Clinical trial OPH2005: Efficacy and safety of Zimura in the treatment of Stargardt’s disease 10/05/2018
OPH2005 is a randomised, double-masked phase 2b study to establish the efficacy and safety of Zimura™ compared with placebo in subjects diagnosed with Stargardt’s disease inherited in an autosomal recessive manner.
The patients will be randomly assigned to different groups.
Men and women between 18 and 50 years old with a minimum of two pathogenic mutations in the gene ABCA4 that will be confirmed by the study laboratories.
The visual acuity of patients should be between 20/20 and 20/200.
The study aim of the study is to assess mean rate of change in the area of ellipsoid zone defect as measured by OCT.
About the drug
Zimura is designed to inhibit C5 complement protein.
Zimura binds to and inhibits C5 being cleaved into C5a and C5b, potentially preventing the formation of inflammasomes and the accumulation of the membrane attack complex (MAC), which would prevent cell death.
FREQUENTLY ASKED QUESTIONS
Who can take part in this clinical trial?
Subjects diagnosed with Stargardt’s disease between 18 and 50 years old with a minimum of two pathogenic mutations in the gene ABCA4 and whose visual acuity must be between 20/20 and 20/200. Moreover, the patients cannot present any other eye disease.
What are the benefits/risks of taking part in a trial?
Participation in a clinical trial offers patients a series of advantages: the new treatment gives them the chance to receive state-of-the-art medical care from experts. This is, for the time being, also the only means of access to new drugs which are not available to
Clinical trials are performed in accordance with strict ethical and scientific principles. We at the Institut apply national and international standards and policies in protection of the rights, safety and welfare of participants.
The risks of participation in a clinical trial may derive from the route of administration of the drug on the one hand and the medication on the other. The risks of the former are well-known and generally low; those deriving from the medication are only partially known but are habitually low and restricted to the eye. In any case, the patient will be informed by the investigator of the possible adverse effects and discomfort resulting from his/her participation in the study. These may vary from one patient to another.
Do patients need to pay?
The patients chosen for clinical trials are not charged for treatment and therefore it is completely free of charge
How long will the trial last?
The duration will be 18 months.
How many times will I have to attend to the Institut de la Màcula?
Once selected to be part of this clinical trial, the patient will have to compulsorily attend the visits scheduled by the study protocol.
These visits will have a fortnightly periodicity during the first three months and monthly from the third month until the end of the study.
Доктор Jordi Monés, M.D., Ph.D.
Номер лицензии COMB: 22.838
Доктор Медицины и Хирургии
Специалист по Офтальмологии
Специалист по лечению Сетчатки, Макулы и Стекловидного тела