Nitric Oxide Production in MELAS Syndrome
Primary Purpose
MELAS Syndrome
Status
Completed
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Arginine and citrulline supplementations
Sponsored by
About this trial
This is an interventional treatment trial for MELAS Syndrome
Eligibility Criteria
Inclusion Criteria:
- Age from 3 - 65 years
- Clinical diagnosis of MELAS syndrome
- Carrying the m.3243A>G mutation
Exclusion Criteria:
- Having acute or chronic disease or physical disability that will interfere with the ability to undergo the study procedures
- Being pregnant
Sites / Locations
- Texas Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Active Comparator
Arm Label
arginine supplementation
citrulline supplementation
Arm Description
Oral L-arginine supplementation will be administered to subjects with MELAS for 48 hours at a dose of 10 grams per M2 per day. Arginine will be given every 4 hours.
Oral L-citrulline supplementation will be administered to subjects with MELAS for 48 hours at a dose of 10 grams per M2 per day. Citrulline will be given every 4 hours
Outcomes
Primary Outcome Measures
Change in nitric oxide production
Secondary Outcome Measures
Full Information
NCT ID
NCT01339494
First Posted
April 19, 2011
Last Updated
April 1, 2016
Sponsor
Baylor College of Medicine
1. Study Identification
Unique Protocol Identification Number
NCT01339494
Brief Title
Nitric Oxide Production in MELAS Syndrome
Official Title
Arginine Flux and Nitric Oxide Production in Patients With MELAS Syndrome and the Effect of Arginine and Citrulline Supplementation
Study Type
Interventional
2. Study Status
Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
July 2009 (undefined)
Primary Completion Date
April 2016 (Actual)
Study Completion Date
April 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Baylor College of Medicine
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
Detailed Description
What does the study involve? Individuals with MELAS syndrome will be admitted twice to the General Clinical Research Center (GCRC) at Texas Children's Hospital. Each time they will stay for five days, during which a special diet will be provided. Nitric oxide production will be measured by stable isotopes infusion technique that involves placing small tubes in veins (IV catheter), blood sampling, and injecting a harmless stable isotope. Stable isotopes are forms of normal compounds that can be measured accurately. They are not radioactive and there are no known risks to giving them; they are already part of your body in small amounts.
During the first admission nitric oxide levels will be measured, then arginine supplementation will be provided for 48 hours, after which nitric oxide levels will be re-measured to determine the effect of arginine supplementation. During the second admission, the effect of citrulline supplementation will be measured.
Who can participate in the study? Adults or children affected with MELAS syndrome and carrying the DNA change that causes the condition (3243 A>G mutation) can participate. Adults without MELAS disease will be recruited to participate as control subjects.
How to get more information? Subjects interested in participation or getting more information can contact Dr. Ayman El-Hattab at email: elhattab@bcm.edu, office phone: 832-822-4289, cell phone: 646-660-5666, or pager: 832-824-7243 (5523).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MELAS Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
arginine supplementation
Arm Type
Active Comparator
Arm Description
Oral L-arginine supplementation will be administered to subjects with MELAS for 48 hours at a dose of 10 grams per M2 per day. Arginine will be given every 4 hours.
Arm Title
citrulline supplementation
Arm Type
Active Comparator
Arm Description
Oral L-citrulline supplementation will be administered to subjects with MELAS for 48 hours at a dose of 10 grams per M2 per day. Citrulline will be given every 4 hours
Intervention Type
Dietary Supplement
Intervention Name(s)
Arginine and citrulline supplementations
Intervention Description
Arginine or citrulline will be given orally at dose of 10 grams per meter square body surface area per day divided every 4 hours.
Primary Outcome Measure Information:
Title
Change in nitric oxide production
Time Frame
Nitric oxide production will be measured at baseline before supplementation and after 48 hours of arginine or citrulline supplementation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Age from 3 - 65 years
Clinical diagnosis of MELAS syndrome
Carrying the m.3243A>G mutation
Exclusion Criteria:
Having acute or chronic disease or physical disability that will interfere with the ability to undergo the study procedures
Being pregnant
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fernando Scaglia, M.D.
Organizational Affiliation
Baylor College of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Yes the data have been made available to
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Nitric Oxide Production in MELAS Syndrome
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