Clinical trial NCT04543331: Observational Study to Evaluate Fluid Resolution and Effectiveness in Patients Receiving Beovu in Neovascular Age-related Macular Degeneration and Visual Impairment Due to Diabetic Macular Edema
Official title: OBservationaL stUdy to Evaluate Fluid reSolution and Effectiveness in Patients Receiving Beovu (Brolucizumab) Under KeY Treatment Schemes in Neovascular Age-related Macular Degeneration and Visual Impairment Due to Diabetic Macular Edema
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Neovascular Age-related Macular Degeneration (nAMD)
Diabetic Macular Edema (DME)
Keywords: Beovu
Brolucizumab
nAMD
DME
non-interventional trial
effectiveness
fluid resolution
treatment intervals
real world study
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2020-09-10
Enrollment: 573
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients being treated for nAMD or DME with brolucizumab will be enrolled in the study upon signing an informed consent.

Brief summary: This study is a prospective, non-interventional, multicenter, open-label study in nAMD and DME patients being treated with brolucizumab according to the EU SmPC.

An observational study design, without a strict, mandated visit schedule or mandated treatment regimen was chosen as the most appropriate to collect available data in a real life setting. For that reason, this NIS does not impose a therapy protocol, diagnostic/therapeutic procedure or a visit schedule. The diagnostic or monitoring procedures are only those ordinarily applied to the therapeutic strategy and to routine clinical care and will take place as per investigator's discretion. This includes e.g. visit frequency, injection frequency and types of assessments performed - only data from routine medical practice will be collected as part of the study.

Detailed description: BLUE SKY consists of the nAMD-related module BIRL (Brolucizumab In Real Life) and two general, indication-related study modules "BLUE SKY AMD" and "BLUE SKY DME".

Patients will be enrolled at approximately 55 centers, which will be representatively distributed amongst the 16 German federal states. This will ensure to reflect all state-specific health insurance laws and guidelines. Naïve and pre-treated patients will be included after decision to start treatment with brolucizumab and consent is given.

All patients will be documented either in the AMD or DME-module (Full Analysis Set).

For the BIRL module, only naïve nAMD patients enrolled in approximately 7 selected centers will be documented. All these centers are equipped with the Zeiss PlexElite OCT-A, needed to assess and analyze BIRL specific objectives in a comparable setting. Only images from routine medical practice will be collected as part of the study. The study eye will be defined as the first eye treated during the study, the other eye will be considered as fellow eye.

If both eyes are treated at baseline, the eye with the worse VA will be chosen as the study eye (if VA is measured equal, the treating ophthalmologist defines the study eye upon his discretion).

The prospective observation period per patient will be up to 24 months.

Eligibility criteria: Inclusion Criteria:

The unit of analysis is patient-eye, i.e. all criteria should be applied to the study eye, unless otherwise specified. Patients will be included in the study if they fulfil the following requisites:

1. Diagnosis of nAMD or visual impairment due to DME
2. Male and Female nAMD and DME patients with ≥18 years of age at index
3. Decision to treat with brolucizumab at baseline visit
4. Signed written informed consent
5. Patients for whom a therapy with brolucizumab is medically indicated
6. Intraretinal and/or subretinal fluid affecting the central subfield of the study eye at Screening

Exclusion Criteria:

1. Patients that have any contraindication or are not eligible for treatment with brolucizumab as according to the SmPC
2. Patients treated for RVO or CNV other than nAMD
3. Receipt of any anti-VEGF treatment other than brolucizumab in the study eye at index date
4. Central subfield of the study eye affected by fibrosis or geographic atrophy or total area of fibrosis \>50% of the total lesion (nAMD)
5. Any active intraocular or periocular infection or active intraocular inflammation in either eye at index date
6. Patients who have been on anti-VEGF treatment for longer than 3 years (before index date)
7. Any medical or psychological condition in the treating physician's opinion which may prevent the patient from the 24 months study participation
8. Patients participating in parallel in an interventional clinical trial
9. Patients participating in parallel in any other NIS generating primary data for an anti-VEGF drug
10. Laser photocoagulation (focal/grid or panretinal) in the study eye during the 3-month period prior to baseline
11. Prior use of intraocular or periocular corticosteroids in the study eye provided at least 4 months prior to baseline
12. Pregnancy and breast-feeding

