search

Active clinical trials for "MELAS Syndrome"

Results 1-10 of 18

L-Citrulline Dose Finding Safety Study in MELAS

MELAS Syndrome

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.

Recruiting21 enrollment criteria

Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome

MELAS Syndrome

The purpose of this study is to assesses the clinical efficacy of oral supplementation with glutamine over 3 years.

Active3 enrollment criteria

The KHENEREXT Study

Mitochondrial DiseasesMitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation4 more

This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202. In the KH176-203 study subjects will be receiving KH176 100 mg BID or KH176 50 mg bid in die (BID) (as determined by the investigator based on safety / tolerability considerations) for a year, thereby ensuring continued treatment with KH176 after study KH176-202. A final follow-up visit is scheduled 4 weeks after the intake of the last dose of study medication for patients not rolling over into the compassionate use program. Primary safety data and secondary efficacy (endpoint) data will be monitored and reviewed every three months by an independent Data Safety Monitoring Board (DSMB) to evaluate potential risks and benefits.

Active39 enrollment criteria

Global Registry and Natural History Study for Mitochondrial Disorders

Mitochondrial DiseasesKearns-Sayre Syndrome16 more

The main goal of the project is provison of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.

Recruiting3 enrollment criteria

Effectiveness of Ketogenic Diet in MELAS Syndrome

MELAS SyndromeKetogenic Dieting

The goal of this clinical trial is to evaluate the effectiveness of ketogenic diet in patients with MELAS syndrome. The main questions it aims to answer are: Clarify the curative effects of ketogenic diet in the treatment of MELAS disease. Prevent the aggravation of MELAS disease, and improve the quality of life of patients. Provide reliable evidence-based medical basis for the clinical application of ketogenic diet in the treatment of MELAS syndrome patients. The clinical data of the participants treated with ketogenic diet will be collected, including the completion of ketogenic diet and clinical data at the start of treatment and after 1 month, 3 months, 6 months and 12 months

Not yet recruiting2 enrollment criteria

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis,...

MELAS

This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.

Terminated16 enrollment criteria

Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers

MELAS SyndromeMitochondrial Respiratory Chain Deficiencies

The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.

Completed20 enrollment criteria

A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial...

Mitochondrial DiseasesMitochondrial Respiratory Chain Deficiencies2 more

This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C. Part A and Part B include healthy volunteers only and will be completed before Part C including patients with primary mitochondrial disease will be initiated. The starting dose in the first cohort of Part A will be 25 mg. The dose level in the additional cohorts will be decided following review of data of the previous cohorts.

Completed27 enrollment criteria

Nitric Oxide Production in MELAS Syndrome

MELAS Syndrome

Introduction Baylor College of Medicine and Texas Children's Hospital are recruiting individuals with MELAS syndrome for a clinical study. MELAS syndrome is a mitochondrial disease; patients with this disease have muscle weakness and often develop brain strokes, where blood does not flow normally to different parts of the brain. It is believed that these strokes could be due to decreased production of nitric oxide, a naturally occurring compound important for normal blood vessel function. Nitric oxide is made from arginine and citrulline that are normally found in our bodies. What is the purpose of this study? The purpose of this study is to measure nitric oxide in individuals with MELAS and see if giving arginine or citrulline will increase the formation of nitric oxide. Nitric oxide is thought to be helpful in preventing strokes. Therefore, if arginine and/or citrulline are shown to increase the formation of nitric oxide, they could be used to prevent or treat the strokes in patients with MELAS syndrome.

Completed5 enrollment criteria

Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes)...

MELAS Syndrome

The purpose of this study is to assesses the efficacy of oral supplementation with glutamine over three months on several amino acids and lactate concentration measured in cerebrospinal fluid and cerebral lactate measured by magnetic resonance spectroscopy.

Completed4 enrollment criteria
12

Need Help? Contact our team!


We'll reach out to this number within 24 hrs