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Home > "E" Clinical Trials Conditions > Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome  Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
For Condition: MELAS Syndrome
Status: Recruiting
Sponsor(s): National Institute of Child Health and Human Development (NICHD) ,
Synopsis: Patients with the MELAS syndrome experience devastating mental impairment. This study will evaluate the effectiveness of the drug dichloroacetate (DCA) to reduce the symptoms of MELAS.
Details: Although many organ systems are affected by mitochondrial (mt) DNA point mutations, the nervous system is particularly vulnerable. Maternally inherited mtDNA point mutations may cause chronic progressive encephalopathies and mental retardation. Patients with MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes) syndrome have the A3243G point mutation and elevated brain lactate levels. Research has shown that lactic acidodis is associated with progressive impairment in patients with MELAS. This study will evaluate the effectiveness of DCA in lowering lactate levels and slowing the progression of MELAS. Patients with the A3243G mitochondrial mutation and who have had either a stroke or a seizure will be enrolled in this study. Patients will be randomized to receive either DCA or a placebo. At a predetermined time point, patients receiving DCA will be switched to placebo and patients receiving placebo will be switched to DCA. Patients will have study visits every 3 months for 3 years. Study visits will include neurological exams, cognitive testing, nerve conduction tests, and MRIs. Study medicine, testing, hospitalization for research purposes, and travel expenses will be fully covered by the study.
Eligibility:
Study Type: Interventional, Treatment, Randomized, Double-Blind, Placebo Control, Crossover Assignment, Safety/Efficacy Study
Minimum Age/Maximum Age: 6 Years/
Genders: Both
Protocol Entry Criteria: Inclusion Criteria - A3243G mtDNA point mutation or maternally related to someone who has the mutation - Symptomatic with MELAS, including previous seizure or stroke - Certain laboratory values - Ability to comply with the study protocol
Total Enrollment: 35
Location and Contact Information:
Overall Study Official:
DarrylDe Vivo, Principal Investigator, Columbia University
New York Presbyterian Hospital *Recruiting*
New York City, New York, 10032
United States
Recruiting Kris Engelstad 212-305-8025
Additional Information:
Study ID Numbers: P01HD32062;
Study Start Date: March 2000
Record last reviewed: September 2003
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00068913
Other Melas Syndrome Studies:
1. Study of the Metabolism of Pyruvate and Related Problems in Patients with Lactic Acidemia
2. Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
Related Studies:
Other MELAS Syndrome Clinical Trials
Other New York Clinical Trials
Other New York City Clinical Trials
Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
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