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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
15

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University Hospital, Bonn

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 2, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 2, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 21, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 1, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 7, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 5, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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