Clinical Trial: Phase III Trial of Coenzyme Q10 in Mitochondrial Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase 3 Trial of Coenzyme Q10 in Mitochondrial Disease

Brief Summary: 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.

Detailed Summary:

In many patients mitochondrial disease leads to progressive nerve and muscle damage that may be associated with problems with thinking, talking, remembering, walking or balancing. Sometimes it may also cause abnormal build up in the blood and spinal fluid of a substance called lactic acid. This problem makes the blood and spinal fluid too acid, which can be life-threatening. There is no known specific or effective treatment for mitochondrial diseases. Sometimes certain diets can improve the condition but seldom prevent the nerve or muscle damage or reduce the chance of developing life-threatening acidity of the blood.

CoQ10 is not approved by the Food and Drug Administration (FDA) for the treatment of mitochondrial diseases. It is an investigational drug that we believe may help people with certain mitochondrial diseases, like the one you have, both in terms of reducing the acidity of your blood and preventing or decreasing nerve and muscle damage. Our belief is based on previous studies that have been conducted in children with mitochondrial diseases of various types. Therefore, a 12 month study has been designed to determine the safety and benefit of taking CoQ10 every day. This will be done by comparing the subjects response to taking CoQ10 for 6 months to taking a placebo for 6 months. A placebo looks, smells, and tastes like the drug being tested (in this case, CoQ10) but has no effect. Placebo studies such as this one are very common in evaluating investigational drugs for any disease and are usually required by the FDA before a drug can be approved.

CoQ10 or placebo will be given as a liquid once per day in the evening, by mouth or feeding tube. The CoQ10 dose will be 10 mg/kg with a maximum dose of 400 mg a day. Neither the subject nor the health care givers will know exactly when subjects are receiving CoQ10 or wh
Sponsor: University of Florida

Current Primary Outcome:

  • McMaster Gross Motor Function (GMFM 88) [ Time Frame: Taken at 6 and 12 Months ]
    The McMaster Gross Motor Function is a validated scale ranging from 0 to 100 (the higher the better). Since there was the possibility of a subject becoming totally disabled our FDA peer reviewed design called for its use as follows: If the subject completed both periods, the score was calculated as the difference in scores between the end of Period 2 (at 12 months) minus that at the end of Period 1 (6 months). If a subject became totally disabled, this difference was considered as plus infinity if it occurred in period 1 (Penalizes period 1), and minus infinity if it occurred in Period 2 (Penalizes period 2). The two treatments were compared via the Wilcoxon test, and the effect size was estimated using Kendall's Tau-B. This is interpreted in a similar manner to correlation with positive values favoring COQenzyme10 and negative values favoring placebo. One of the links in this report is to the the GMFM scale and how it is scored. A link to the instrument is included.
  • Pediatric Quality of Life Scale [ Time Frame: At 6 and 12 Months ]
    The Pediatric Quality of Life Scale is a validated scale ranging from 0 to 100 (the higher the better). Since there was the possibility of a subject becoming totally disabled our FDA peer reviewed design called for its use as follows: If the subject completed both periods, the score was calculated as the difference in scores between the end of Period 2 (at 12 months) minus that at the end of Period 1 (6 months). If a subject became totally disabled, this difference was considered as plus infinity if it occurred in period 1 (Penalizes period 1), and minus infinity if it occurred in Period 2 (Penalizes period 2). The

    Original Primary Outcome:

    • Gross motor function and
    • home quality of life (QOL)


    Current Secondary Outcome:

    Original Secondary Outcome:

    • diagnostic laboratory data;
    • H&P: patient demographic information and pertinent physiologic measures;
    • motor assessments: Gross Motor Function Measure,
    • neurobehavioral assessments: NIHM neurobehavioral examination, the Child Development Inventory and the American Association on Mental Retardation Adaptive Behavior Scales;
    • QOL assessment;
    • sleep questionnaire;
    • CoQ10 profile data;
    • LiQ-nol parent survey Effects Report and
    • QOL Validity Studies


    Information By: University of Florida

    Dates:
    Date Received: February 6, 2007
    Date Started: January 2007
    Date Completion:
    Last Updated: April 30, 2014
    Last Verified: April 2014