Phase III Trial of Coenzyme Q10 in Mitochondrial Disease

This study has been completed.
Information provided by (Responsible Party):
University of Florida Identifier:
First received: February 6, 2007
Last updated: August 13, 2013
Last verified: August 2013

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.

Condition Intervention Phase
Mitochondrial Diseases
Drug: CoenzymeQ10
Drug: Placebo
Phase 3

Study Type: Interventional
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

Resource links provided by NLM:

Further study details as provided by University of Florida:

Primary Outcome Measures:
  • Gross motor function and [ Time Frame: Every six months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 26
Study Start Date: January 2007
Study Completion Date: May 2013
Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: CoenzymeQ10
CoenzymeQ10, patients will be randomized to receive CoenzymeQ10.
Drug: 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.
Drug: 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

  Show Detailed Description


Ages Eligible for Study:   12 Months to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Please refer to this study by its identifier: NCT00432744

United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45267
Case Western Reserve University
Cleveland, Ohio, United States, 44106
Canada, Ontario
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
University of Florida
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
  More Information

Additional Information:
Kerr DS. Treatment of congenital lactic acidosis: a review. Intern Pediatr, 1995;10:75-81.

Responsible Party: University of Florida Identifier: NCT00432744     History of Changes
Other Study ID Numbers: 1 R01 FD003032-01A1
Study First Received: February 6, 2007
Last Updated: August 13, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Florida:
mitochondrial diseases
respiratory chain complex I deficiencies
respiratory chain complex II deficiencies
respiratory chain complex III deficiencies
respiratory chain complex IV deficiencies
mutations of a gene coding for a respiratory chain component

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases
Coenzyme Q10
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
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