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Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia

2016-10-18 02:08:21 | BioPortfolio

Summary

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Description

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Subjects will be enrolled sequentially in four groups. Subjects in Groups 1, 2 and 3 will be between 18 and 55 years of age, inclusive, and will receive a lower, middle or higher dose of study agent. Subjects in Group 4 will be between 6 and 55 years of age, inclusive, and will receive the maximum tolerated dose identified in Groups 1, 2 and 3.

Safety will be monitored by evaluation of ocular and non ocular adverse events and hematology and clinical chemistry parameters. Efficacy parameters will include visual acuity, light discomfort testing, color vision, static visual field, ERG, adaptive optics retinal imaging and OCT.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Achromatopsia

Intervention

AGTC-402

Location

VitreoRetinal Associates
Gainesville
Florida
United States
32607

Status

Not yet recruiting

Source

Applied Genetic Technologies Corp

Results (where available)

View Results

Links

Published on BioPortfolio: 2016-10-18T02:08:21-0400

Clinical Trials [3 Associated Clinical Trials listed on BioPortfolio]

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of rAAV2tYF-PR1.7-hCNGB3 administered to one eye by subretinal injection in individuals with achromato...

Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients With CNGA3-linked Achromatopsia

The purpose of this study is to proof the safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia.

Gene Therapy for Achromatopsia (CNGB3)

Achromatopsia is a recessively inherited condition characterised by a lack of cone photoreceptor function resulting in impairment of colour vision and visual acuity, central scotoma often ...

PubMed Articles [7 Associated PubMed Articles listed on BioPortfolio]

A Quantitative and Qualitative Exploration of Photoaversion in Achromatopsia.

Photoaversion (PA) is a disabling and ubiquitous feature of achromatopsia (ACHM). We aimed to help define the characteristics of this important symptom, and present the first published assessment of i...

Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia.

Adeno-associated virus (AAV) vectors are important gene delivery tools for the treatment of many recessively inherited retinal diseases. For example, a wild-type (WT) AAV5 vector can deliver a full-le...

Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.

Applying CNGA3 gene augmentation therapy to cure a novel causative mutation underlying achromatopsia (ACHM) in sheep.

Spatially Resolved Spectral Sensitivities as a Potential Read-out Parameter in Clinical Gene Therapeutic Trials.

Spatially resolved functional assessment of rods and cones under photopic and scotopic conditions is desirable to evaluate the treatment outcome of gene therapeutic applications in inherited retinal d...

The Visual Agnosias and Related Disorders.

There are many disorders of higher visual processing that result from damage to specific areas of the cerebral cortex that have a specific role in processing certain aspects (modalities) of vision. Th...

Medical and Biotech [MESH] Definitions

None available.

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