The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
75
Single subretinal administration
Retina Consultants of Texas
Bellaire, Texas, United States
RECRUITINGRetina Foundation of the Southwest
Dallas, Texas, United States
RECRUITINGMoorfields Eye Hospital NHS Foundation Trust
London, England, United Kingdom
RECRUITINGTo measure the number and severity of treatment related adverse events following treatment with AAVB-039
Time frame: 0-60 months
Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039
Time frame: 0-60 months
Change in ellipsoid zone (EZ) loss following treatment with AAVB-039
Time frame: 0-60 months
Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
Time frame: 0-60 months
Change in visual acuity following treatment with AAVB-039
Time frame: 0-60 months
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The Retina Clinic
London, England, United Kingdom
RECRUITING