An Open-Label Extension Study to Evaluate the Safety Tolerability Efficacy and Pharmacokinetics of QR-110 in Subjects with Leber's Congenital Amaurosis (LCA) due to the C.2991+1655aG Mutation (P.Cys998x) in the Cep290 Gene

This Phase Ib/II clinical trial has a primary objective of evaluating the safety and tolerability of the experimental drug, QR-110, and secondary objectives of evaluating long-term and sustained efficacy of QR-110 administered by IVT injection, as assessed by functional and structural outcome measures, evaluating changes in Patient Reported Outcome measures in subjects treated with QR-110 and evaluating long-term serum pharmacokinetics (PK) of the study drug. An exploratory outcome will be to evaluate additional efficacy endpoints that may be collected at the investigator's discretion. All subjects will have completed the preceding study, have been diagnosed with Leber's Congenital Amaurosis (LCA) and have at least one copy of the p.Cys998X mutation in the CEP290 gene. LCA is a severe inherited retinal degenerative disease resulting in blindness, often in early childhood. There are currently no approved treatments for LCA caused by this mutation. QR-110 is an antisense oligonucleotide (AON) that will be administered via intravitreal (IVT) injection at the 80ug dose level with a 6-month dosing interval. Treatment of the contralateral eye may be initiated, with a 160ug loading dose followed by 80ug maintenance doses, with 6-month dosing intervals. Subjects will be assessed for safety and tolerability at follow up visits (3-month intervals). UPenn will have a maximum of 4 subjects enrolled who have completed Study PQ-110-001. Blood and serum tests, ophthalmic and physical evaluations, visual function tests, ocular imaging, mobility-course assessments and visual function questionnaires will be used to assess the safety of the subjects, the serum PK, and efficacy of the study drug in subjects.

You may be eligible for this study if you meet the following criteria:

• Conditions:
  LCA,Leber's Congenital Amaurosis
• Age: - 99 Years
• Gender: All