L-Citrulline Dose Finding Safety Study in MELAS (NCT03952234) | Clinical Trial Compass
CompletedPhase 1
L-Citrulline Dose Finding Safety Study in MELAS
United States10 participantsStarted 2021-04-22
Plain-language summary
The main purpose of this study is to determine the safest maximum dose of an amino acid, citrulline, which will be used as potential treatment for adult patients with a disorder of energy metabolism called Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS). Once established, this dose will be used in a future clinical trial.
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion criteria
✓. Clinical diagnosis of MELAS (stroke-like events, seizures, exercise intolerance or muscle weakness).
✓. Subject must be aged 18 to 65 years.
✓. The m.3243A\>G mutation in the MTTL1 gene.
✓. Elevated plasma lactate (\>2.2 mmol/L) taken at any point in the screening period (6 months prior to screening visit, including and up to the baseline visit).
✓. Negative urine pregnancy test, if applicable.
✓. Score of 26 or higher on the Montreal Cognitive Assessment (MOCA). -
Exclusion criteria
✕. Evidence of acute illness or physical disability that may interfere with their ability to undergo the study.
✕. Tobacco use
✕. Orthostatic hypotension defined as a decrease in systolic blood pressure of 20 mm Hg, or a decrease in diastolic blood pressure of 10 mm Hg, between one and three minutes of standing when compared with blood pressure from the sitting or supine position at the baseline visit.
✕. Presence of the following signs or symptoms in the past 12 months at grade 3 or higher based on the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03: hypotension, syncope, dizziness, blurred vision, fatigue, concentration impairment, nausea, vomiting, diarrhea, hypoglycemia, or headache.
✕. \> 2 seizures in week prior to baseline visit.
What they're measuring
1
Establishment of the maximum tolerable dose of L-citrulline in patients with MELAS syndrome by measuring the incidence of dose limiting toxicities (DLTs)