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.

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

Fuchs Focus (ongoing, recruiting)

Phase 1 clinical trial of QR-504a, an investigational RNA therapy for TCF4 mediated Fuchs endothelial corneal dystrophy. This trial recruits individuals that have FECD due to trinucleotide repeat expansion mutations in the TCF4 gene.

Read about the Fuchs Focus trial

Fuchs Focus logomark

Sirius (planned, not yet recruiting)

Phase 2/3 clinical trial of QR-421a, an investigational RNA therapy for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will recruit adults (age 18 and up) that have advanced vision loss due to mutations in exon 13 of the USH2A gene.

Read about QR-421a

Sirius logomark

Celeste (planned, not yet recruiting)

Phase 2/3 clinical trial of QR-421a, an investigational RNA therapy for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial will recruit adults (age 18 and up) that have early to moderate vision loss due to mutations in exon 13 of the USH2A gene.

Read about QR-421a

Celeste 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

Stellar (ongoing, not recruiting)

Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy for USH2A mediated retinitis pigmentosa and Usher syndrome. This trial is recruiting adults (age 18 and up) that have RP due to mutations in exon 13 of the USH2A gene.

Read about the Stellar trial

Stellar logomark

Aurora (ongoing, not recruiting)

Phase 1/2 clinical trial of QR-1123, an investigational RNA therapy for RHO mediated retinitis pigmentosa. This trial includes adults (age 18 and up) that have RP due to the P23H (c.68C>A) mutation in the RHO gene.

Read about the Aurora trial

Aurora logomark