Zimura™ in Autosomal Recessive Stargardt Disease
Research type
Research Study
Full title
A PHASE 2B RANDOMIZED, DOUBLE-MASKED, CONTROLLED TRIAL TO ESTABLISH THE SAFETY AND EFFICACY OF ZIMURA™ (COMPLEMENT C5 INHIBITOR) COMPARED TO SHAM IN SUBJECTS WITH AUTOSOMAL RECESSIVE STARGARDT DISEASE
IRAS ID
242268
Contact name
Michel Michaelides
Contact email
Sponsor organisation
Ophthotech Corporation
Eudract number
2017-004783-35
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 5 months, 13 days
Research summary
Stargardt disease is a genetic eye disorder which causes damage to cells of the central portion of the retina (nerve layer part of eye that works like film in a camera to pick up the picture) called the macula. The macula’s responsible for fine central vision needed for driving a car, reading fine print, recognizing faces, etc.
It is commonly caused by an inherited genetic disorder due to mutation in the gene called “ABCA4”. Cell damage in the macula over time causes slow progression of central vision loss in both eyes.
This study is for subjects with Stargardt disease. In order to participate genetic testing will be performed to determine if subjects have the common type of Stargardt disease, caused by mutation in the ABCA4 gene.
Zimura™ (ARC1905), investigational drug being studied for treatment of Stargardt’s disease. Zimura ™ (Study Drug) is given by injection into the vitreous (intravitreous injection), which is clear, jelly-like substance that fills the middle of the eye.
The purpose of this study is to evaluate the safety and effectiveness of Zimura™.
Approximately 120 men and women, 18-50 years old, in 35 centers from: Belgium, Canada, France, Germany Israel, Italy, Singapore, Spain, Switzerland, United States of America and the United Kingdom will take part. The study will last approximately 18 months. A total of 36 injections will be given per subject.
Not all subjects will receive Zimura™. It’s assigned by chance (like the flip of a coin) to one of these groups:
Zimura™
Sham (Placebo)Sham (Placebo) is an empty syringe with no needle. A trained ophthalmologist (doctor who specialises in medical and surgical eye disease) will place the syringe on the conjunctiva (mucous membrane that covers front of the eye and lines the inside of the eyelids) to simulate the pressure on the eyeball of an injection. The sham’s used to prevent bias in study results. The chance of receiving Zimura™ or Sham (Placebo) is 50% for each group.
Neither subject nor study doctor will know which study drug group they are in.
The study consists of:
A 14-day Screening Phase, tests taken for eligibility to this study.
Induction Phase: Subjects who pass eligibility on Day 1 (randomization) will receive study drug/Sham 14 days apart on Day 1, Month 1 and Month 2:
o D0: Zimura™ 2 mg/eye or Sham
o D14: Zimura™ 2 mg/eye or ShamThree days post injection a telephone call is made to ensure they are well and have no signs or symptoms of retinal detachment or endophthalmitis (inflammation of the interior of the eye)
Maintenance Phase: Following Month 2, subjects will have study visits every month for a total of 18 months. They will have the following study drug dosage monthly, beginning at month 3:
o Zimura™ 4 mg/eye (administered as two injections of Zimura™ 2 mg) or Sham (two sham injections)
Three days post the injection a telephone call is made to ensure they are well and have no signs or symptoms of retinal detachment or endophthalmitis (inflammation of the interior of the eye)
A final visit (follow up) at Month 18. If they withdraw early from the study, they will have a follow up visit at the time they withdraw.
All injections will be performed on one eye only. This is the “study eye.” If the other eye (the “fellow eye”) requires treatment during the study, they will be permitted to be treated with approved therapy throughout the entire study.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
18/SC/0135
Date of REC Opinion
21 May 2018
REC opinion
Further Information Favourable Opinion