This is a clinical study to evaluate the safety, tolerability and efficacy of CTx001, administered via a single subretinal injection, for GA (secondary to AMD). Safety and efficacy will be measured at regular intervals for 2 years after which long-term safety will be assessed annually for up to 5 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
75
Subretinal administration of CTx001
Midwest Eye Institute
Carmel, Indiana, United States
RECRUITINGSierra Eye Associates
Reno, Nevada, United States
RECRUITINGRetina Foundation of the Southwest
Dallas, Texas, United States
RECRUITINGTo monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels
Incidence and severity of ocular and non-ocular adverse events (AE)s and serious AEs (SAEs) up to Week 52 (Year 1)
Time frame: From dosage to Week 52
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
Change from baseline in the rate of total ellipsoid zone (EZ) attenuation as measured by spectral domain-optical coherence tomography (SD-OCT) at Week 52 (Year 1)
Time frame: From dosage to Year 1
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
Rate of change from baseline in sensitivity as measured by microperimetry at Week 52 (Year 1)
Time frame: From dosage to Year 1
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
Change from baseline in low luminance visual acuity (LLVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart and neutral density filter at Week 52 (Year 1)
Time frame: From dosage to Year 1
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
Change from baseline in the rate of enlargement of lesion area of geographic atrophy as measured by Fundus Auto Fluorescence (FAF) at Week 104 (Year 2)
Time frame: From dosage to Year 2
To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
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Gundersen Health System
La Crosse, Wisconsin, United States
RECRUITINGChange from baseline in best corrected visual acuity (BCVA) as measured by Early Treatment Diabetic Retinopathy Study (EDTRS) chart at Week 104 (Year 2)
Time frame: From dosage to Year 2
To monitor the long-term safety and tolerability of a single administration of CTx001 at 3 dose levels
* Incidence and severity of ocular and non-ocular AEs and SAEs up to Week 260 (Year 5) * Incidence and extent of retinal pigmentary changes up to Week 260 (Year 5)
Time frame: From dosage up to Week 260 (Year 5)
To assess immunogenicity to adeno-associated virus Serotype 2 (AAV2)-vector and mini-CR1 transgene product
Measurement of systemic antibody levels to AAV2-vector and mini-CR1 transgene up to Week 260 (Year 5)
Time frame: From dosage up to Week 260 (Year 5)