Baylor College of Medicine

Trial of a Recombinant Adeno-Associated Virus Vector in Patients with Congenital Achromatopsia caused by Mutations in the CNGB3 Gene (H-45520)

Description

Content

A Multiple Site Phase  1/2, Safety and Efficacy Trial of a Recombinant Adeno-Associated Virus Vector Expressing CNGB3 (RAAV2TYF-PR1.7-HCNGB3) In Patients with Congenital Achromatopsia caused by Mutations in the CNGB3 Gene 

Global, non-randomized, open-label, Phase 1/2 dose escalation study. We are looking for male or female both adult and pediatric patients diagnosed with Achromatopsia caused by the CNGB3 gene - (congenital autosomal recessive inherited disease that causes severe vision loss.) Participants will be enrolled into seven groups. Each will receive AGTC-401 in a volume of up to 0.30mL administered by subretinal injection in one eye on a single occasion. Trial duration is five years. Participants will have frequent follow - up visits during the first year after study agent administration. To monitor for delayed adverse events and assess the duration of any visual changes that occur. Continue annual visits for an additional four years after the month-12 visit. A series of diagnostic imaging will be done at visits to assess efficacy 

Contact

Hope Johnson

Phone 1: 713–798–4123

IRB: H-45520

Status:

Active

Created:

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