Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE)

NCT02537054 | Completed Phase 2

• 1 other identifier: AUG-201202-EyNeP
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether Aflibercept (Eylea) is effective in the treatment of choroidal neovascularization and fibrovascular proliferation in patients with pseudoxanthoma elasticum (PXE) in terms of preservation or improvement of visual acuity.

Conditions

Pseudoxanthoma Elasticum

Keywords

Choroidal neovascularization; Angioid streaks; Pseudoxanthoma Elasticum; vascular endothelial growth factor; aflibercept

Outcome Measures

Primary Outcomes (1)

• Change in distance best corrected visual acuity between end-of study visit and screening visit

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Secondary Outcomes (7)

• Change in light increment sensitivity of central visual field between end-of study visit and screening visit measured by microperimetry

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

• Change in chorioretinal neovascularization and leakage measured by angiography between end-of study visit and screening visit

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

• Change in sub- and intraretinal und subpigmentepithelial fluids assessed using optical coherence tomography (OCT)

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

• Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

• Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)

  Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Study Arms (1)

Aflibercept

EXPERIMENTAL

2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use

Drug: Aflibercept

Interventions

Aflibercept DRUG

Intravitreal injection

Also known as: Eylea

Aflibercept

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of PXE by moleculargenetic diagnosis and/or skin biopsy
• Diagnosed CNV or FVP
• Age 18-65 years
• Voluntary participation in this study as proven by written informed consent
• Ability to follow study instructions and likely to attend and complete all required visits
• Best corrected visual acuity between 20/400 and 20/20 at treated eye
• Male and female patients with childbearing potential must use an approved contraceptive method (Pearl Index \< 1) before and during the trial
• Pre-menopausal female patients with childbearing potential: a negative pregnancy test must be obtained

You may not qualify if:

• Subject is unable to understand the nature, scope, significance and consequences of this clinical trial
• Patients with known allergy or hypersensitivity to Eylea or preparations with similar chemical structure
• Treatment in another clinical trial with therapeutic intervention or use of any other investigational medicinal product (IMP) during the trial of within 30 days before enrolment
• Known or persistent abuse of medication, drugs or alcohol
• Women who are pregnant or breast feeding
• Lack of eligibility at discretion of the investigator
• Ocular operations within a month prior to enrolment
• Non-controlled glaucoma
• Active intraocular inflammation or inflammation of ocular adnexa
• Other diseases resulting in distinct visual constraint
• Distinct opacification of optical media
• Distinct subretinal fibrosis and /or atrophy that prevents a relevant treatment effect by Aflibercept at discretion of investigator
• Serious cardiovascular problems or stroke within 6 months before enrolment
• Simultaneous use of other Vascular Endothelial Growth Factor (VEGF)-inhibiting medication (systemic or ocular) within a month prior to enrolment

Sponsors & Collaborators

• University Hospital, Bonn: lead

Study Sites (1)

Department of Ophthalmology, Universtiy of Bonn

Bonn, 53127, Germany

Location

MeSH Terms

Conditions

Pseudoxanthoma Elasticum; Choroidal Neovascularization; Angioid Streaks

Interventions

aflibercept

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Choroid Diseases; Uveal Diseases; Eye Diseases; Neovascularization, Pathologic; Metaplasia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Retinal Diseases

Study Officials

• Frank G. Holz, MD, DPhil

  University Clinic Bonn

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Dr. med. Frank G. Holz

Study Record Dates

• First Submitted: August 21, 2015
• First Posted: September 1, 2015
• Study Start: September 1, 2015
• Primary Completion: July 2, 2018
• Study Completion: July 2, 2018
• Last Updated: May 7, 2019

Record last verified: 2019-04

Locations

DE (1)