This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Phase III Trial of Coenzyme Q10 in Mitochondrial Disease (NCT00432744), a randomized phase 3 study. The primary outcome is McMaster Gross Motor Function (GMFM 88).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00432744 |
| Phase | Phase 3 |
| Condition | Mitochondrial Diseases |
| Allocation | Randomized |
| Design model | Crossover |
| Masking | Triple |
| Enrollment | 24 |
| Arms in the primary outcome | 2 |
| Lead sponsor | University of Florida |
| Results posted | Yes |
2. Clinical Question
Population
Children with inborn errors of mitochondrial energy metabolism due to defects in specific respiratory chain complexes or mitochondrial DNA mutations, as described in the trial summary.
Intervention
Oral CoenzymeQ10, assigned for one of the two treatment periods.
Comparator
Placebo, assigned for the other treatment period.
Primary question
How did the gross motor function outcome compare between the randomized treatment sequences?
The trial summary describes an aim to evaluate whether oral CoQ10 was safe and effective and whether its action was reflected in motor and neurobehavioral function. The registry record identifies McMaster Gross Motor Function (GMFM 88) as the primary outcome. This is a question about the recorded outcome and its comparison under the trial’s crossover arrangement; the posted statistics do not establish effects on every aspect of function or safety mentioned in the summary.
Participants were assigned to receive CoenzymeQ10 in one period and placebo in the other, with the order randomized. The record labels the sequences Placebo First and CoenzymeQ10 Frist. The reported primary analysis compares those sequence groups using a Wilcoxon (Mann-Whitney) test and estimates Kendall’s Tau B. The record says positive Tau B values favor CoenzymeQ10 and negative values favor placebo.
The registry record describes the primary measure as a difference based on scores at the end of the two periods, with a special rule for a participant becoming totally disabled. It does not report a separate primary result for neurobehavioral function. The statistical question addressed by the posted primary result is therefore narrower than the full set of aims in the summary: it concerns the specified GMFM outcome and the reported comparison of treatment sequences.
3. Trial Design
This was a Phase 3, randomized, triple-masked crossover trial sponsored by the University of Florida. The condition listed is mitochondrial diseases. The registry record reports enrollment of 24 and a completed status. The recorded start is 2007-01, and primary completion is recorded as 2013-05.
The interventions were CoenzymeQ10 and placebo. In a crossover design, participants receive more than one intervention in successive periods, and treatment order is part of the design. The record describes two treatment periods and says participants were randomized to receive CoenzymeQ10 in Period #1 or Period #2. It also describes placebo as assigned to either period. Its wording for the placebo schedule says “months 1-6” for Period #1, while the CoenzymeQ10 description says “Months 0-6”; both descriptions place Period #2 at months 7-12. The registry record does not explain this difference in wording.
The two reported sequence groups are Placebo First and CoenzymeQ10 Frist. The primary outcome is described as taken at 6 and 12 Months. For participants completing both periods, the record defines the analyzed score as the end-of-Period-2 score minus the end-of-Period-1 score. It also specifies a special scoring rule for total disability during either period. The record does not describe a washout interval, if any, or how possible period or carryover effects were handled.
