Clinical trials

Clinical trials

Our broad pipeline of investigational RNA therapies is evolving, and we are currently conducting several clinical trials. Find below the ongoing and planned clinical trials.

COVID-19 vaccine statement

COVID-19 vaccine statement

There is no contraindication for patients who have taken part or wish to take part in the ProQR sponsored studies to receive the COVID-19 vaccine in combination with antisense oligonucleotides including our investigational RNA therapies. The protocol doesn’t exclude patients who are enrolled in studies from receiving the vaccine or who may have received it in the past or who may receive it in the course of any ProQR sponsored study.

Sirius (ongoing, recruiting)

Phase 2/3 clinical trial of ultevursen (QR-421a), an investigational RNA therapy for USH2A mediated retinitis pigmentosa and Usher syndrome. The trial is recruiting adults and children (age 12 and up) that have vision loss due to mutations in exon 13 of the USH2A gene.

Read about Sirius

Sirius logomark

Brighten (ongoing, recruiting)

Pediatric clinical trial of sepofarsen, an investigational RNA therapy for CEP290 mediated Leber congenital amaurosis. This trial is recruiting children (under 8 years old) that have LCA10 due to the p.Cys998X (c.2991+1655A>G) mutation in the CEP290 gene.

Read about the Brighten trial

Brighten logomark

Illuminate (ongoing, not recruiting)

Phase 2/3 clinical trial of sepofarsen, an investigational RNA therapy for CEP290 mediated Leber congenital amaurosis. This trial is recruiting adults and children (age 8 and up) that have LCA10 due to the p.Cys998X (c.2991+1655A>G) mutation in the CEP290 gene.

Read about the Illuminate trial

Illuminate logomark