Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Completed · Phase 3 · Has a placebo group
Conditions studied: Mitochondrial Diseases
In brief
To show that oral CoQ10 is a safe and effective treatment for children with inborn errors of mitochondrial energy metabolism due to defects in specific respiratory chain (RC) complexes or mitochondrial DNA (mtDNA) mutations, and that this beneficial action is reflected in improved motor and neurobehavioral function.
Key facts
- Study ID
- NCT00432744
- Run by
- University of Florida
- People needed
- 24
- Starts
- 2007-01-01
- Expected to finish
- 2013-05-01
- Last updated by the study team
- 2017-09-11
Who can join
Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Age 12 m - 17 y
- Biochemical proof of a deficiency of complex I, III or IV of the RC or a molecular genetic proof of a mutation in mtDNA, or an nDNA mutation in a gene known to be associated with dysfunction of the electron transport chain (e.g., SURF1)
- Willingness to stop all other medication regimens and supplements other than what the Steering and Planning Committee deems medically necessary
You may not qualify if…
- A genetic mitochondrial disease other than those stipulated under inclusion criteria
- Intractable epilepsy, defined as grand mal seizures occurring with a frequency > 4/month, despite treatment with conventional antiepileptic drugs
- Primary, defined organic acidurias other than lactic acidosis (e.g., propionic aciduria
- Primary disorders of amino acid metabolism
- Primary disorders of fatty acid oxidation
- Secondary lactic acidosis due to impaired oxygenation or circulation (e.g., due to severe cardiomyopathy or congenital heart defects)
- Severe anemia, defined as a hematocrit <30%
- Malabsorption syndromes associated with D-lactic acidosis
- Renal insufficiency, defined as (1) a requirement for chronic dialysis or (2) serum creatinine ≥ 1.2 mg/dl or creatinine clearance <60 ml/min
- Primary hepatic disease unrelated to mitochondrial disease
- Allergy to CoQ10 or placebo ingredients
- Pregnancy
Where it is running
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
- Case Western Reserve University — Cleveland, Ohio, United States
- Hospital for Sick Children — Toronto, Ontario, Canada
Full record on ClinicalTrials.gov
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