Gene Therapy for Achromatopsia (CNGB3)

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-16

Study Completion Date

2019-10-25

Brief Summary

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A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

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Detailed Description

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CNGB3 retinal gene therapy for patients with achromatopsia

Conditions

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Achromatopsia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Biological-Low dose AAV - CNGB3

Subretinal administration of a single low dose of AAV - CNGB3

Group Type EXPERIMENTAL

AAV - CNGB3

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV-CNGB3

Biological-Medium dose AAV - CNGB3

Subretinal administration of a single intermediate dose of AAV - CNGB3

Group Type EXPERIMENTAL

AAV - CNGB3

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV-CNGB3

Biological-High dose AAV - CNGB3

Subretinal administration of a single high dose of AAV - CNGB3

Group Type EXPERIMENTAL

AAV - CNGB3

Intervention Type BIOLOGICAL

Comparison of different dosages of AAV-CNGB3

Interventions

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AAV - CNGB3

Comparison of different dosages of AAV-CNGB3

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Are aged 3 years or older
* Have achromatopsia confirmed by a retinal specialist (CI or PI)

Exclusion Criteria

* Are females who are pregnant or breastfeeding
* Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
* Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial

Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No