E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Retinopathy of Prematurity (ROP) |
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E.1.1.1 | Medical condition in easily understood language |
Retinopathy of prematurity (ROP) is a pathologic process that occurs in the developing retina of low birth-weight preterm neonates. |
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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 | 19.0 |
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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess visual function of the better seeing eye |
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E.2.2 | Secondary objectives of the trial |
To assess:
- number of patients having any ocular Adverse Event
- number of patients having any systemic Adverse Event
- visual function of the worse seeing eye
- absence of active Retinopathy of Prematurity
- absence of ocular structural abnormalities
- recurrence of ROP
- number of Lucentis administrations
- refraction in each eye
- standing/sitting height and leg length
- weight
- head circumference
- respiratory function
- hearing function
- duration of hospitalization
- weight at first hospital discharge |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The patient successfully completed the core study CRFB002H2301
The patient received study treatment in both eyes at baseline of study CRFB002H2301 |
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E.4 | Principal exclusion criteria |
Patient has a medical condition or personal circumstance which precludes study participation or compliance with study procedures, as assessed by the Investigator
Patient has been discontinued from the core study CRFB002H2301 at any time
Other protocol-defined inclusion/exclusion criteria may apply |
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E.5 End points |
E.5.1 | Primary end point(s) |
Visual acuity (VA) in the better-seeing eye |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
VA is being assessed at the patient's fifth birthday. |
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E.5.2 | Secondary end point(s) |
Ocular Adverse Events
Systemic Adverse Events
VA of the worse-seeing eye
Active ROP
Ocular structural abnormalities
Recurrence of ROP
Lucentis administrations
Refraction
Height/leg length
Weight
Head circumference
Respiratory function
Hearing function
Hospitalization (duration)
Weight (discharge) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At the patient's fifth birthday
At the patient's fifth birthday
At the patient's fifth birthday
40 and 52 weeks after initial treatment
40 and 52 weeks after initial treatment, at patient's two years corrected age and fifth birthday
40 and 52 weeks after initial treatment
40 weeks after initial treatment
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
at patient's two years corrected age
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
At patient's two years corrected age and fifth birthday
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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 | 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 |
extension study to CRBF002H2301 (RAINBOW study) |
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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.8.3 |
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 | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 38 |
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 |
Austria |
Belgium |
Canada |
Chile |
Colombia |
Croatia |
Czech Republic |
Denmark |
Egypt |
Estonia |
Finland |
France |
Germany |
Greece |
Hungary |
India |
Italy |
Japan |
Lithuania |
Malaysia |
Mexico |
Poland |
Romania |
Russian Federation |
Saudi Arabia |
Slovakia |
Sweden |
Taiwan |
Turkey |
United Arab Emirates |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
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 | 3 |