FindTrial
Sign In

QR-110

DRUG2 trials

Sponsors

Laboratoires Thea

Conditions

BlindnessEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalLeber Congenital AmaurosisLeber Congenital Amaurosis 10Leber's Congenital AmaurosisNeurologic Manifestations

Phase 1

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
CompletedNCT03140969
Laboratoires TheaLeber's Congenital Amaurosis
Start: 2017-10-16End: 2019-10-02Updated: 2024-10-15
Extension Study to Study PQ-110-001 (NCT03140969)
TerminatedNCT03913130
Laboratoires TheaBlindness, Eye Diseases, Eye Diseases, Hereditary +7
Start: 2019-05-13End: 2022-10-03Updated: 2024-10-15

Related Papers

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Nature Medicine2022-04-04121 citations
Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report.
2021-04-0149 citations
Antisense oligonucleotide treatment in CEP290‐related leber congenital amaurosis
Acta Ophthalmologica2019-12-01
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect.
2018-12-17174 citations