E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pseudoxanthoma elasticum (PXE) is an inherited systemic disease characterized by changes in the elastic tissue. Pseudoxanthoma elasticum mainly affects the skin, eyes, heart, and GI system. The cutaneous and ocular findings of PXE are referred to as Grönblad-Strandberg syndrome. Pseudoxanthoma elasticum may be autosomal dominant or autosomal recessive. In Pseudoxanthoma elasticum, choroidal nevoscualrization (CNV) is a frequently occuring complication in late stage disease |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10037150 |
E.1.2 | Term | Pseudoxanthoma elasticum |
|
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 investigate the efficacy of Ranibizumab (0.5 mg) in improving or maintaining visual acuity in choroidal neovascularization due to Pseudoxanthoma elasticum |
|
E.2.2 | Secondary objectives of the trial |
To investigate the effect of ranibizumab on • Vascular leakage as shown by fluorescein and indocyanin green angiography, • Retinal thickness as shown by optical coherence tomography, • Funduscopical/morphological aspects of the disease as documented by dilated fundus exam, auto-fluorescence and photography, • Quality of life of the investigated sample.
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• 18 – 65 yrs. of age. • PXE-diagnosis ascertained by genetic analysis. • Angiographically diagnosed CNV in the study eye. • Written informed consent. • Best corrected visual acuity: 20/200 – 20/32 in the study eye. |
|
E.4 | Principal exclusion criteria |
• Patients with other retinal diseases including diseases of the retinal vessels or diabetic retinopathy. • Ocular surgery mess than three months prior to the commencment of the trial. • History of a not well meneged glaucoma. • Active intraocular inflammation or inflammation of the ocular adnexa. • Subfoveal fibrosis in the study eye. • Inability, to comply with the study protocol. • Major surgical interventions less than 1 month prior to the commencment of the trial. • Known allergy to humanized antibodies. • History of clinically significant cardiovascular or cerebrovascular disease less than 6 months before commencment of the trial
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
• Visual acuity (as measured by the ETDRS chart at 4 meters or 1 meter distance one month after the 12th injection of Lucentis compared to the baseline visual acuity). |
|
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) | 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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | Information not present in EudraCT |
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
|
End of the trial will be the last visit of the last subject participating in the trial. Specifications are given in the protocol. |
|
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 | |
E.8.9.1 | In the Member State concerned days | |