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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06155500 | Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) | PHASE1 | ACTIVE NOT_RECRUITING | 4 | — | — | Apr 16, 2024 | Jan 11, 2039 | Oct 3, 2025 | 3 | United States |
Number of participants with delayed adverse events including new secondary malignancies, new incidence or exacerbation of a prior autoimmune disorder, new incidence or exacerbation of a prior rheumatologic disorder, new hematologic disorder, and other adverse events considered to be related to OTQ923 therapy.
| Arm | Type | Description |
|---|---|---|
| OTQ923 | EXPERIMENTAL | Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment. |
| Name | Type | Description |
|---|---|---|
| OTQ923 | BIOLOGICAL | There is no treatment allocation. Patients administered were OTQ923 while enrolled on the treatment protocol CADPT03A12101 (NCT04443907) |
Inclusion Criteria: 1. Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101). 2. Patients must provide informed consent prior to their entry into this study. Exclusion Criteria: 1\. Completion of less than 1 year of safety follow-up in the treatment...