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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05139316 | A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) | PHASE3 | COMPLETED | 49 | — | — | Nov 8, 2021 | Feb 20, 2026 | Mar 25, 2026 | 20 | United States, Brazil +7 |
| Arm | Type | Description |
|---|---|---|
| DTX401, Then Placebo | EXPERIMENTAL | Participants receive single peripheral intravenous (IV) infusion of DTX401 in solution. At week 48 participants receive single peripheral IV infusion of Placebo. |
| Placebo, Then DTX401 | PLACEBO_COMPARATOR | Participants receive single peripheral IV infusion of Placebo. At week 48 eligible participants receive single peripheral IV infusion of DTX401 solution. |
| DTX401 (Japan Only) | EXPERIMENTAL | Participants receive single peripheral intravenous (IV)infusion of DTX401 in solution. |
| Name | Type | Description |
|---|---|---|
| DTX401 | GENETIC | nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8) |
| Placebo | OTHER | Normal Saline infusion |
| Oral prednisolone | DRUG | Participants who receive DTX401 solution will receive oral prednisolone |
| Placebo for oral prednisolone | DRUG | Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind |
Key Inclusion Criteria: * Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy * Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status. *...