Trial · NCT00432744
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
NCT00432744, Phase 3, Completed, University of Florida; results first posted 2014-05-01
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase III Trial of Coenzyme Q10 in Mitochondrial Disease. Trials, record NCT00432744. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00432744
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
- NCT number
- NCT00432744
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 24 participants, actual
- Lead sponsor
- University of Florida
- Conditions
- Mitochondrial Diseases
- Interventions
- CoenzymeQ10, Placebo
- First posted
- 2007-02-08
- Last updated
- 2017-09-11
- Results first posted
- 2014-05-01
- Limitations and caveats
- The trial intended to accrual 40 subjects, but accrual was below expectations and the granting agency turned down a request for extended accrual.
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | McMaster Gross Motor Function (GMFM 88) | 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. |
| Primary | Pediatric Quality of Life Scale | 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. |
| Primary | Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. | 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. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | McMaster Gross Motor Function (GMFM 88) | Taken at 6 and 12 Months | units on a scale | MEDIAN | |
| PRIMARY | Pediatric Quality of Life Scale | At 6 and 12 Months | 7 subjects in each group were evaluable for this endpoint. The two subjects who became disabled are evaluable. One subject did not have period 2 Quality of Life data and is excluded. | units on a scale | MEDIAN |
| PRIMARY | Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. | end of 12 month minus end of 6 month difference. | Subjects contributes at least one difference (either GMFM or Peds QOL). Thirteen contributed both and one each was missing GMFM or Peds QOL. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| McMaster Gross Motor Function (GMFM 88) | Placebo First | -0.002 | Inter-Quartile Range -0.47 to 1.36 | units on a scale | |
| McMaster Gross Motor Function (GMFM 88) | CoenzymeQ10 Frist | -0.12 | Inter-Quartile Range -2.75 to 1.75 | units on a scale | |
| Pediatric Quality of Life Scale | Placebo First | -1.1 | Inter-Quartile Range -4.3 to 7.1 | units on a scale | |
| Pediatric Quality of Life Scale | CoenzymeQ10 Frist | -11.9 | Inter-Quartile Range -43.5 to 4.3 | units on a scale | |
| Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. | Placebo First | 7 | participants | ||
| Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. | CoenzymeQ10 Frist | 8 | participants |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Months 0-6 | STARTED | Placebo First | 12 | |
| Months 0-6 | STARTED | CoenzymeQ10 Frist | 12 | |
| Months 0-6 | COMPLETED | Placebo First | 7 | |
| Months 0-6 | COMPLETED | CoenzymeQ10 Frist | 8 | |
| Months 0-6 | NOT COMPLETED | Placebo First | 5 | |
| Months 0-6 | NOT COMPLETED | CoenzymeQ10 Frist | 4 | |
| Months 7 - 12 | STARTED | Placebo First | 7 | |
| Months 7 - 12 | STARTED | CoenzymeQ10 Frist | 8 | |
| Months 7 - 12 | COMPLETED | Placebo First | 6 | |
| Months 7 - 12 | COMPLETED | CoenzymeQ10 Frist | 7 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events28
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Decreased White Blood Count | Blood and lymphatic system disorders | CoenzymeQ10 | 1 | 19 | 1 |
| other | Decreased White Blood Count | Blood and lymphatic system disorders | Placebo | 0 | 20 | 0 |
| other | Low ANC Levels | Blood and lymphatic system disorders | CoenzymeQ10 | 0 | 19 | 0 |
| other | Low ANC Levels | Blood and lymphatic system disorders | Placebo | 1 | 20 | 1 |
| other | Nose Bleed | General disorders | CoenzymeQ10 | 0 | 19 | 0 |
| other | Nose Bleed | General disorders | Placebo | 1 | 20 | 1 |
| other | Elevated Liver Enzymes | Blood and lymphatic system disorders | CoenzymeQ10 | 1 | 19 | 1 |
| other | Elevated Liver Enzymes | Blood and lymphatic system disorders | Placebo | 1 | 20 | 1 |
| other | Ear Infection | Ear and labyrinth disorders | CoenzymeQ10 | 1 | 19 | 1 |
| other | Ear Infection | Ear and labyrinth disorders | Placebo | 1 | 20 | 1 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CoenzymeQ10 | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | CoenzymeQ10 | 10 | 19 |
| Total, other adverse events | Placebo | 10 | 20 |
| Total, serious adverse events | CoenzymeQ10 | 1 | 19 |
| Total, serious adverse events | Placebo | 2 | 20 |
Source ClinicalTrials.gov
Papers25
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD003032 | PHASE 3 TRIAL OF COENZYME IN MITOCHONDRIAL DISEASE | STACPOOLE, PETER WALLACE | UNIVERSITY OF FLORIDA | FOOD AND DRUG ADMINISTRATION | 2006 to 2011 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| CoenzymeQ10 Frist vs Placebo First: Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. | Trial comparisons |
| CoenzymeQ10 vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00432744
GET https://conductscience.org/api/trials/v1/NCT00432744/events?page=2