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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04483440 | Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia | PHASE1 | ACTIVE NOT_RECRUITING | 13 | — | — | Jun 2, 2020 | Aug 31, 2027 | May 22, 2025 | 2 | United States |
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
| Arm | Type | Description |
|---|---|---|
| 4D-110 Dose 1 | EXPERIMENTAL | 4D-110 IVT injection |
| 4D-110 Dose 2 | EXPERIMENTAL | 4D-110 IVT injection |
| 4D-110 Dose 3 | EXPERIMENTAL | 4D-110 IVT injection |
| Name | Type | Description |
|---|---|---|
| 4D-110 | BIOLOGICAL | 4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. |
Key Inclusion Criteria: * Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing * Both eyes must have ≥ 34 ETDRS letters (\~20/200) Key Exclusion Criteria: * Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye *...