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

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ClinicalTrials.gov Identifier: NCT02537054
Recruitment Status : Completed
First Posted : September 1, 2015
Last Update Posted : May 7, 2019
Sponsor:
Information provided by (Responsible Party):
Frank G. Holz, University Hospital, Bonn

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.

Condition or disease Intervention/treatment Phase
Pseudoxanthoma Elasticum Drug: Aflibercept Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Intravitreal Aflibercept (Eylea) for Therapy of Choroidal Neovascularization (CNV) and Fibrovascular Proliferation (FVP) in Patients With Pseudoxanthoma Elasticum (PXE)
Study Start Date : September 2015
Actual Primary Completion Date : July 2, 2018
Actual Study Completion Date : July 2, 2018


Arm Intervention/treatment
Experimental: Aflibercept
2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use
Drug: Aflibercept
Intravitreal injection
Other Name: Eylea




Primary Outcome Measures :
  1. 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) ]

Secondary Outcome Measures :
  1. 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) ]
  2. 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) ]
  3. 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) ]
  4. 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) ]
  5. 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) ]
  6. Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 12 months ]
  7. 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) ]


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02537054


Locations
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Germany
Department of Ophthalmology, Universtiy of Bonn
Bonn, Germany, 53127
Sponsors and Collaborators
University Hospital, Bonn
Investigators
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Principal Investigator: Frank G. Holz, MD, DPhil University Clinic Bonn

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Responsible Party: Frank G. Holz, Prof. Dr. med. Frank G. Holz, University Hospital, Bonn
ClinicalTrials.gov Identifier: NCT02537054     History of Changes
Other Study ID Numbers: AUG-201202-EyNeP
First Posted: September 1, 2015    Key Record Dates
Last Update Posted: May 7, 2019
Last Verified: April 2019
Keywords provided by Frank G. Holz, University Hospital, Bonn:
Choroidal neovascularization
Angioid streaks
Pseudoxanthoma Elasticum
vascular endothelial growth factor
aflibercept
Additional relevant MeSH terms:
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Choroidal Neovascularization
Pseudoxanthoma Elasticum
Choroid Diseases
Uveal Diseases
Eye Diseases
Neovascularization, Pathologic
Metaplasia
Pathologic Processes
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Hematologic Diseases
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Connective Tissue Diseases
Skin Diseases