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

May 5, 2019 updated by: Frank G. Holz, University Hospital, Bonn

Intravitreal Aflibercept (Eylea) for Therapy of Choroidal Neovascularization (CNV) and Fibrovascular Proliferation (FVP) in Patients With Pseudoxanthoma Elasticum (PXE)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Bonn, Germany, 53127
        • Department of Ophthalmology, Universtiy of Bonn

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Aflibercept
2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use
Drug: Aflibercept
Intravitreal injection
Other Names:
  • Eylea

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in distance best corrected visual acuity between end-of study visit and screening visit
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in light increment sensitivity of central visual field between end-of study visit and screening visit measured by microperimetry
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
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
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
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)
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: 12 months
12 months
Change in the health status of the total population measured by quality of life questionnaire Visual Function Questionnaire (VFQ)-25
Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Bonn

Investigators

  • Principal Investigator: Frank G. Holz, MD, DPhil, University Clinic Bonn

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2015

Primary Completion (Actual)

July 2, 2018

Study Completion (Actual)

July 2, 2018

Study Registration Dates

First Submitted

August 21, 2015

First Submitted That Met QC Criteria

August 27, 2015

First Posted (Estimate)

September 1, 2015

Study Record Updates

Last Update Posted (Actual)

May 7, 2019

Last Update Submitted That Met QC Criteria

May 5, 2019

Last Verified

April 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AUG-201202-EyNeP

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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