Ignite Creation Date:
2024-05-05 @ 11:30 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00068913
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
2003-09-10
Brief Title:
Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Study Overview
Official Title:
Investigation of Clinical Syndromes Associated With mtDNA Point Mutations MELASDCA Clinical Trial
Status:
UNKNOWN
Status Verified Date:
2004-09
Last Known Status:
ACTIVE_NOT_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
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 acidosis 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
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None