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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02629393 | Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A | PHASE2 | COMPLETED | 5 | — | — | May 1, 2016 | Oct 1, 2022 | Oct 17, 2023 | 11 | United States, Israel +4 |
| NCT02047461 | Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP | PHASE2 | COMPLETED | 8 | — | — | Apr 1, 2014 | Oct 1, 2022 | Oct 17, 2023 | 6 | United States, Australia +3 |
Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.
Treatment Emergent Serious Adverse Events
| Arm | Type | Description |
|---|---|---|
| ORGN001 (formerly ALXN1101) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ORGN001 (formerly ALXN1101) | DRUG | - |
Patients must meet all of the following inclusion criteria to be considered for enrollment in this study: 1. Male or female neonatal patient (1 to 28 days of age \[inclusive\] at the time of ORGN001 administration, with day 1 of age corresponding to the day of birth) or infant (29 days to \<2 years...