Pseudoxanthoma ElasticumPhase 2EyNePNCT02537054
Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE)
A Phase 2 study testing Aflibercept in people with Pseudoxanthoma Elasticum, run by University Hospital, Bonn at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Pseudoxanthoma Elasticum
- What is being tested?
- Aflibercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteGermany
- Study dates
- Sep 2015to 2 Jul 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Pseudoxanthoma Elasticum
- Being tested
- Aflibercept
- Keywords
- Choroidal neovascularization, Angioid streaks, Pseudoxanthoma Elasticum, vascular endothelial growth factor, aflibercept
- Medical subject headings
- Pseudoxanthoma Elasticum, Choroidal Neovascularization, Angioid Streaks
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AfliberceptDrug
Also called Eylea
Intravitreal injection
| Study group | Role | What they receive |
|---|---|---|
| Aflibercept | Experimental | 2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use Receives: Drug: Aflibercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in distance best corrected visual acuity between end-of study visit and screening visit
Measured: 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
Measured: 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
Measured: 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)
Measured: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging
Measured: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)
Measured: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: 12 months
Change in the health status of the total population measured by quality of life questionnaire Visual Function Questionnaire (VFQ)-25
Measured: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion2 Jul 2018
- Study completion2 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pseudoxanthoma ElasticumStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bonn
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Frank G. Holz, University Hospital, Bonn
- Investigators
- Frank G. Holz, MD, DPhil, Principal investigator, University Clinic Bonn
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02537054
- Sponsor's protocol ID
- AUG-201202-EyNeP
- Acronym
- EyNeP
- Status last verified
- Apr 2019
- First submitted
- 21 Aug 2015
- Last updated
- 7 May 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravitreal Aflibercept (Eylea) for Therapy of Choroidal Neovascularization (CNV) and Fibrovascular Proliferation (FVP) in Patients With Pseudoxanthoma Elasticum (PXE)
- ClinicalTrials.gov ID
- NCT02537054
- Acronym / sponsor ID
- EyNeP / AUG-201202-EyNeP
- Conditions
- Pseudoxanthoma Elasticum
- Keywords
- Choroidal neovascularization, Angioid streaks, Pseudoxanthoma Elasticum, vascular endothelial growth factor, aflibercept
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University Hospital, BonnUniversity, hospital or other
- Study start
- Sep 2015
- Primary completion
- 2 Jul 2018 (actual)
- Study completion
- 2 Jul 2018 (actual)
- First posted
- 1 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Aflibercept | Experimental | Drug: Aflibercept2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AfliberceptAlso: EyleaIntravitreal injection | Drug | Aflibercept |
Primary outcome
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)
7 secondary outcomes
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)
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)
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)
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)
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)
Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Frank G. Holz, MD, DPhil principal investigator
University Clinic Bonn
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Aflibercept in people with Pseudoxanthoma Elasticum, run by University Hospital, Bonn at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2019.