MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes) syndrome is a genetic disorder affecting energy metabolism. Researchers at Baylor College of Medicine are conducting a Phase 1 clinical trial to test L-citrulline, an amino acid treatment, in patients with MELAS.
Patients with this disorder suffer from stroke-like episodes, headaches, muscle weakness, fatigue and hearing loss. A major contributing factor is a decreased amount of nitric acid in the body.
"The lack of nitric oxide can cause blood vessels in the brain to constrict, leading to metabolic strokes," said Dr. Fernando Scaglia, principal investigator of the study and professor of molecular and human genetics at Baylor and attending physician at Baylor St. Luke's Medical Center. "The amino acid citrulline is a foundation for nitric oxide. We have found that people who take L-citrulline treatment produce more nitric oxide."
This Phase 1 safety study will test the safest maximum dose of L-citrulline for patients with MELAS. Once established, this dose will be used in a future clinical trial that will test the efficacy of L-citrulline.
