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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03921541 | Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency | PHASE3 | COMPLETED | 32 | — | — | Apr 10, 2019 | Jan 27, 2023 | Nov 19, 2024 | 32 | United States, Austria +5 |
The primary analysis will test the change in the level of plasma arginine between baseline and completion of week 24 assessments. It will compare the change from baseline in plasma arginine between participants treated with pegzilarginase and those treated with placebo.
| Arm | Type | Description |
|---|---|---|
| Pegzilarginase | EXPERIMENTAL | Weekly IV infusions of pegzilarginase plus individualized disease management for 24 weeks |
| Placebo | PLACEBO_COMPARATOR | Weekly IV infusions of placebo plus individualized disease management for 24 weeks |
| Pegzilarginase Long Term Extension | EXPERIMENTAL | After completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study. |
| Name | Type | Description |
|---|---|---|
| Pegzilarginase | DRUG | Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated |
| Placebo | DRUG | Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated |
Inclusion Criteria: Subjects are eligible to be included in the study only if all the following criteria apply: 1. The subject and/or parent/guardian provides written informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF)...