The design matters for interpreting the result. The reported groups represent treatment order, not simply separate parallel groups that each received only one intervention. The posted analysis compares those orders, using the outcome definition in the registry record. The record does not report further detail about randomization procedures, masking roles, adherence, or the analysis population.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Months 0-6
| Stage | Placebo First | CoenzymeQ10 Frist |
|---|---|---|
| STARTED | 12 | 12 |
| COMPLETED | 7 | 8 |
| NOT COMPLETED | 5 | 4 |
Reasons for not completing this period:
| Reason | Placebo First | CoenzymeQ10 Frist |
|---|---|---|
| Withdrawal by Subject | 4 | 4 |
| Too disabled to score | 1 | 0 |
Months 7 - 12
| Stage | Placebo First | CoenzymeQ10 Frist |
|---|---|---|
| STARTED | 7 | 8 |
| COMPLETED | 6 | 7 |
| NOT COMPLETED | 1 | 1 |
Reasons for not completing this period:
| Reason | Placebo First | CoenzymeQ10 Frist |
|---|---|---|
| Withdrawal by Subject | 1 | 0 |
| Too disabled to score | 0 | 1 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: This is the set of subjects that met all eligibility requirements
| Characteristic | Unit | Placebo First | CoenzymeQ10 Frist | Total |
|---|---|---|---|---|
| Participants analysed | 12 | 12 | 24 | |
| Age, Continuous | years | 9.0 (SD 6.3) | 7.5 (SD 4.5) | 8.3 (SD 5.4) |
| Age, Categorical: <=18 years | Participants | 12 | 12 | 24 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 6 | 8 | 14 |
| Sex: Female, Male: Male | Participants | 6 | 4 | 10 |
| Region of Enrollment: United States | participants | 10 | 9 | 19 |
| Region of Enrollment: Canada | participants | 2 | 3 | 5 |
6. Primary Endpoint
The registry lists the primary outcome as McMaster Gross Motor Function (GMFM 88), measured over Taken at 6 and 12 Months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | units on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is McMaster Gross Motor Function (GMFM 88), reported in units on a scale. The registry record describes it as a validated scale ranging from 0 to 100, with higher scores indicating better gross motor function. A measure of gross motor function is relevant to the trial’s stated interest in motor function among children with mitochondrial disease.
Scores were taken at 6 and 12 Months. For a participant completing both periods, the record defines the analyzed score as the score at the end of Period 2 minus the score at the end of Period 1. This makes the endpoint a within-participant change across the two period-end measurements, as described in the record, rather than a single end-of-study score. For someone becoming totally disabled, the protocol description assigns plus infinity if this occurred in Period 1 and minus infinity if it occurred in Period 2. The record says this rule was intended to penalize the relevant period.
The outcome is summarized using a median and an inter-quartile range, and the comparison is described as a Wilcoxon test with Kendall’s Tau B as the effect-size estimate. These choices do not make the outcome a direct measure of every dimension of health or function. The posted primary endpoint is specifically the GMFM measure; separate posted primary results for neurobehavioral function are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The reported primary comparison is a Wilcoxon (Mann-Whitney) test, with Kendall’s Tau B reported as .015. Its 95% confidence interval runs from -0.44 to 0.48, and the reported p-value is 0.95. The record explains that positive Tau B values favor COQenzyme10 and negative values favor placebo. The estimate is close to zero, while the interval spans values in either direction and includes zero.
This result does not sharply distinguish the treatment sequences on the reported primary outcome. The confidence interval expresses uncertainty about the rank-based effect estimate: values across a broad range remain compatible with the data under the method used. It does not establish that the treatments are equivalent, nor does the p-value quantify the probability that either treatment is effective. No equivalence or non-inferiority margin is reported in the registry record.
The group summaries give a median of -0.002 for Placebo First, with listed bounds from -0.47 to 1.36, and a median of -0.12 for CoenzymeQ10 Frist, with listed bounds from -2.75 to 1.75. The record identifies the endpoint summary as median and inter-quartile range, but the arm-level fields do not label the listed bounds. They should therefore be read as posted bounds, not assigned a more specific interpretation. The reported sequence-group sizes are 6 and 8, respectively. The registry record reports enrollment of 24, but does not explain why the primary outcome analysis group sizes differ from the enrollment figure.
The significance test and the effect-size interval answer related but distinct questions. The p-value reports the result of the stated test under its null hypothesis; the interval indicates the precision of the estimated rank association. A large p-value is not evidence that there is no possible effect, especially when the interval allows effects in both directions. Conversely, a directionally positive or negative estimate alone would not establish a reliable treatment difference.
The outcome’s unusual scoring rule is important. For participants completing both periods, the difference is defined as Period 2 minus Period 1, and total disability is assigned an infinite value according to the period in which it occurred. That definition makes the interpretation dependent on treatment order and the prespecified scoring rule. The posted aggregate statistics do not allow readers to inspect individual trajectories or assess how many observations, if any, were affected by the disability rule.