Primary outcomes:
measure: Percentage of patients' eyes that are absent of subretinal fluid (SRF) and intra-retinal fluid (IRF)
timeFrame: Month 12
measure: Mean change in visual actuity (VA) under clinic specific routine treatment schemes
timeFrame: Baseline, month 12
measure: Morphological CNV-Changes under clinic specific routine treatment schemes
timeFrame: Baseline, month 12
measure: Percent of patients maintained on q12w dosing after loading through Week 52
timeFrame: After loading, month 12
measure: Change in interval length from last interval before switch (baseline) to last interval at end of follow-up (12 months)
timeFrame: Baseline, month 12
Secondary outcomes:
measure: Characterize nAMD patients who initiated treatment with brolucizumab with the respect to baseline characteristics
timeFrame: Baseline
measure: Evaluate anatomical parameters during treatment with brolucizumab
timeFrame: Up to month 24
measure: Evaluate VA change from baseline during treatment with brolucizumab
timeFrame: Baseline, month 24
measure: Estimate number of anti-VEGF injections, visits and injection intervals
timeFrame: Up to month 24
measure: Estimate percentage of switchers during first 6 months and characterize switchers
timeFrame: Baseline, month 6
measure: Estimate discontinuation rate and time to discontinuation
timeFrame: Month 1-12 and 13-24
measure: Assess retreatment criteria
timeFrame: Up to month 24
measure: Estimate the number of OCT
timeFrame: Month 1-12 and 13-24
measure: Assess the safety of brolucizumab
timeFrame: Up to month 24
measure: Evaluate anatomical effectiveness
timeFrame: Up to month 24
measure: Evaluate treatment intervals
timeFrame: Up to month 24
measure: Describe implementation of treatment schemes into clinical practice
timeFrame: Up to month 24
measure: Evaluate morphological changes under brolucizumab treatment using multimodal imaging
timeFrame: Up to month 24
measure: Percentage of patient's eyes with absence of IRF and/or SRF at month 12 (naive and switch)
timeFrame: Month 12
measure: Evaluate anatomical parameters during treatment with brolucizumab
timeFrame: Month 1-12 and 13-24
measure: Characterize VA change from baseline during treatment with brolucizumab
timeFrame: Month 1-12 and 13-24
measure: Estimate discontinuation rate during first year of brolucizumab treatment
timeFrame: Baseline, month 12
measure: Estimate time to discontinuation (persistence)
timeFrame: Up to month 24
measure: Estimate the treatment burden
timeFrame: Month 1-12 and 13-24
measure: Estimate the number of OCT and the number of visits with/without OCT
timeFrame: Month 1-12 and 13-24
measure: Assess safety of brolucizumab
timeFrame: Up to month 24
Other outcomes:
Locations:
city: Wuerzburg
country: Germany
facility: Novartis Investigative Site
state: Bayern
city: Frankfurt am Main
country: Germany
facility: Novartis Investigative Site
state: Hessen
city: Goettingen
country: Germany
facility: Novartis Investigative Site
state: Niedersachsen
city: Osnabrueck
country: Germany
facility: Novartis Investigative Site
state: Niedersachsen
city: Trier
country: Germany
facility: Novartis Investigative Site
state: RP
city: Halle Saale
country: Germany
facility: Novartis Investigative Site
state: Sachsen-Anhalt
city: Leipzig
country: Germany
facility: Novartis Investigative Site
state: Sachsen
city: Schneeberg
country: Germany
facility: Novartis Investigative Site
state: Sachsen
city: Aschersleben
country: Germany
facility: Novartis Investigative Site
city: Bayreuth
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Bonn
country: Germany
facility: Novartis Investigative Site
city: Breisach am Rhein
country: Germany
facility: Novartis Investigative Site
city: Chemnitz
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Esslingen
country: Germany
facility: Novartis Investigative Site
city: Glauchau
country: Germany
facility: Novartis Investigative Site
city: Hoesbach
country: Germany
facility: Novartis Investigative Site
city: Homburg
country: Germany
facility: Novartis Investigative Site
city: Jena
country: Germany
facility: Novartis Investigative Site
city: Kiel
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
city: Leipzig
country: Germany
facility: Novartis Investigative Site
city: Magdeburg
country: Germany
facility: Novartis Investigative Site
city: Marienberg
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Muenster
country: Germany
facility: Novartis Investigative Site
city: Neubrandenburg
country: Germany
facility: Novartis Investigative Site
city: Neuburg
country: Germany
facility: Novartis Investigative Site
city: Rostock
country: Germany
facility: Novartis Investigative Site
city: Saarbruecken
country: Germany
facility: Novartis Investigative Site
city: Schoenebeck
country: Germany
facility: Novartis Investigative Site
city: Stralsund
country: Germany
facility: Novartis Investigative Site
city: Sulzbach
country: Germany
facility: Novartis Investigative Site
city: Torgau
country: Germany
facility: Novartis Investigative Site
city: Waren
country: Germany
facility: Novartis Investigative Site
city: Wuerzburg
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: