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

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Disease Information

Descriptive Information
Brief Title † Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia
Official Title † 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
Brief Summary 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 achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
Detailed Description 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 achromatopsia caused by mutations in the CNGB3 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 at least 18 years of age and will receive a lower, middle or higher dose of study agent. Subjects in Group 4 will be at least 6 years of age 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 Phase Phase 1/Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Adverse events
Secondary Outcome Measure † Visual acuity
Condition † Achromatopsia
Intervention † BiologicalrAAV2tYF-PR1.7-hCNGB3
Study Arms / Comparison Groups Lower dose rAAV2tYF-PR1/7-hCNGB3 will be administered at the lowest of three planned dose levels. Middle dose rAAV2tYF-PR1/7-hCNGB3 will be administered at the middle of three planned dose levels. Higher dose rAAV2tYF-PR1/7-hCNGB3 will be administered at the highest of three planned dose levels. Maximum tolerated dose rAAV2tYF-PR1/7-hCNGB3 will be administered at the maximum tolerated dose identified from Groups 1, 2 and 3.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Biological
Estimated Enrollment † 24
Start Date † February 2016
Completion Date December 2022
Primary Completion Date December 2018
Eligibility Criteria † Inclusion Criteria include: 1. Retinal disease consistent with a diagnosis of achromatopsia and documented mutations in both alleles of the CNGB3 gene; 2. At least 18 years of age for Groups 1, 2 and 3 and at least 6 years of age for Group 4; 3. Able to perform tests of visual and retinal function; 4. Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit; 5. Acceptable laboratory parameters; 6. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent. Exclusion Criteria include: 1. Refractive error of ≥ -8.00 diopters (spherical equivalent) of myopia in the study eye; 2. Evidence of degenerative myopia regardless of the refractive error in the study eye; 3. Pre-existing eye conditions that would contribute to vision loss in either eye or increase the risk of subretinal injection in the study eye.
Gender All
Ages 6 Years - N/A
Accepts Healthy Volunteers No
Contacts †† Jill Dolgin, PharmD, 617-843-5781, jdolgin@agtc.com
Location Countries † United States
Administrative Information
NCT ID † NCT02599922
Organization ID AGTC_CNGB3-001
Secondary IDs †† R24EY022023
Responsible Party Sponsor
Study Sponsor † Applied Genetic Technologies Corp
Collaborators †† National Eye Institute (NEI)
Investigators † Study Director: Mike Goldstein, MD, Applied Genetics Technologies Corporation
Information Provided By
Verification Date March 2017
First Received Date † November 5, 2015
Last Updated Date March 7, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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