The record does not report a threshold for a clinically meaningful difference on this endpoint. Accordingly, the posted estimate and its uncertainty should be interpreted as statistical evidence about the defined outcome, not as a judgment about the practical importance of a particular score difference. The result concerns the primary comparison as analyzed; it does not resolve effects on unreported outcomes, safety beyond the posted adverse-event summaries, or all possible outcomes in mitochondrial disease.
9. Statistical Methods Explained
What does the crossover structure mean?
In general, a crossover trial assigns treatment sequences: a participant receives one intervention in one period and the other intervention in another period. The order of treatment is consequently central to interpretation. The registry record labels the sequences Placebo First and CoenzymeQ10 Frist and describes measurements at the end of both periods. The primary score for participants completing both periods is defined as the later-period score minus the earlier-period score.
This design can make within-participant comparisons informative because each participant contributes measurements under both treatment periods. That general advantage depends on the assumptions and analysis being appropriate for the particular trial. Period effects, changes over time, and carryover from one period to the next can matter in crossover studies. Whether and how such issues were evaluated is not reported in the registry record. A washout interval is also not reported in the registry record.
Why use the Wilcoxon (Mann-Whitney) test?
The registry record names the Wilcoxon (Mann-Whitney) test for the primary comparison. In general, this is a rank-based method: it uses the ordering of observed values rather than relying on a comparison of means under a normal-distribution model. Rank methods can be useful when the data are skewed or when the outcome’s distribution makes a mean-based analysis less suitable. They do not automatically adjust for every aspect of a study design. The registry record does not explain further how the test was adapted, if at all, to the crossover structure.
The reported comparison is between the two treatment sequence groups. It should not be described as a simple comparison of two independent groups each assigned to a single treatment, because the record describes a crossover. The test’s interpretation also depends on how the endpoint was constructed, including the special rule for total disability. The analysis details beyond the named test and endpoint definition are not reported in the registry record.
What does Kendall’s Tau B describe?
The effect-size measure is Kendall’s Tau B. In general, Tau B is a rank association measure that describes how often pairs of observations are ordered consistently in two variables, with an adjustment for tied ranks. Its values are interpreted as a measure of association rather than as a difference in GMFM scale units. The registry record describes its direction: positive values favor COQenzyme10, and negative values favor placebo. Thus the reported .015 is not a median score change and should not be read as a change in GMFM points.
Rank effect measures can complement a significance test because they describe the direction and magnitude of an association in a way that is not tied to the original scale’s units. The confidence interval for Tau B communicates uncertainty around that estimate. The reported 95% interval, -0.44 to 0.48, includes zero and covers estimates favoring either direction. The registry record does not give a threshold for interpreting a Tau B value as clinically important.
How should the confidence interval and p-value be read?
In general, a confidence interval is a range of estimates compatible with the observed data and the method’s assumptions at the stated confidence level. It gives information about precision and direction that a single p-value cannot. A wide interval indicates limited precision; an interval that includes zero does not establish that the true effect is exactly zero. The reported interval is consistent with a range of rank associations on both sides of zero.
A p-value is, in general, a measure of how unusual data at least as inconsistent with a null hypothesis would be if that null model were true and the test assumptions held. It is not the probability that the null hypothesis is true, nor the probability that the result occurred by chance. The reported p-value of 0.95 is the result of the named test. It should be interpreted alongside the effect estimate, interval, design, and outcome definition, rather than treated as a stand-alone measure of importance.
What do the medians and bounds contribute?
The record identifies the endpoint summary as a median with inter-quartile range. In general, a median describes the central position of observed values and is less affected by extreme values than a mean. An inter-quartile range describes the middle half of a distribution. The posted arm-level summaries list medians and bounds, but do not explicitly identify the bounds in those fields. The primary description of the endpoint’s dispersion does not justify labeling those specific bounds as an inter-quartile range or as a confidence interval without an explicit label.
Arm-level summaries and the rank-based comparison serve different purposes. Medians describe the observed group summaries, while the Wilcoxon test and Tau B use the ordering of observations. Aggregate medians and bounds do not contain all of the individual ranks, so they are not enough to reproduce the reported test or effect estimate. The registry record does not post individual participant data.
