseline to Day 85 in Corneal Fluorescein Staining (CFS) in the most severe region |
- Cambio medio desde el inicio hasta el día 29, 57 y 85 en la puntuación de la tinción corneal con fluoresceína (CFS) en la región central - Cambio medio desde el inicio hasta el día 85 en la puntuación de la tinción corneal con fluoresceína (CFS) en la región más afectada |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Days 29, 57 y 85 |
Días 29, 57 y 85 |
|
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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 8 |
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 | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
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 | |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |