Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03913143 | A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) | PHASE2 | ACTIVE NOT_RECRUITING | 36 | — | — | Apr 4, 2019 | Mar 1, 2023 | Mar 17, 2022 | 14 | United States, Belgium +7 |
Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
| Arm | Type | Description |
|---|---|---|
| Group 1: Dose 1 sepofarsen (QR-110) | EXPERIMENTAL | Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated |
| Group 2: Dose 2 sepofarsen (QR-110) | ACTIVE_COMPARATOR | Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated |
| Group 3: Sham | SHAM_COMPARATOR | Sham procedure (no experimental drug administered), Day 1, month 3 and every six months there after. After 12 months cross over to active study drug may be initiated |
| Name | Type | Description |
|---|---|---|
| sepofarsen | DRUG | RNA antisense oligonucleotide for intravitreal injection |
| Sham | OTHER | Sham-Procedure (no experimental drug administered) |
Main Inclusion Criteria Relating to Study Initiation: * Male or female, ≥ 8 years of age at Screening with a clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation, based on genotyping analysis at Screening. A historic genot...