drugset / Trial / NCT05397665
Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency in patients for up to 24 months.
Timeline
- Start
- 2022-01-01
- Primary completion
- 2024-10-12
- Completion
- 2026-06-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Govorestat | Small molecule | 20 mg/kg | — |