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DTX301 · 1 trial · 1 indication
| 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...
DTX301 is an investigational gene therapy being developed for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare inherited metabolic disorder. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
DTX301 targets the OTC gene, which encodes the enzyme ornithine transcarbamylase. The therapy is designed to deliver a functional copy of this gene to address the underlying genetic cause of OTC deficiency.
DTX301 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol RARE.
DTX301 is currently in Phase 3 clinical development. It is an investigational therapy and has not yet been approved by the FDA or other regulatory agencies.
DTX301 is being evaluated in a Phase 3 clinical trial with the identifier NCT05345171. This randomized, double-blind, controlled study is investigating the safety and efficacy of DTX301 in patients with OTC deficiency and is currently active but not recruiting.
DTX301 is an AAV-mediated gene transfer therapy specifically designed for OTC deficiency. It is the only gene therapy candidate for this condition mentioned in the available clinical trial data, and it is being studied in a Phase 3 trial.