Extended Data Table 1 Study drug administration in trial PQ-110-001

From: Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

  1. Based on the benefit–risk from the first two dose levels, the decision was made, in consultation with the data monitoring committee, to not initiate the 500-µg/270-µg dose group. A dosing interval of at least 6 months for the 160-µg/80-µg dose cohort was also applied, and the 320-µg/160-µg cohort was discontinued. NA, dose not administered; OD, right eye; OS, left eye.
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