Celeste

• Research type

  Research Study

• Full title

  A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss

• IRAS ID

  305684

• Contact name

  Susan Downes

• Contact email

  susan.downes@ouh.nhs.uk

• Sponsor organisation

  ProQR Therapeutics IV B.V.

• Eudract number

  2021-002728-19

• Clinicaltrials.gov Identifier

  NCT05176717

• Duration of Study in the UK

  3 years, 5 months, 12 days

• Research summary

  This is a study designed to study the effectiveness, safety and tolerability of QR-421a in different dose levels in the participants with early to moderate vision loss due to mutations in exon 13 USH2A gene. QR-421a is designed to specifically target mutations in exon 13 of USH2A gene which cause the Retinitis Pigmentosa (RP) and to overcome the genetic defect in the DNA and to restore this important protein, which may stop some effects of RP, or possibly reverse some effects.
  This study will compare the effects of QR-421a with the effects of a sham procedure. The study drug will be injected into the participant’s eye (Intravitreal (IVT) Injection). Approximately 120 patients are planned to be recruited for this study worldwide. Participants will be 12 years or older.

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  21/EM/0279

• Date of REC Opinion

  20 Jan 2022

• REC opinion

  Further Information Favourable Opinion