NCT03952234CompletedPhase 1
L-Citrulline Dose Finding Safety Study in MELAS
Phase-1, Dose Finding and Safety Study on L- Citrulline Treatment of Nitric Oxide Deficiency in MELAS
Source: ClinicalTrials.gov · Last updated 26 Jun 2024
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (actual)
- First submitted
- 8 May 2019
- Start date
- 22 Apr 2021
- Primary completion date
- 30 Apr 2024
- Lead sponsor
- Baylor College of Medicine
- Diseases
- Stroke
- NCT number
- NCT03952234
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 10
- From 22 Apr 2021 to the primary completion date, 30 Apr 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- \> 2 seizures in week prior to baseline visit.
- Arginine supplementation within one week prior to baseline visit.
- Intake of drugs that increase NO synthesis, vasodilators, or amino acid supplements that cannot be stopped during the study period.
Other illness
- Evidence of acute illness or physical disability that may interfere with their ability to undergo the study.
- 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.
- Subjects with no evidence of neurological disease, muscle weakness, or exercise intolerance.
Lab values
- Subjects with evidence of moderate to severe renal impairment ( eGFR \< 60 mL/min/1.73 m2 ) at the baseline visit.
Age
- Unwillingness of sexually active female subjects of childbearing age to practice reliable methods of contraception.
Other
- 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.
- Hypotension defined as systolic blood pressure ≤ 90 mm Hg or diastolic blood pressure ≤ 60 mm Hg at the baseline visit.
- Inability to travel to the study site.
- Subjects with poor cognitive ability to provide consent and to understand and report hypoglycemia.
- Positive urine pregnancy test.
- Score of less than 26 on the Montreal Cognitive Assessment (MOCA). -
Who the study is looking for
- 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). -
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Baylor St. Luke's Medical Center Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.