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

Age
99 years or below
Gender
All
participants needed
24
Location
1
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

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 04 Aug 2024. Study ID: 826563
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Study is selecting its participants from a population, or group of people, decided on by the researchers in advance.

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