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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04411654 | Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | PHASE1 | ACTIVE NOT_RECRUITING | 7 | — | — | Jun 29, 2021 | May 1, 2028 | Apr 1, 2026 | 5 | United States, United Kingdom |
| Arm | Type | Description |
|---|---|---|
| Low Dose | EXPERIMENTAL | - |
| High Dose | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| LY3884961 | GENETIC | Participants will receive a single dose of LY3884961 administered intracisternally. |
| Methylprednisolone | DRUG | Single IV pulse administered as concomitant medication. |
| Sirolimus | DRUG | Loading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication. |
| Prednisone | DRUG | Administered orally as concomitant medication, followed by dose tapering. |
Inclusion Criteria: * Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory. * Clinical diagnosis of GD2 * Parent/legal guardian is capable of providing signed informed consent; including compliance with the requirements and restrictions listed in the info...