Antisense oligonucleotide treatment in CEP290‐related leber congenital amaurosis

Purpose To assess safety and efficacy of the antisense oligonucleotide (QR‐110) in LCA10 patients carrying the c.2991+1655A>G allele in the ciliopathy gene CEP290. Methods In a three‐center open‐label clinical trial (NCT03140969), subjects received intravitreal injections of QR‐110 in the worse s...

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Bibliographic Details
Published in:Acta ophthalmologica (Oxford, England) England), 2019-12, Vol.97 (S263), p.n/a
Main Authors: Leroy, Bart P, V Cideciyan, Artur, Jacobson, Samuel G, V Drack, Arlene, Ho, Allen C, Garafalo, Alexandra, Roman, Alejandro J, Schwartz, Mike, Biasutto, Patricia, Wit, Wilma, Cheetham, Michael E, Adamson, Peter S, Rodham, David, Girach, Aniz, De Zaeytijd, Julie, Van Cauwenbergh, Caroline, Russell, Stephen R
Format: Article
Language:English
Subjects:
Acuity
Antisense oligonucleotides
Cataracts
Durability
Edema
Injection
Leber congenital amaurosis
Patients
Safety
Statistical analysis
Visual acuity
Visual perception
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Summary:Purpose To assess safety and efficacy of the antisense oligonucleotide (QR‐110) in LCA10 patients carrying the c.2991+1655A>G allele in the ciliopathy gene CEP290. Methods In a three‐center open‐label clinical trial (NCT03140969), subjects received intravitreal injections of QR‐110 in the worse seeing eye every three months. There were two combinations of loading/maintenance doses: 160/80 μg or 320/160 μg. Systemic and ocular safety was evaluated with standard methods. Efficacy was evaluated with visual acuity (VA), oculomotor control and instability (OCI), two‐colour full‐field stimulus test (FST), and mobility course testing among others. In a patient with an exceptional response, additional specialized tests of visual function were performed. Study was planned for 4 injections over one year but based on exceptional results in one patient an interim analysis was performed with the data cutoff date of Aug 2018. Results Ten patients received at least 1 and up to 4 injections, and were followed for at least 1 and up to 9 months. At 3 months after the first (loading) injection, treated eyes were significantly improved compared to baseline by an average of 0.67 log (7 lines) in VA (P=0.022), 0.62 log in red FST (P=1E‐6) and 0.81 log in blue FST (P
ISSN:1755-375X
1755-3768
DOI:10.1111/j.1755-3768.2019.8296