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Gene Therapy Trial Report

Summary

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)


NCTID NCT02599922 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Achromatopsia
Disease Ontology Term DOID:0110008
Compound Name AGTC-401
Compound Description rAAV2tYF-PR1.7-hCNGB3
Sponsor Beacon Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 32 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CNGB3
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Target Tissue/Cell Cone cells
Delivery System Viral transduction
Vector Type AAV2tYF
Editor Type none
Dose 1 2E11 vg/ml
Dose 2 4E10 vg/ml
Dose 3 1.2E11 vg/ml
Dose 4 3.6E11 vg/ml
Dose 5 1.1E12 vg/ml; Dose 6: 3.2E12

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2015-11-05
Completion Date 2026-07
Last Update 2022-07-22

Participation Criteria


Eligible Age >=4 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria include: 1. Male or female subjects with documented mutations in both alleles of the CNGB3 gene; 2. Retinal disease consistent with a clinical diagnosis of achromatopsia; 3. At least 18 years of age for Groups 1, 2, 3, 4, 5 and 6. At least 6 years of age for Group 4a, and 4-8 years of age for Groups 5a and 7; 4. Able to perform tests of visual and retinal function; 5. 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; 6. Acceptable laboratory parameters; 7. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent. Exclusion Criteria include: 1. Best-corrected visual acuity difference between the two eyes of \> 15 ETDRS letters (3 lines); 2. Evidence of degenerative myopia 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 8
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Development discontinued by Sponsor

Resources/Links