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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05345171 | Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency | PHASE3 | ACTIVE NOT_RECRUITING | 37 | — | — | Oct 18, 2022 | Mar 1, 2031 | Jun 2, 2026 | 16 | United States, Argentina +8 |
| Arm | Type | Description |
|---|---|---|
| DTX301 | EXPERIMENTAL | Participants receive single peripheral intravenous (IV) infusion of DTX301 in solution. Between Week 36 and Week 64, participants may receive single peripheral IV infusion of placebo. |
| Placebo, Then DTX301 | EXPERIMENTAL | Participants receive single peripheral IV infusion of placebo. Between Week 36 and Week 64, participants receive single peripheral IV infusion of DTX301 in solution. |
| Name | Type | Description |
|---|---|---|
| DTX301 | GENETIC | non-replicating, self-complementary recombinant adeno-associated virus serotype 8 (AAV8) vector |
| Placebo | OTHER | normal saline infusion |
| Oral Corticosteroids | DRUG | Participants who receive DTX301 solution will receive oral corticosteroids. |
| Placebo for oral corticosteroids | DRUG | Participants who receive Placebo will receive placebo corticosteroids to maintain the study blind |
| Sodium Acetate | DRUG | A tracer for the Ureagenesis Rate Test (URT) |
Key Inclusion Criteria: * Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation by enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing...