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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 [2 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.

PubMed Articles [5 Associated PubMed Articles listed on BioPortfolio]

Dissociations in Coherence Sensitivity Reveal Atypical Development of Cortical Visual Processing in Congenital Achromatopsia.

While basic visual functions have been described in subjects with congenital achromatopsia (ACHM), little is known about their mid- or high-level cortical visual processing. We compared midlevel corti...

Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.

Congenital achromatopsia (ACHM) is an autosomal recessive disorder in which cone function is absent or severely reduced. Gene therapy in animal models of ACHM have shown restoration of cone function, ...

In vivo imaging of a cone mosaic in a patient with achromatopsia associated with a GNAT2 variant.

The 2 most common causative genes for achromatopsia (ACHM) are CNGA3 and CNGB3; other genes including GNAT2 account for only a small portion of ACHM cases. The cone mosaics in eyes with CNGA3 and CNGB...

Segregation of Incomplete Achromatopsia and Alopecia Due to PDE6H and LPAR6 Variants in a Consanguineous Family from Pakistan.

We report on two brothers with visual impairment, and non-syndromic alopecia in the elder proband. The parents were first-degree Pakistani cousins. Whole exome sequencing of the elder brother and pare...

Social priming of hemispatial neglect affects spatial coding: Evidence from the Simon task.

In the Simon effect (SE), choice reactions are fast if the location of the stimulus and the response correspond when stimulus location is task-irrelevant; therefore, the SE reflects the automatic proc...

Medical and Biotech [MESH] Definitions

None available.

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