E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration |
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E.1.1.1 | Medical condition in easily understood language |
Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10075567 |
E.1.2 | Term | Dry age-related 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 objectives of this study are to evaluate the safety and efficacy of Zimura™ intravitreous administration when administered in subjects with geographic atrophy secondary to dry age-related macular degeneration (AMD). |
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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 |
Ophthalmic Inclusion Criteria
The following inclusion criteria apply to the SE:
- Non-foveal GA secondary to dry AMD.
- The atrophic lesion must be able to be photographed in its entirety.
- Best corrected visual acuity in the SE between 20/25 – 20/320, inclusive.
General Inclusion Criteria
- Subjects of either gender aged ≥50 years.
- Women must be using two forms of effective contraception, be post-menopausal for at least 12 months prior to trial entry, or surgically sterile; if of child-bearing potential, a serum pregnancy test must be performed within 14 days prior to the first injection with a negative result. The two forms of effective contraception must be implemented during the trial and for at least 60 days following the last dose of test medication.
- Provide written informed consent.
- Ability to return for all trial visits.
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E.4 | Principal exclusion criteria |
Exclusion Criteria Subjects will not be eligible for the trial if subjects cannot attend all trial required visits, or if any of the following criteria are present systemically or in the SE:
Ophthalmic Exclusion Criteria The following exclusion criteria apply to the SE:
- Evidence of CNV in either eye.
- Any prior treatment for AMD or any prior intravitreal treatment for any indication in either eye, except oral supplements of vitamins and minerals.
- Presence of other causes of choroidal neovascularization.
- Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region, regardless of indication.
- Any ocular or periocular infection (including blepharitis), or ocular surface inflammation in the past 12 weeks.
- Any sign of diabetic retinopathy in either eye. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoint: Mean rate of change in GA over 12 months measured by FAF at three time points: Baseline, Month 6, and Month 12
Safety Endpoints: - AEs - Vital signs (pulse, systolic and diastolic blood pressure) - Ophthalmic variables (IOP, ophthalmic examination, FA, FAF, and OCT) - ECG (12-lead) - Laboratory variables (blood: hematology, renal function, hepatic function, and electrolytes; urinalysis) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Primary Efficacy Endpoint: From Baseline to Month 12
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E.5.2 | Secondary end point(s) |
Secondary Efficacy Endpoints: - The mean change in best corrected visual acuity (ETDRS letters) from Baseline to Month 12 - Mean change in low luminance best corrected visual acuity (ETDRS letters) from Baseline to Month 12 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
From Baseline to Month 12 |
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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 | Yes |
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) | 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 |
Zimura and sham administered by an unmasked investigator |
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E.8.2.4 | Number of treatment arms in the trial | 6 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 49 |
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 |
Croatia |
Czech Republic |
Estonia |
Hungary |
Israel |
Italy |
Latvia |
Spain |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 22 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 29 |