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Amino Acid
L-Citrulline for MELAS Syndrome
Phase 1
Recruiting
Led By FERNANDO SCAGLIA, M.D
Research Sponsored by Baylor College of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Subject must be aged 18 to 65 years
Clinical diagnosis of MELAS (stroke-like events, seizures, exercise intolerance or muscle weakness)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up one week
Awards & highlights
Study Summary
This trial is testing a potential treatment for MELAS, a disorder of energy metabolism. The goal is to find the safest maximum dose of an amino acid, citrulline.
Who is the study for?
Adults aged 18-65 with MELAS Syndrome, a specific genetic mutation (m.3243A>G), and elevated plasma lactate can join this trial. They must have cognitive health to pass the MOCA test and not be pregnant or using tobacco. Those with recent seizures, no neurological symptoms, severe kidney issues, or taking certain medications are excluded.Check my eligibility
What is being tested?
The study is testing the safety of different doses of L-Citrulline in adults with MELAS Syndrome to find the highest safe dose for future research. Participants will receive increasing amounts of L-Citrulline to establish its maximum tolerable level without significant side effects.See study design
What are the potential side effects?
Potential side effects may include hypotension (low blood pressure), dizziness, blurred vision, fatigue, difficulty concentrating, nausea, vomiting, diarrhea, hypoglycemia (low blood sugar), or headache as indicated by previous criteria for adverse events.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 18 and 65 years old.
Select...
I have been diagnosed with MELAS, experiencing symptoms like stroke-like events, seizures, or muscle weakness.
Select...
My genetic test shows I have the m.3243A>G mutation.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ four weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~four weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Establishment of the maximum tolerable dose of L-citrulline in patients with MELAS syndrome by measuring the incidence of dose limiting toxicities (DLTs)
Secondary outcome measures
Changes effected by the use of citrulline in the concentration of plasma guanidino compounds
Changes effected by the use of citrulline in the micromolar concentration of plasma alanine and in the concentration of plasma lactate (expressed in millimole per liter)
Changes effected by the use of citrulline supplementation in the micromolar concentration of plasma amino acids citrulline, arginine, ornithine, and alanine levels.
+2 moreSide effects data
From 2021 Phase 2 trial • 65 Patients • NCT045703849%
Infusion Site Reaction
6%
Deep Vein Thrombosis
6%
Dyspnoea
6%
Clostridium Difficile Infection
6%
Urinary Tract Infection
3%
Staphylococcal Sepsis; Alpha Haemolytic Streptococcal Infection
3%
Pain In Extremity
3%
Skin Wound
3%
Acute kidney injury
3%
Cardiac failure; metabolic acidosis
3%
Chest pain; Dyspnoea; COVID-19
3%
Intracranial hemorrhage
3%
Acute Myocardial Infarction
3%
Status epilepticus
3%
Syncope
3%
Enterococcal Infection
3%
Enterococcus Test Positive
3%
Epistaxis
3%
Hypoglycaemia
3%
Hypoxia
3%
Tachycardia
3%
Ventricular Extrasystoles
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo Arm
IV L-Citrulline (Turnobi) Arm
Trial Design
1Treatment groups
Experimental Treatment
Group I: Dose finding safety studyExperimental Treatment1 Intervention
In this study, the highest acceptable dose of an amino acid called citrulline will be established in people who have a mitochondrial disorder. Previous research conducted by several groups including our center at Baylor College of Medicine has determined that there is a deficiency of a compound called nitric oxide in people affected with MELAS.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
L-Citrulline
2023
Completed Phase 2
~340
Find a Location
Who is running the clinical trial?
Columbia UniversityOTHER
1,434 Previous Clinical Trials
2,448,014 Total Patients Enrolled
University of South FloridaOTHER
413 Previous Clinical Trials
188,243 Total Patients Enrolled
National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,342 Previous Clinical Trials
650,328 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I can understand and report low blood sugar levels.You have taken arginine supplements within one week before the baseline visit.If your blood pressure drops significantly when you stand up, you may not be able to participate.I am between 18 and 65 years old.I am taking medication or supplements that affect blood flow and cannot stop them for the study.My cognitive test score is below 26.I have been diagnosed with MELAS, experiencing symptoms like stroke-like events, seizures, or muscle weakness.If you can get pregnant, you must have a negative urine pregnancy test.I do not have any illness or disability that could affect my participation in the study.You scored 26 or higher on the Montreal Cognitive Assessment (MOCA).I do not have neurological disease, muscle weakness, or trouble exercising.I cannot travel to the study location.Your blood pressure is too low (systolic blood pressure ≤ 90 mm Hg or diastolic blood pressure ≤ 60 mm Hg) at the start of the study.I've had severe symptoms like fainting, dizziness, or headaches in the past year.You use tobacco.You are pregnant according to a urine test.I have had more than 2 seizures in the week before my first visit.I am not willing to use birth control.My kidney function is moderately to severely reduced.My genetic test shows I have the m.3243A>G mutation.Your blood test shows high lactate levels at any time during the screening period.
Research Study Groups:
This trial has the following groups:- Group 1: Dose finding safety study
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any other precedents in which this therapy has been tested and evaluated?
"Currently, the total number of active research studies for this treatment stands at four. None are currently in Phase 3; however all trials can be found near Durham, North carolina and spread across 4 separate sites."
Answered by AI
What criteria must an individual meet to participate in this experiment?
"24 individuals with MELAS Syndrome aged 18-65 are eligible for this trial. The enumerated requirements include: a clinical diagnosis of MELAS, the m.3243A>G mutation in MTTL1 gene, negative pregnancy test (if applicable), 26 or higher on Montreal Cognitive Assessment (MOCA), and raised plasma lactate (>2.2 mmol/L) at baseline visit."
Answered by AI
What is the current limit of participants enrolling in this clinical experiment?
"Indeed, the listing on clinicaltrials.gov confirms that this particular medical trial is actively enrolling participants. Initially posted on April 22nd 2021 and updated most recently in April 15th 2022, it seeks to include 24 patients from a single location."
Answered by AI
Are there still openings available for the clinical trial participants?
"Affirmative. Clinicaltrials.gov states that this health investigation, initially posted on April 22nd 2021, is presently recruiting participants. They hope to find 24 patients from one medical facility."
Answered by AI
Has this remedy been given the thumbs-up by federal health authorities?
"Limited data has been collected to date on the efficacy and safety of this treatment, thus our team at Power gave it a rating of 1."
Answered by AI
Does this research trial accept adults as participants?
"This medical trial is accepting patients between the ages of 18-65. For those outside this age bracket, there are two trials available for minors and seven trials that target senior citizens."
Answered by AI
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