| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Neovascular Age-Related Macular Degeneration (AMD) |
|
| E.1.1.1 | Medical condition in easily understood language |
| Degenerative pathology affecting the macular retina of older people |
|
| 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 | 10071129 |
| E.1.2 | Term | Neovascular age-related macular degeneration |
| 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 |
| Assess the safety and tolerability of topical ocular PAN-90806 Eye Drops, Suspension |
|
| E.2.2 | Secondary objectives of the trial |
| Assess the biologic response to topical ocular treatment with PAN-90806 Eye Drops, Suspension |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
1. A treatment-naïve eye diagnosed with active, pathologic, newly diagnosed subfoveal and/or juxtafoveal with center involvement CNV lesions secondary to neovascular AMD (all lesion subtypes: older classifications include predominantly classic, minimally classic, or occult; newer classifications include Type 1 [sub RPE] and Type 2 [subretinal]) with the following characteristics:
• active subfoveal leakage determined by FA;
• total lesion area ≤ 4 disc areas (i.e., ≤ 10 mm2; 1 disc area = 2.54 mm2) determined by FA;
• < 50% of total lesion area with blood;
• < 25% of total lesion area comprised of fibrosis/scar and outside the fovea;
• total area of CNV (including both classic and occult components) must comprise ≥ 50% of the total lesion area; and
• central point and/or central subfield retinal thickness ≥ 300 microns, exclusive of any PEDs in the fovea, determined by SD-OCT.
2. Best-corrected ETDRS visual acuity in the study eye ranging between 68 and 39 letters, inclusive, (approximately equivalent to 20/50 to 20/160 Snellen). Study eye vision must meet this criterion at both Screening and Day 1 Visit. If the BCVA changes by 10 letters or more between Screening and Day 1, participant is NOT eligible for the study. |
|
| E.4 | Principal exclusion criteria |
Lesion Characteristics-Study Eye
3. Significant serous PEDs will be excluded. PEDs will be considered significant if the PED is > 50% of the CNV lesion area or if it is > 400 microns in any diameter. Additional PED characteristics warranting exclusion include a contour that is irregular or corrugated, or evidence of an RPE tear. Borderline PEDs that cannot be clarified for significance after application of these criteria will be excluded if additional assessment by the IRC cannot clearly determine significance or insignificance.
5. The following in the macula: RAP, symptomatic vitreomacular adhesion (sVMA), retinal tear(s)/rip(s), or polypoidal polyps identified by FA and/or SD-OCT (confirmation by ICG angiography is not required). Non-ICG features of polypoidal polyps are well documented in the literature, and include but are not limited to nodular, orange-red RPE elevations observed during ophthalmoscopy, SD-OCT, and/or OCT angiography (OCT-A), neurosensory detachment, and subretinal lipid exudation and hemorrhage. In addition, typically FA evidence of leaking hyperfluorescence is present, which is most often an occult pattern of leakage.
Fellow Eye
7. Prior use (within the last 3 months) or a high possibility of requiring treatment with anti-VEGF therapy in the fellow eye during the study in the opinion of the investigator. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
Ocular safety and tolerability:
1) Ocular AEs (serious and non-serious)
2) Reductions from baseline in ETDRS BCVA
3) Changes from baseline in IOP
4) Changes from baseline in findings from slit-lamp and fundus examinations
5) Changes from baseline in findings from color fundus photography, fluorescein angiography, and SD-OCT
Systemic safety:
6) Non-ocular AEs (serious and non-serious AEs)
7) Changes from baseline in vital signs
8) Changes from baseline in laboratory parameters |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
1) Throughout the study
2 to 5) Screening, Day 1, Week 2, Week 4, Week 8, Week 12, 1 Week post treatment, 1 Month post treatment
6) Throughout the study
7) Screening, Day 1, Week 2, Week 4, Week 8, Week 12, 1 Week post treatment, 1 Month post treatment
8) Screening, Week 12, 1 Week post treatment |
|
| E.5.2 | Secondary end point(s) |
1) Retinal thickness, utilizing center point thickness, center subfield thickness and macular volume by SD-OCT
2) ETDRS BCVA
3) Need for additional treatment with ranibizumab
4) CNV lesion characteristics, such as total area of leakage, total area of CNV lesion, and total area of lesion by FA |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 and 2) Screening, Day 1, Week 2, Week 4, Week 8, Week 12
3) Week 2, Week 4, Week 8, Week 12, 1 Week post treatment
4) Screening, Week 4, Week 8, Week 12 |
|
| 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 | No |
| 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 | Yes |
| E.6.13.1 | Other scope of the trial description |
|
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | Yes |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | Yes |
| 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 |
| Other strengths of the same IMP |
|
| E.8.2.4 | Number of treatment arms in the trial | 3 |
| 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 | 16 |
| 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 |
| Czech Republic |
| Hungary |
| Latvia |
| United Kingdom |
| United States |
|
| 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
|
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 0 |
| E.8.9.1 | In the Member State concerned months | 9 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 0 |
| E.8.9.2 | In all countries concerned by the trial months | 11 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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