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Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

Primary Purpose

MELAS Syndrome

Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Dichloroacetate
Sponsored by
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for MELAS Syndrome focused on measuring Lactic Acidosis, Mitochondrial Disorder, Stroke, Seizure

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • New York Presbyterian Hospital

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
September 10, 2003
Last Updated
June 23, 2005
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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1. Study Identification

Unique Protocol Identification Number
NCT00068913
Brief Title
Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
Official Title
Investigation of Clinical Syndromes Associated With mtDNA Point Mutations: MELAS/DCA Clinical Trial
Study Type
Interventional

2. Study Status

Record Verification Date
September 2004
Overall Recruitment Status
Unknown status
Study Start Date
March 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MELAS Syndrome
Keywords
Lactic Acidosis, Mitochondrial Disorder, Stroke, Seizure

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
Double
Allocation
Randomized
Enrollment
35 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Dichloroacetate

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Darryl C De Vivo, MD
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
New York Presbyterian Hospital
City
New York City
State/Province
New York
ZIP/Postal Code
10032
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

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