Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
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ClinicalTrials.gov Identifier: NCT00432744 |
Recruitment Status :
Completed
First Posted : February 8, 2007
Results First Posted : May 1, 2014
Last Update Posted : September 11, 2017
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Condition or disease | Intervention/treatment | Phase |
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Mitochondrial Diseases | Drug: CoenzymeQ10 Drug: Placebo | Phase 3 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 24 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | Phase 3 Trial of Coenzyme Q10 in Mitochondrial Disease |
Study Start Date : | January 2007 |
Actual Primary Completion Date : | May 2013 |
Actual Study Completion Date : | May 2013 |

Arm | Intervention/treatment |
---|---|
Active Comparator: CoenzymeQ10
CoenzymeQ10: patients will be randomized to receive CoenzymeQ10 in either Period #1 (Months 0-6) or Period #2 (Months 7-12).
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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. |
Placebo Comparator: Placebo
Placebo: patients will be randomized to receive placebo either ion Period #1 (months 1-6) or Period #2 (months 7-12).
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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. |
- 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 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. Goggle "pedsQL and Mapi" to browse the copyrighted manual. A link to the instrument is included.
- Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. [ Time Frame: end of 12 month minus end of 6 month difference. ]This is a multivariate analysis of the first two outcomes: Period 2 minus Period 1 GMFM88 and Peds Quality of Life, analyzed as follows: First, to be in the analysis, subjects must contribute at least one of these endpoints. Second, if the subject became totally disabled during period 1, the difference was defined as + infinity, (highest possible evidence favoring period 2), and if the subject became totally disabled in period 2, the subject was scored as - infinity (highest possible evidence favoring period 1). Period 2 minus period 1 differences were ranked form low to high with missing values scores at the mid-rank. The Hotelling T-square was computed on these ranks and the P-value was obtained from 100,000 rerandomizations as the fraction of rerandomizations with T-sq at least as large as that observed.

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Ages Eligible for Study: | 12 Months to 17 Years (Child) |
Sexes Eligible for Study: | All |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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 |
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 |
Publications:
Responsible Party: | University of Florida |
ClinicalTrials.gov Identifier: | NCT00432744 |
Other Study ID Numbers: |
1R01FD003032-01A1 ( U.S. FDA Grant/Contract ) R01FD003032-01A1 ( U.S. FDA Grant/Contract ) |
First Posted: | February 8, 2007 Key Record Dates |
Results First Posted: | May 1, 2014 |
Last Update Posted: | September 11, 2017 |
Last Verified: | September 2017 |
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 |
Mitochondrial Diseases Metabolic Diseases |