Baylor College of Medicine

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Baylor recruiting MELAS syndrome patients for Phase 1 clinical trial

Molly Chiu

713-798-4710

Houston, TX -
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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.

Participants will be required to take oral L-citrulline for a month and will be followed for an additional month after treatment. Participants will be evaluated once a week at the Baylor St. Luke’s Medical Center Clinical Research Center while receiving treatment and will come for two additional safety visits during the following month. Two brain imaging tests will be conducted, once before treatment and once at week four of the study to measure the effect of L-citrulline on blood vessels of the brain. For more information on how to join, contact May Ali at maali@bcm.edu or 832-822-1630.

This clinical trial is sponsored by the National Institutes of Health and the Mervar Foundation. Read more about the study protocol here.

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