A Multiple-Site Phase 1/2 Safety and Efficacy Trial of AGTC 402 a Recombinant Adeno-associated Virus Vector Expressing CNGA3 in Patients with Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene
Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, nystagmus, photoaversion (intolerance to bright light), a small central scotoma, eccentric viewing, and complete or severe loss of color discrimination. The genetic mutations of the disease can be established in the majority of individuals. Approximately 25% are caused by mutations in the cone-specific cyclic nucleotide gated channel alpha subunit (CNGA3) gene.
- Study Identifier: 826563
Recruitment StatusEnrolling By Invitation
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