drugset / Trial / NCT05092685
Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn
NaSingle-groupOpen-labelTreatment
Summary
Ornithine transcarbamylase deficiency (OTCD) is an inherited metabolic liver disease which means that the body cannot maintain normal levels of ammonia. Ammonia levels can rise (called hyperammonaemic decompensations) which can be life-threatening and may result in impaired neurological development in children. OTCD is a rare genetic disorder characterised by complete or partial lack of the enzyme ornithine transcarbamylase (OTC).
Timeline
- Start
- 2023-11-01
- Primary completion
- 2026-06-30
- Completion
- 2027-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAVLK03hOTC | Gene therapy (AAV / viral vector) | 6e+11 vg/kg | Intravenous |
| Subject | AAVLK03hOTC | Gene therapy (AAV / viral vector) | 2e+12 vg/kg | Intravenous |
| Subject | AAVLK03hOTC | Gene therapy (AAV / viral vector) | 6e+12 vg/kg | Intravenous |