NCT00068913

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.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2000

Completed
3.5 years until next milestone

First Submitted

Initial submission to the registry

September 10, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 11, 2003

Completed
Last Updated

June 24, 2005

Status Verified

September 1, 2004

First QC Date

September 10, 2003

Last Update Submit

June 23, 2005

Conditions

MELAS Syndrome

Keywords

Lactic AcidosisMitochondrial DisorderStrokeSeizure

Interventions

DichloroacetateDRUG

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

New York Presbyterian Hospital

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

MELAS SyndromeAcidosis, LacticMitochondrial DiseasesStrokeSeizures

Interventions

Dichloroacetic Acid

Condition Hierarchy (Ancestors)

Mitochondrial EncephalomyopathiesMitochondrial MyopathiesMuscular DiseasesMusculoskeletal DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersNeuromuscular DiseasesVascular DiseasesCardiovascular DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesAcidosisAcid-Base ImbalanceNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ChloroacetatesAcetatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsHydrocarbons, ChlorinatedHydrocarbons, HalogenatedHydrocarbons

Study Officials

  • Darryl C De Vivo, MD

    Columbia University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
NIH

Study Record Dates

First Submitted

September 10, 2003

First Posted

September 11, 2003

Study Start

March 1, 2000

Last Updated

June 24, 2005

Record last verified: 2004-09

Locations

US(1)