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 intravitreal administration in patients 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 study eye (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
- Patients of either gender aged ≥ 50 years.
- Women must be using two forms of effective contraception, be postmenopausal 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 |
Patients will not be eligible for the trial if patients 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 (dry or wet) or any prior intravitreal treatment for any indication in either eye, except oral supplements of vitamins and minerals.
- Any ocular condition in the SE that would progress during the course of the study that could affect central vision or otherwise be a confounding factor.
- 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 (square root transformation)
Safety Endpoints:
• AEs
• Vital signs (pulse, systolic and diastolic blood pressure)
• Ophthalmic variables (BCVA, LLBCVA, IOP, and ophthalmic examination)
• 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 |
Baseline, Month 6, and Month 12 |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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) | 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 | 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 | 2 |
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 | 58 |
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 |
Argentina |
Australia |
Brazil |
Canada |
Colombia |
Croatia |
Czech Republic |
Estonia |
France |
Germany |
Hungary |
Israel |
Italy |
Latvia |
Poland |
Spain |
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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |