| NCT ID | Title | Condition | Status |
|---|---|---|---|
| Phase 3 | |||
| NCT05163288 | A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | Niemann-Pick Disease, Type C | Recruiting |
| NCT06673056 | A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) | Ataxia-Telangiectasia | Active, Not Recruiting |
| NCT07221292 | Pivotal Study of N-acetyl-L-leucine for CACNA1A | CACNA1A | Not Yet Recruiting |