ProQR completes patient enrollment in phase 2/3 sepofarsen trial
ProQR Therapeutics completed patient enrollment in the phase 2/3 Illuminate trial, investigating sepofarsen for the treatment of Leber congenital amaurosis 10, according to a press release.
The randomized trial includes 36 patients 8 years of age or older. Patients will receive sepofarsen at the target registration dose, a low dose of sepofarsen or sham treatment. The primary endpoint is the mean change in best corrected visual acuity from baseline to month 12 in those receiving the registration dose vs. sham, according to the release.
“In surpassing our enrollment target, we were able to accommodate the broad interest to participate in the trial,” Aniz Girach, MD, chief medical officer of ProQR, said in the release. “This speaks to the fact that there are currently no approved treatments for patients with LCA10. If approved, sepofarsen has the potential to be the first therapy to address this high unmet medical need for patients who would otherwise face blindness.”
Illuminate will support the application for marketing approval of sepofarsen to treat LCA10 due to the p.Cys998X mutation in the CEP290 gene, the release said.
ProQR expects topline results from the study in the first half of 2022.
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