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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03780257 | Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | PHASE1 | COMPLETED | 20 | — | — | Mar 6, 2019 | Oct 14, 2021 | Apr 20, 2022 | 7 | United States, Canada +1 |
Incidence and severity of ocular AEs
| Arm | Type | Description |
|---|---|---|
| QR-421a | EXPERIMENTAL | Single dose administration |
| Sham-procedure (dose cohort 1&2 only) | SHAM_COMPARATOR | Sham-procedure (no experimental drug administered) |
| Name | Type | Description |
|---|---|---|
| QR-421a | DRUG | RNA antisense oligonucleotide for intravitreal injection |
| Sham-procedure (dose cohort 1&2 only) | OTHER | Sham-procedure (no experimental drug administered) |
Inclusion Criteria: 1. Male or female, ≥ 18 years of age. 2. Clinical presentation consistent with RP with Usher syndrome type 2 or non-syndromic RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations. 3. A molecular diagnosis of homozygosity or compound heterozygosity for 1 or mor...