Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||Phase 3 Trial of Coenzyme Q10 in Mitochondrial Disease|
- Gross motor function and [ Time Frame: Every six months ] [ Designated as safety issue: No ]
- diagnostic laboratory data; [ Time Frame: Every three months ] [ Designated as safety issue: Yes ]
- H&P: patient demographic information and pertinent physiologic measures; [ Time Frame: Every three months ] [ Designated as safety issue: Yes ]
- neurobehavioral assessments: the American Association on Mental Retardation Adaptive Behavior Scales; [ Time Frame: Every six months ] [ Designated as safety issue: No ]
|Study Start Date:||January 2007|
|Estimated Study Completion Date:||May 2013|
|Estimated Primary Completion Date:||May 2013 (Final data collection date for primary outcome measure)|
Active Comparator: CoenzymeQ10
CoenzymeQ10, patients will be randomized to receive CoenzymeQ10.
CoenzymeQ10 will be given in 10 mg/kg daily up to 400 mg. Then a draw of CoQ10 troughs every three months will be performed.
Other Name: CoenzymeQ10
Placebo Comparator: Placebo
Placebo, patients will be randomized to receive placebo.
Placebo will be given in 10 mg/kg daily up to 400 mg. Then a draw of placebo troughs every three months will be performed. This treatment group will be treated as the active group.
Other Name: Placebo
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|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45267|
|Case Western Reserve University|
|Cleveland, Ohio, United States, 44106|
|Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Principal Investigator:||Douglas S. Kerr, MD, PhD||Case Western Reserve University|
|Principal Investigator:||Ton J deGrauw, MD, PhD||Children's Hospital Medical Center, Cincinnati|
|Principal Investigator:||Annette S. Feigenbaum, MD||SickKids, Toronto, Canada/University of Toronto|