Clinical Trials Logo

Clinical Trial Summary

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment


Clinical Trial Description

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment. At study start subjects will be randomized to one of 3 treatment groups with either active study drug or sham treatment. Sepofarsen (QR-110) will be administered via intravitreal (IVT) injection into the subject's treatment eye (the subject's worse eye). Subjects in the sham-procedure group will undergo a procedure that will closely mimic the active injection. After each dosing subjects will be assessed for safety and tolerability at follow up visits. After the first eye has been treated for at least 12 months, treatment of the contralateral eye and cross-over of subjects assigned to sham procedure may be initiated in eligible eyes (in a masked manner) based on assessment of benefit/risk (including review of data from all clinical trials), and with concurrence of the Medical Monitor. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03913143
Study type Interventional
Source ProQR Therapeutics
Contact
Status Active, not recruiting
Phase Phase 2/Phase 3
Start date April 4, 2019
Completion date March 2023

See also
  Status Clinical Trial Phase
Recruiting NCT05414994 - Assessment of the Ocular Microbiome in Health and Disease
Active, not recruiting NCT04271709 - Manhattan Vision Screening and Follow-Up Study (NYC-SIGHT) N/A
Completed NCT02811692 - Study for Collection of Aflibercept Data in Routine Practice
Recruiting NCT06045299 - Evaluation of the Efficacy and Safety of LNZ101 and LNZ100 for the Presbyopia Phase 3
Recruiting NCT01521065 - An Open-label Study to Evaluate the Clinical and Economic Benefits of I-Ray in Patients With Choroidal Neovascularization Secondary to Age-related Macular Degeneration Phase 2
Completed NCT04799704 - Tear Film SARS-nCoV-2 Detection in Symptomatic and Pauci-symptomatic Patients.
Recruiting NCT05876689 - Swept Source OCT Imaging With the DREAM VG-OCT
Active, not recruiting NCT04123626 - A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene Phase 1/Phase 2
Recruiting NCT04150432 - Correlation Between Exhaled Propofol Concentration With Plasma Concentration in Children N/A
Not yet recruiting NCT05550740 - Repeated Low-Level Red-Light Therapy for Shortening Axial Length N/A
Completed NCT02332343 - Sparing of the Fovea in Geographic Atrophy Progression N/A
Completed NCT01727973 - Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' Orbitopathy Phase 1/Phase 2
Terminated NCT01225146 - Study of 2.0 mg Ranibizumab in Subjects With Ischemic Central Retinal Vein Occlusion (RAVE2) Phase 1
Completed NCT00333203 - Next Generation Ophthalmic Irrigating Solution Posterior Segment Study Phase 3
Not yet recruiting NCT05565547 - Multimodal Equipment for Teleophthalmology Assessment (META)
Recruiting NCT05158699 - Effectiveness of Periocular Drug Injection in CATaract Surgery Phase 3
Recruiting NCT06112340 - A Phase 2b Extension Study of Two Doses of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED) Phase 2/Phase 3
Completed NCT05211089 - Manual Versus Automated Choroidal Thickness Measurements Using Swept-source Anterior Segment OCT
Not yet recruiting NCT06070467 - Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases
Completed NCT02946879 - Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)