Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-03-31

Brief Summary

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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

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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.

Conditions

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MELAS Syndrome

Keywords

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Lactic Acidosis Mitochondrial Disorder Stroke Seizure

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

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Dichloroacetate

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Darryl C De Vivo, MD

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Locations

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New York Presbyterian Hospital

New York, New York, United States

Site Status

Countries

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United States

Other Identifiers

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P01HD032062

Identifier Type: NIH

Identifier Source: org_study_id

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