[Development and validation of novel clinical endpoints in intermediate age-related macular degeneration in MACUSTAR].
MACUSTAR: Entwicklung und klinische Validierung von funktionellen, strukturellen und patientenberichteten Endpunkten bei intermediärer altersabhängiger Makuladegeneration.
Clinical endpoint
Disease progression
Intermediate age-related macular degeneration
Patient-reported outcome
Structure-function correlation
Journal
Der Ophthalmologe : Zeitschrift der Deutschen Ophthalmologischen Gesellschaft
ISSN: 1433-0423
Titre abrégé: Ophthalmologe
Pays: Germany
ID NLM: 9206148
Informations de publication
Date de publication:
Dec 2019
Dec 2019
Historique:
pubmed:
16
5
2019
medline:
18
12
2019
entrez:
16
5
2019
Statut:
ppublish
Résumé
Currently, no validated clinical endpoints for treatment studies exist for intermediate age-related macular degeneration (iAMD). The European MACUSTAR study aims to develop and clinically validate adequate clinical endpoints for future treatment studies in iAMD and to identify early determinants of disease progression to late stage AMD. The MACUSTAR study protocol was developed by an international consortium of researchers from academia, the pharmaceutical industry and medical device companies. The MACUSTAR project is funded by the Innovative Medicines Initiative 2 (IMI2) of the European Union. The MACUSTAR study consists of a cross-sectional and a longitudinal investigation. A total of 750 subjects with early, intermediate and late AMD as well as control subjects with no signs of AMD will be included with a follow-up period of 3 years. Overall, 20 European study centers are involved. The MACUSTAR project will generate large high-quality datasets, which will allow clinical validation of novel endpoints for future interventional trials in iAMD. The aim is that these endpoints will be accepted as suitable for medication approval studies by the regulatory authorities and that understanding of the disease process will be improved.
Sections du résumé
BACKGROUND
BACKGROUND
Currently, no validated clinical endpoints for treatment studies exist for intermediate age-related macular degeneration (iAMD).
OBJECTIVE
OBJECTIVE
The European MACUSTAR study aims to develop and clinically validate adequate clinical endpoints for future treatment studies in iAMD and to identify early determinants of disease progression to late stage AMD.
MATERIAL AND METHODS
METHODS
The MACUSTAR study protocol was developed by an international consortium of researchers from academia, the pharmaceutical industry and medical device companies. The MACUSTAR project is funded by the Innovative Medicines Initiative 2 (IMI2) of the European Union.
RESULTS
RESULTS
The MACUSTAR study consists of a cross-sectional and a longitudinal investigation. A total of 750 subjects with early, intermediate and late AMD as well as control subjects with no signs of AMD will be included with a follow-up period of 3 years. Overall, 20 European study centers are involved.
CONCLUSION
CONCLUSIONS
The MACUSTAR project will generate large high-quality datasets, which will allow clinical validation of novel endpoints for future interventional trials in iAMD. The aim is that these endpoints will be accepted as suitable for medication approval studies by the regulatory authorities and that understanding of the disease process will be improved.
Identifiants
pubmed: 31087116
doi: 10.1007/s00347-019-0907-1
pii: 10.1007/s00347-019-0907-1
doi:
Types de publication
Journal Article
Review
Langues
ger
Sous-ensembles de citation
IM
Pagination
1186-1193Investigateurs
F Asmus
(F)
M Berger
(M)
A Binns
(A)
M Böttger
(M)
C Bouchet
(C)
J E Brazier
(JE)
T Butt
(T)
C Carapezzi
(C)
J Carlton
(J)
M Costa
(M)
D P Crabb
(DP)
J Cunha-Vaz
(J)
H Dunbar
(H)
M Durbin
(M)
R Finger
(R)
F Holz
(F)
C Hoyng
(C)
J Krätzschmar
(J)
U Luhmann
(U)
A Lüning
(A)
Ph Margaron
(P)
C Martinho
(C)
B Melício
(B)
G Normand
(G)
D Rowen
(D)
G S Rubin
(GS)
J Sahel
(J)
C I Sánchez
(CI)
D Sanches Fernandes
(D)
M Schmid
(M)
S Schmitz-Valckenberg
(S)
A Skelly
(A)
J Terheyden
(J)
A Tufail
(A)
C Wojek
(C)
P Zamiri
(P)
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