L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome
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|ClinicalTrials.gov Identifier: NCT01603446|
Recruitment Status : Completed
First Posted : May 22, 2012
Last Update Posted : December 17, 2013
|Condition or disease||Intervention/treatment||Phase|
|MELAS Syndrome||Drug: L-Arginine||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study to Investigate the Efficacy of L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome.|
|Study Start Date :||May 2012|
|Actual Primary Completion Date :||May 2013|
|Actual Study Completion Date :||December 2013|
Experimental: MELAS Patients
Three siblings with MELAS (A3243G) syndrome (1 male; 2 females) aged 17-23 years, followed or previously followed in the Neurometabolic Clinic at the Hospital for Sick Children will be studied.
NOW® L-Arginine powder
No Intervention: Control Group
Four age- and sex-matched controls and female controls will be matched according to phase in menstrual cycle corresponding with their age-matched MELAS subjects
- Muscle function investigation via 31P-Magnetic resonance spectroscopy [ Time Frame: 60 to 105 minutes post dose ]We will study exercising quadriceps using our MR-compatible up-down ergometer and our well established aerobic exercise protocol at 65 % of maximal voluntary contraction.
- Total body maximal aerobic capacity [ Time Frame: 60-75 mins post dose ]Maximal incremental cycle ergometry is conducted in our CardioRespiratory Exercise Lab at HSC by our established protocols (26). Serum CK and quantitative AA (for arginine, ornithine and citrulline) will be measured pre- and post- exercise as well as eNO in order to correlate aerobic exercise parameters with serum arg and eNO levels..
- CerebroVascular Reactivity [ Time Frame: 75-105 mins post dose ]Functional MRI-Blood oxygen level dependent (BOLD) of brain
- Exhaled Nitric Oxide (eNO) [ Time Frame: 75 mins pre dose, 75 mins post dose ]eNO will be measured using single breath on-line measurements for the assessment of lower airway Nitric Oxide
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01603446
|The Hospital for Sick Children|
|Toronto, Ontario, Canada, M5V1X8|
|Principal Investigator:||Ingrid Tein, MD||The Hospital for Sick Children|