E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Geographic Atrophy Secondary to Age-related Macular Degeneration |
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E.1.1.1 | Medical condition in easily understood language |
Geographic Atrophy Secondary to Age-related Macular Degeneration |
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E.1.1.2 | Therapeutic area | Body processes [G] - Ocular Physiological Phenomena [G14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10025409 |
E.1.2 | Term | Macular degeneration |
E.1.2 | System Organ Class | 10015919 - Eye disorders |
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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 |
The study objectives are to assess: (1) the safety of the treatment; (2) the effects of treatment on the mean change in atrophic lesion area quantified from fundus autofluorescence images; (3) the effects of treatment on the mean change in low luminance best corrected visual acuity (BCVA); (4) the effects of treatment on the mean change in standard BCVA; (5) the effects of treatment on the mean change in retinal sensitivity quatified using scotopic/mesopic microperimetry; (6) to characterize the systemic pharmacokinetic profile of Brimo DDS. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Male or female patients 55 years of age or older as assessed at Screening Visit 1 The study eye must have: • Diagnosis of GA secondary to AMD, as assessed with fundus autofluorescence (FAF) at Screening Visit 1 and confirmed by the central reading center (CRC); these atrophic areas consist of funduscopically visible discrete areas characterized by a marked decrease in fundus autofluorescence intensity and loss of outer-retinal layers with choroidal signal enhancement as assessed with SD-OCT. Areas of atrophy must be multifocal lesions that contain all of the following characteristics: - At least one focal lesion must be equal to or larger than 0.2 mm2 - The total lesion area must be greater than 1.25 mm2, but not larger than 18 mm2 - The presence of banded or diffuse perilesional hyper-autofluorescence evident on fundus autofluorescence examination. - The distance between the optic disc or peripapillary atrophy and any atrophic lesion must be greater than 300 μm • Presence of drusen, yellow or white accumulations of extracellular material formed between Bruch's membrane and the retina pigmented epithelial cell layer, assessed with fundus photography and/or SD-OCT at Screening Visit 1 and confirmed by the CRC - BCVA better than or equal to 45 letters (20/125 Snellen equivalent) at Screening Visit 1 and the Baseline visit as assessed using the standard ETDRS (Early Treatment of Diabetic Retinopathy Study) visual acuity protocol - Clear ocular media and adequate pupil dilation to permit good quality retinal imaging as assessed at Screening Visit 1 and confirmed by the CRC - For patients participating in microperimetry assessments: abitity to complete repeat assessments of scotopic/mesopic microperimetry with an intra-visit, mean, retinal sensitivity reproducibility of 6 dB or less, as assessed at Screening Visit 2 and confirmed by the CRC • The Fellow Eye must have Standard BCVA of 34 letters (Snellen equivalent 20/200) or better at Screening Visit 1
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E.4 | Principal exclusion criteria |
• History or evidence of choroidal neovascularization in either eye at Screening Visit 1, as confirmed by the CRC • Diagnosis of GA secondary to AMD that presents as unifocal atrophic lesions, lesions with no perilesional hyper-autofluorescence, lesions with only focal areas of perilesional hyper-autofluorescence on FAF examination of the study eye at Screening Visit 1 and confirmed by the CRC • Spherical equivalent of the refractive error of -6 diopters of myopia or worse (prior to cataract or refractive surgery) in the study eye at Screening Visit 1 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Atrophic lesion area as assessed with fundus autofluorescence and quantified by the CRC |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
24 months following the first treatment. |
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E.5.2 | Secondary end point(s) |
• Retinal sensitivity as assessed with scotopic/mesopic microperimetry • Low luminance BCVA as assessed using a 2.0 log unit neutral density filter and the ETDRS visual acuity protocol • Standard BCVA assessed using the ETDRS visual acuity protocol |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
24 months following the first treatment. |
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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 | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Genotyping and gene expression will also be performed. |
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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 | 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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Australia |
France |
Germany |
Italy |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 3 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
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 |