Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as pariglasgene brecaparvovec, AAV8G6PC
DTX401 · 2 trials · 2 indications
The least square (LS) mean and standard error (SE) are based on mixed-model for repeated measures (MMRM) analysis.
An AE is defined as any untoward medical occurrence, regardless of its causal relationship to study product. An SAE is defined as any event that: results in death; is immediately life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; or an important medical event, in the opinion of the investigator. The relationship to study drug was categorized as unrelated, possible, probable or definite. A DLT is defined as any AE/SAE ≥ Grade 3 that is considered by the Investigator and/or Sponsor to be related to DTX401, based on the Nation Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 or later version. Per protocol, SAEs that occurred \> 30 days after EOS or Early withdrawal visit, did not need to be reported unless Investigator considered them related to study product.
| Arm | Type | Description |
|---|---|---|
| Placebo Followed by DTX401 | EXPERIMENTAL | Participants received a single peripheral intravenous (IV) infusion of placebo on Day 1 along with a placebo-to-match (PTM) of the prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of 1.0 × 10\^13 genome copies per kilogram \[GC/kg\]) of DTX401 along with a prophylactic steroid regimen of prednisolone. |
| DTX401 Followed by Placebo | PLACEBO_COMPARATOR | Participants received a single peripheral IV infusion of 1.0 × 10\^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of placebo with a PTM of the prophylactic steroid regimen of prednisolone. |
| DTX401 (Japan Only) | EXPERIMENTAL | Participants received a single peripheral IV infusion of 1.0 × 10\^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. |
| DTX401 Cohort 1 | EXPERIMENTAL | Dose 1 (2.0 × 10\^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after alanine aminotransferase \[ALT\] elevation) |
| DTX401 Cohort 2 | EXPERIMENTAL | Dose 2 (6.0 × 10\^12 GC/kg) with a reactive steroid regimen (6 weeks, at a starting dose of 40 mg/day, after ALT elevation) |
| DTX401 Cohort 3 | EXPERIMENTAL | Dose 2 (6.0 × 10\^12 GC/kg) with an optimized reactive steroid regimen (7 weeks, at a starting dose of 60 mg/day, after ALT elevation) |
| DTX401 Cohort 4 | EXPERIMENTAL | Dose 2 (6.0 × 10\^12 GC/kg) with a prophylactic steroid regimen (8 weeks, at a starting dose of 60 mg/day, starting on Day 1) |
| 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 |
| steroid regimen | DRUG | prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation |
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. *...
DTX401 is an investigational gene therapy being developed by Ultragenyx Pharmaceutical Inc. for glycogen storage disease type Ia, also known as GSD1. It is designed to deliver a functional copy of the glucose-6-phosphatase gene to liver cells. DTX401 has been studied in clinical trials and is not yet approved for commercial use.
DTX401 is being developed for the treatment of glycogen storage disease type Ia (GSD1), a rare inherited metabolic disorder. The goal is to address the underlying enzyme deficiency by providing a working copy of the G6PC gene. It remains investigational and has not been approved for any indication.
DTX401 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare and ultra-rare diseases. The company trades on the Nasdaq under the ticker symbol RARE. Ultragenyx is responsible for the clinical development program of DTX401.
DTX401 has been evaluated in a Phase 1 clinical trial and a Phase 3 clinical trial. The Phase 1 trial is completed, and the Phase 3 trial is also completed. DTX401 is not approved by the FDA and remains an investigational therapy.
DTX401 has been studied in two clinical trials: NCT03517085, a Phase 1 safety and dose-finding study in adults with GSD1, and NCT05139316, a Phase 3 study in patients with glycogen storage disease type Ia. Both trials are completed. The Phase 3 trial enrolled 52 patients across multiple countries.