Stargardt Disease 1 Clinical Trial
Official title:
Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)
The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1). JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene.
This clinical study is a single-center, non-randomized, non-blinded, self-controlled, dose-escalation clinical study, aiming to evaluate the safety, tolerability, and preliminary effectiveness of JWK006 injection in the treatment of approximately 12 patients(aged 10 to 18 years) with Stargardt Disease(STGD1),genotyped with ABCA4 gene mutations. In this study, a "3+3" dose escalation trial was adopted, with 3 doses (low dose, medium dose, and high dose) set up, and 9 subjects were planned to be enrolled, 3 in each dose group. Following the principle of dose escalation, starting from the low-dose treatment group, one subject was enrolled in each dose group. In order to fully protect the safety of subjects, it is required to adopt an orderly enrollment method within each dose group, with one subject enrolled at a time. No obvious toxic or side effects are observed 28 days after the completion of the administration. The next subject in the group. At the same time, 28 days after the last subject in each dose group is administered, the researchers and others will jointly evaluate the safety results of the dose group before deciding whether to enter the next dose group. ;
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