Guide To Diagnosing And Treating Leber Congenital Amaurosis

Clinical Trials

Clinical trials are available to individuals affected by Leber congenital amaurosis who are not eligible to have the only FDA-cleared gene therapy treatment. A clinical trial involves the administration of specific interventions such as medical products, behavioral changes, drugs, procedures, and use of devices to treat a disease or condition. The new method can be compared to a placebo, no intervention, or an existing standard treatment method. Investigators who conduct clinical trials measure certain outcomes in the patients to determine the efficacy and safety of their intervention. Some clinical trials are done in phases defined by the FDA. Clinical trials for patients with Leber congenital amaurosis caused by the p.Cys998X mutation or the c.2991+1655A>G mutation in the CEP290 gene has been cleared by the FDA, and they are available for eligible individuals. Another clinical trial is available for individuals who have LCA caused by the GUCY2D mutation. Individuals who are not eligible to undergo a clinical trial may be able to obtain experimental devices or drugs through FDA-expanded access.

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