drugset / Trial / NCT07221292
Pivotal Study of N-acetyl-L-leucine for CACNA1A
RandomizedCrossoverQuadruple-blindTreatment
Summary
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Timeline
- Start
- 2026-09-01
- Primary completion
- 2027-08-01
- Completion
- 2028-11-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Levacetylleucine | Small molecule | 2 g | Oral |
| Subject | Levacetylleucine | Small molecule | 4 g | Oral |