First patients dosed in phase 2/3 gene therapy trial for retinitis pigmentosa
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The first patients have been dosed in the phase 2/3 Sirius and Celeste clinical trials investigating QR-421a in patients with USH2A mediated retinitis pigmentosa and Usher syndrome, according to a press release from ProQR Therapeutics.
The double-masked, randomized, sham-controlled, 24-month, multiple-dose studies are investigating the safety and effectiveness of QR-421a. Both studies will enroll patients 12 years and older, who have vision loss attributed to mutations in exon 13 of the USH2A gene, in one of three arms. Patients in the first arm will receive a 180 µg loading dose followed by 60 µg maintenance doses, patients in the second arm will receive a 60 µg loading dose followed by 60 µg maintenance doses, and patients in the third arm will serve as a control.
The Sirius study will enroll 81 patients with advanced vision loss. The primary endpoint is the mean change in best corrected visual acuity at 18 months from baseline compared with the control arm. The Celeste study will enroll 120 patients with early to moderate vision loss. The primary endpoint is the mean change in static perimetry at 12 months from baseline compared with the control arm.
“In a previous clinical study, QR-421a appeared to be well tolerated and demonstrated concordant benefit in multiple measures of vision in treated eyes compared to untreated. Our goal is that the Sirius and Celeste studies further validate QR-421a’s ability to stabilize vision loss in people with USH2A mediated retinitis pigmentosa and Usher syndrome,” Aniz Girach, MD, chief medical officer of ProQR, said in the release.
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