3913143
Last Update Posted: 2022-03-17
Recruiting has ended
All Genders accepted | 8 Years + |
36 Estimated Participants | No Expanded Access |
Interventional Study | Does not accept healthy volunteers |
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
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
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.
Eligibility
Relevant conditions:
Leber Congenital Amaurosis 10
Blindness
Leber Congenital Amaurosis
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Eye Diseases
Eye Diseases, Hereditary
Eye Disorders Congenital
Retinal Disease
If you aren't sure if you meet the criteria above speak to your healthcare professional. Criteria may be updated but not reflected here, do not hesitate to contact the trial if you think are close to fitting criteria.
locations
Contact Information
Overall Contact
No valid contacts available
Data sourced from ClinicalTrials.gov