Ignite Creation Date:
2024-05-06 @ 1:02 PM
Last Modification Date:
2024-10-26 @ 1:07 PM
Study NCT ID:
NCT03913143
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2022-03-17
First Post:
2019-02-21
Brief Title:
A Study to Evaluate Efficacy Safety Tolerability and Exposure After a Repeat-dose of Sepofarsen QR-110 in LCA10 ILLUMINATE
Sponsor:
ProQR Therapeutics
Organization:
ProQR Therapeutics
Study Overview
Official Title:
Double-masked Randomized Controlled Multiple-dose Study to Evaluate Efficacy Safety Tolerability and Syst Exposure of QR-110 in Lebers Congenital Amaurosis LCA Due to c29911655AG Mutation pCys998X in the CEP290 Gene
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2022-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ILLUMINATE
Brief 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 Lebers Congenital Amaurosis LCA due to the CEP290 pCys998X mutation after 24 months of treatment
Detailed 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 Lebers Congenital Amaurosis LCA due to the CEP290 pCys998X 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 subjects treatment eye the subjects 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 benefitrisk including review of data from all clinical trials and with concurrence of the Medical Monitor
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 subjects treatment eye the subjects 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 benefitrisk including review of data from all clinical trials and with concurrence of the Medical Monitor
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2018-003501-25 | EUDRACT_NUMBER | None | None |