E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Retinopathy of prematurity |
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E.1.1.1 | Medical condition in easily understood language |
Retinopathy of prematurity |
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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 | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10038933 |
E.1.2 | Term | Retinopathy of prematurity |
E.1.2 | System Organ Class | 10015919 - Eye disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate long-term safety outcomes and visual function of subjects included in study 20090 for treatment for retinopathy of prematurity (ROP) |
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E.2.2 | Secondary objectives of the trial |
To describe the visual function and overall development of subjects included in study 20090 for treatment for ROP |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects are eligible to be included in the study only if both of the following criteria apply:
1.Subject was treated in Study 20090
2.Age less than 13 months of chronological age
3.Signed informed consent from parent(s)/legally authorized representative(s) as described in Section 10.1.3 of the clinical trial protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
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E.4 | Principal exclusion criteria |
Subjects are excluded from the study if the following criterion applies:
Medical Conditions
1. Subject has a condition preventing participation in the study, or performance of study procedures
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E.5 End points |
E.5.1 | Primary end point(s) |
• Binocular best-corrected visual acuity in Snellen equivalent score at 5 years of age |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Secondary endpoints addressing primary objective:
•Proportion of subjects with ocular adverse events (AEs) and serious AEs (SAEs) through 5 years of age
•Proportion of subjects with systemic AEs and SAEs through 5 years of age
Secondary endpoints addressing secondary objectives:
•Proportion of subjects developing unfavorable ocular structural outcome (retinal detachment, macular dragging, macular fold, retrolental opacity) at 1, 3, and 5 years of age
•Proportion of subjects with absence of active ROP and unfavorable structural outcomes at 1 year of age
•Best-corrected visual acuity in each eye at 3 and 5 years of age
•Refractive spherical equivalent in each eye at 3 and 5 years of age
•Neurodevelopmental outcomes at 2 and 5 years of age using standardized development tests (eg, Bayley Scales of Infant and Toddler
Development, Third Edition [BSID-III], the Differential Ability Scales® II [DAS-II®], the Wechsler Preschool and Primary Scale of Intelligence™, Fourth Edition [WPSSI-IV], Vineland Adaptive Behavior Scales, Second Edition [VABS-II]).
•Proportion of subjects with recurrence of ROP at 3 and 5 years of age
•Proportion of subjects requiring treatment for ROP during this extension study
•Proportion of subjects requiring ophthalmological treatment during this extension study
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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 | 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 |
Visual function of subjects included in study 20090 for treatment for retinopathy of prematurity (ROP) |
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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) | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 67 |
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 |
Austria |
Belgium |
Brazil |
Bulgaria |
Canada |
Czech Republic |
Estonia |
France |
Germany |
Greece |
Hong Kong |
Hungary |
Israel |
Italy |
Japan |
Korea, Democratic People's Republic of |
Latvia |
Lithuania |
Malaysia |
Netherlands |
Poland |
Portugal |
Romania |
Russian Federation |
Singapore |
Slovakia |
Spain |
Sweden |
Switzerland |
Taiwan |
Turkey |
Ukraine |
United Kingdom |
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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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The end of the study as a whole is defined as the date of the last visit of the last subject in the study in all centers in all participating countries (EU and non-EU). |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
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 |