drugset / Trial / NCT03747328
ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
NaSingle-groupOpen-labelTreatment
Summary
A phase 2a, open-label study to evaluate the safety, tolerability, and clinical activity of ABI-009 (nab-sirolimus) in patients with genetically-confirmed Leigh or Leigh-like syndrome
Timeline
- Start
- 2022-04-30
- Primary completion
- 2024-12-31
- Completion
- 2025-03-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | nab-sirolimus | Unknown | 1 mg/m2 | — |
| Subject | nab-sirolimus | Unknown | 2.5 mg/m2 | — |
| Subject | nab-sirolimus | Unknown | 5 mg/m2 | — |
| Subject | nab-sirolimus | Unknown | 10 mg/m2 | — |