Clinical Trials
Sanofi
A Phase 1/2 study to evaluate the safety, tolerability and efficacy of one-time intravitreal dose of SAR446597 in participants with geographic atrophy secondary to age related macular degeneration.
Overview
This research project is testing a new experimental gene therapy for Geographic Atrophy (GA). The new potential gene therapy is called SAR446597. Gene therapy is a form of treatment that involves adding or otherwise changing genetic material in a patient’s cells to either help change abnormal genes or instruct cells to make more of a good (beneficial) protein.
Genes are made up of DNA (deoxyribonucleic acid) and provide instructions to your body on how to function. SAR446597 is intended to help decrease the activity of the parts of the immune system, called complement cascade, that are believed to cause GA in your study eye (the eye selected to receive treatment in this study).
SAR446597 contains an adeno associated virus (AAV) vector. An AAV vector is a virus that has been modified in the laboratory so it cannot reproduce or cause an infection once it is in your body. The virus helps ‘carry’ the genes into your cells to provide ‘instructions’ for retinal cells (the cells in the eye that help you see) to block some parts of the complement cascade in your study eye.
SAR446597 will be administered by injection into the eye (called intravitreal injection). The main objective of this study is to understand the safety of SAR446597 (the side effects participants may have after getting SAR446597 and how well participants tolerate any side effects).
This study will help the study Sponsor find out how well participants with GA respond to treatment with SAR446597 and whether the progression of the disease can be slowed down after one injection of SAR446597. As part of the study, suitable participants will be screened to determine their eligibility for the trial. If eligible, patients will be randomized to one of 3 groups:
- Two groups will receive 1 of 2 different doses of SAR446597.
- The third group will receive a sham or fake injection.
There is a 3 in 4 (75%) chance of receiving 1 of the 2 doses of SAR446597 and a 1 in 4 (25%) chance of receiving a sham injection. The study duration is 2 years, with the option of extended follow up for a further 3 years. For further information, please contact Cerulea on 99298076 or info@ceruleaclinicaltrials.org.au
- Principal Investigator
Professor Robyn Guymer
This research project is testing a new experimental gene therapy for Geographic Atrophy (GA). The new potential gene therapy is called SAR446597. Gene therapy is a form of treatment that involves adding or otherwise changing genetic material in a patient’s cells to either help change abnormal genes or instruct cells to make more of a good (beneficial) protein.
Genes are made up of DNA (deoxyribonucleic acid) and provide instructions to your body on how to function. SAR446597 is intended to help decrease the activity of the parts of the immune system, called complement cascade, that are believed to cause GA in your study eye (the eye selected to receive treatment in this study).
SAR446597 contains an adeno associated virus (AAV) vector. An AAV vector is a virus that has been modified in the laboratory so it cannot reproduce or cause an infection once it is in your body. The virus helps ‘carry’ the genes into your cells to provide ‘instructions’ for retinal cells (the cells in the eye that help you see) to block some parts of the complement cascade in your study eye.
SAR446597 will be administered by injection into the eye (called intravitreal injection). The main objective of this study is to understand the safety of SAR446597 (the side effects participants may have after getting SAR446597 and how well participants tolerate any side effects).
This study will help the study Sponsor find out how well participants with GA respond to treatment with SAR446597 and whether the progression of the disease can be slowed down after one injection of SAR446597. As part of the study, suitable participants will be screened to determine their eligibility for the trial. If eligible, patients will be randomized to one of 3 groups:
- Two groups will receive 1 of 2 different doses of SAR446597.
- The third group will receive a sham or fake injection.
There is a 3 in 4 (75%) chance of receiving 1 of the 2 doses of SAR446597 and a 1 in 4 (25%) chance of receiving a sham injection. The study duration is 2 years, with the option of extended follow up for a further 3 years. For further information, please contact Cerulea on 99298076 or info@ceruleaclinicaltrials.org.au
- Principal Investigator
Learn more
View this study at ClinicalTrials.gov
This database listing provides more detailed information about this study.