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
Enrolling By Invitation
99 years or below
All
Phase
2
24 participants needed
1 Location
Brief description of study
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.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Achromatopsia
-
Age: 99 years or below
-
Gender: All
Updated on
19 Feb 2024.
Study ID: 826563
If you need help finding a study or have any questions, please contact us at psom-ocr@pobox.upenn.edu
