E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Retinitis Pigmentosa associated Cystoid Macular Oedema |
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E.1.1.1 | Medical condition in easily understood language |
Retinitis Pigmentosa in an inherited retinal degenerative eye disorder that causes progressive loss of vision. It can also cause swelling at the back of the eye that affects central vision. |
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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 | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10038914 |
E.1.2 | Term | Retinitis pigmentosa |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 report mean Central Macular Thickness (CMT) at 6 and 12 months as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and twelve months. |
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E.2.2 | Secondary objectives of the trial |
To report mean change in Central Macular Thickness (CMT) as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol at between baseline and six months, and baseline and twelve months.
To report the mean BCVA ETDRS letter score at 6 and 12 months in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and twelve months.
To report the mean change in BCVA ETDRS letter score in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report mean macular volume at 6 and 12 months as measured wi |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) At least 16 years of age 2) Unilateral or Bilateral CMO (the worse eye only will be treated – defined as the eye with a greater central macular thickness (CMT) on OCT) 3) No previous oral treatment for CMO for last 3 months 4) No previous peribulbar or intravitreal treatment for CMO in the study eye for last 3 months 5) No previous topical treatment for CMO in the study eye for last 1 month 6) Central visual impairment that in the view of the Principal Investigator is due to CMO 7) BCVA better than 3/60. |
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E.4 | Principal exclusion criteria |
1) Insufficient patient cooperation or media clarity to allow adequate fundus imaging 2) Evidence of visually significant vitreo-retinal traction or epiretinal membrane on OCT that in the Principal Investigator’s opinion is highly likely to significantly limit the efficacy of intravitreal therapy 3) History of cataract surgery within prior 3 months or cataract surgery anticipated within 6 months of starting the study 4) Any anti-VEGF treatment to study eye within 3 months 5) History of YAG capsulotomy performed within 3 months 6) Uncontrolled IOP > = 24 mmHg for ocular hypertension (on topical IOP lowering medications) 7) Advanced glaucoma (in the opinion of a glaucoma specialist) 8) Patients with active or suspected ocular or periocular infections 9) Patients with active severe intraocular inflammation 10) Patients with a new, untreated retinal tear or detachment, 11) Patients with a stage 3 or 4 macular hole 12) Thromboembolic event (MI/CVA/Unstable Angina) within 6 months 13) Pregnancy or family planned within 15 months 14) Females who are breast feeding 15) Known allergy or hypersensitivity to anti-VEGF products
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E.5 End points |
E.5.1 | Primary end point(s) |
To report mean Central Macular Thickness (CMT) at 12 months as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months and baseline and twelve months. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
28 days following the last visit of the last recruited patient in the study. The last visit of the last recruited patient in the study is defined as 12 months after they received their first intravitreal injection of Eylea. |
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E.5.2 | Secondary end point(s) |
To report mean Central Macular Thickness (CMT) at 6 months as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report mean change in Central Macular Thickness (CMT) as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report the mean BCVA ETDRS letter score at 6 and 12 months in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months and baseline and twelve months.
To report the mean change in BCVA ETDRS letter score in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report mean macular volume at 6 and 12 months as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months and baseline and twelve months.
To report mean change in macular volume as measured with SDOCT in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report all AEs and SAEs at any time point during the 12 month study of using intravitreal Eylea in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema.
To report the mean retinal sensitivity at 6 and 12 months using Microperimetry in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months and basleine and twelve months.
To report the mean change in retinal sensitivity using Microperimetry in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and six months, and baseline and twelve months.
To report the mean number of intravitreal injections administered in eyes of patients with Retinitis Pigmentosa associated with cystoid macular oedema treated with three loading doses of Eylea at monthly intervals followed by a treat and extend protocol between baseline and twelve months.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
28 days following the last visit of the last recruited patient in the study. The last visit of the last recruited patient in the study is defined as 12 months after they received their first intravitreal injection of Eylea. |
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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 | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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 last visit of the last subject undergoing the trial will occur 12 months after that last patient received their first intravitreal injection of Eylea. The study will end 28 days after this visit. This is to allow time for reporting possible adverse events from the final injection. |
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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 | 7 |
E.8.9.1 | In the Member State concerned days | 28 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 28 |