What do the planning illustrations mean?
In general, sample-size calculations are planning tools: they show how many participants might be required under chosen assumptions about an effect, significance level, allocation, and statistical model. They are not evidence about the power achieved by an already completed trial. The record describes any planning illustration as based on the observed effect, two-sided significance level 0.05, equal allocation, and a normal approximation, with the target effect set as a fraction of the observed effect. Such illustrations do not establish the trial’s original design rationale, which is not reported in the registry record. They also do not transform the observed result into evidence that the trial had a particular observed power.
For this endpoint, interpretation is especially sensitive to the crossover arrangement and its outcome definition. A planning method based on a simplified model may not capture every feature of a rank-based analysis or the disability scoring rule. The registry record does not report enough detail to assess whether the planning assumptions reproduce the original trial’s design or analysis plan. Any sample-size illustration is therefore a conditional planning exercise, not a result of the trial.
10. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| Wilcoxon (Mann-Whitney) | Kendall's Tau B .015 | -0.44 to 0.48 | 0.950 |
The registry record reports the sponsor’s primary analysis as a Wilcoxon (Mann-Whitney) test with Kendall’s Tau B of .015, a 95% confidence interval from -0.44 to 0.48, and p = 0.95. These are the reported inferential results for the primary comparison. No separate recalculated estimate is stated in the registry record, so there is no competing recalculation to set against the sponsor’s analysis.
The posted medians and bounds cannot, by themselves, reproduce a rank-based test or Kendall’s Tau B. Those statistics depend on the ordering of the underlying observations, which is not available in the summary values. The sponsor’s stated analysis is therefore the relevant reported test result; the sequence-group summaries provide descriptive context, not enough information to rebuild the test.
The record does not report further detail about test implementation or an alternative adjusted analysis. The reported effect estimate and uncertainty should be interpreted according to the endpoint and crossover scoring rule described in the registry record. The interval spans zero and the reported p-value is large, but these findings do not prove equivalence or rule out effects in either direction.
11. Multiplicity, Missing Data and Analysis Populations
The registry record lists 3 outcome measures and 3 analyses, with no secondary outcomes posted. It identifies GMFM 88 as the primary outcome. The number of outcomes and analyses makes it important to know whether the primary question was selected in advance and whether any adjustment for multiple testing was planned. Whether a multiplicity adjustment was used, or how the analyses relate to the registered outcomes, is not reported in the registry record.
Multiplicity matters when several outcomes, time points, subgroups, or analyses are examined. In general, evaluating more statistical tests can increase the chance that at least one result appears unusual even when there is no corresponding effect. This does not mean every analysis must be adjusted in the same way; the appropriate approach depends on the prespecified questions and analysis plan. The registry record does not report enough detail to assess the analysis family or any control of multiplicity.
Missing-data handling is also not reported in the registry record. The record reports enrollment of 24 and primary outcome sequence groups of 6 and 8, but does not explain the difference or state why participants are not represented in those reported groups. It does not report whether observations were missing, whether participants failed to complete both periods, or what rules were used for incomplete outcome records. The special disability scoring rule is described, but it does not explain the handling of other missing values.
The analysis population is not reported in the registry record. It is therefore unclear whether the primary results include all randomized participants, only those with outcome measurements, or another analysis set. These omissions limit assessment of possible selection effects and of how broadly the reported estimate represents the randomized population. No assumptions about participant flow or missingness can be inferred from the posted group summaries alone.
12. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| CoenzymeQ10 | 1 | 19 | 5.3% |
| Placebo | 2 | 20 | 10.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| CoenzymeQ10 | 10 of 19 |
| Placebo | 10 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | CoenzymeQ10 | Placebo |
|---|---|---|---|
| Hospitalization for lumbar vertebrae fractures | Musculoskeletal and connective tissue disorders | 0 of 19 | 1 of 20 |
| Altered Mental State | Nervous system disorders | 0 of 19 | 1 of 20 |
| Hospitalization for seizure activity | Nervous system disorders | 1 of 19 | 0 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | CoenzymeQ10 | Placebo |
|---|---|---|---|
| GI Issues | Gastrointestinal disorders | 2 of 19 | 5 of 20 |
| Elevated Liver Enzymes | Blood and lymphatic system disorders | 1 of 19 | 1 of 20 |
| Ear Infection | Ear and labyrinth disorders | 1 of 19 | 1 of 20 |
| Neuromusclar Changes | Musculoskeletal and connective tissue disorders | 1 of 19 | 1 of 20 |
| Decreased White Blood Count | Blood and lymphatic system disorders | 1 of 19 | 0 of 20 |
| Low ANC Levels | Blood and lymphatic system disorders | 0 of 19 | 1 of 20 |
| Nose Bleed | General disorders | 0 of 19 | 1 of 20 |
| Fatigue | General disorders | 1 of 19 | 0 of 20 |
| Mental Status Change | Nervous system disorders | 1 of 19 | 0 of 20 |
| Rash | Skin and subcutaneous tissue disorders | 1 of 19 | 0 of 20 |
Serious events with the highest arm-level rate: hospitalization for seizure activity (5.3%), hospitalization for lumbar vertebrae fractures (5.0%), altered mental state (5.0%).
The adverse-event record lists serious adverse events for CoenzymeQ10 and placebo separately. For CoenzymeQ10, 19 participants were at risk and 1 was affected; for placebo, 20 were at risk and 2 were affected. Other adverse events are listed for 10 participants at risk in each group. These are counts as posted, not comparisons adjusted for exposure time or other differences between treatment periods.
The registry record lists the leading serious-event terms as Hospitalization for seizure activity at 5.3%, Hospitalization for lumbar vertebrae fractures at 5.0%, and Altered Mental State at 5.0%. These terms are not assigned to a treatment group in the posted list. The record does not specify the number of events for each term or provide enough context to interpret the percentages as comparative risks. Deaths are not reported in the registry record.
Adverse-event counts are descriptive. In a crossover trial, the period and treatment sequence are relevant to attribution, and these summary figures do not provide event timing or individual exposure details. The figures alone cannot establish whether an event was caused by a treatment or support a definitive safety comparison. The registry record’s adverse-event summaries should be read as reported counts and terms, not as a complete account of safety or a basis for medical advice.
13. What the Result Does, and Does Not, Mean
The posted primary result is an estimate of the rank-based association between treatment sequence and the defined GMFM outcome, not a direct estimate in GMFM scale points. The reported Tau B is .015, with a 95% confidence interval from -0.44 to 0.48 and p = 0.95. These results do not show a clear separation of the sequence groups under the reported test, and the interval leaves uncertainty in both directions.
That interpretation is narrower than saying the treatments are the same. A result that does not provide strong evidence of a difference does not establish equivalence. Equivalence requires a defined margin and an analysis designed to assess whether differences are confined within it; such a margin is not reported in the registry record. Nor does the result establish that there can be no effect in a subgroup or on a different outcome.
The endpoint is also specific: it is based on GMFM 88 scores at the end of the two periods, with a special rule for total disability. It addresses gross motor function as defined by that scale and scoring procedure. The trial summary refers more broadly to motor and neurobehavioral function, but separate posted primary results for neurobehavioral function are not reported in the registry record.
The design and reporting details constrain interpretation. This was a randomized crossover trial with two treatment sequences, yet the registry record does not report a washout interval, analysis-population definition, missing-data approach, or handling of period and carryover effects. The primary outcome groups are smaller than the enrollment figure, and the reason is not reported. These gaps make it difficult to assess how the reported comparison relates to all randomized participants.
The result should therefore be understood as the registry’s reported analysis of one specified endpoint, using the named rank-based method. It does not settle every question about CoenzymeQ10, placebo, mitochondrial disease, long-term outcomes, or safety. No treatment recommendation follows from these registry statistics alone.
14. Why This Trial Matters Statistically
This trial is statistically notable because its primary endpoint is analyzed within a crossover framework and includes a special rule for total disability. The outcome is not merely a conventional score change: for participants completing both periods, the score is defined as Period 2 minus Period 1, while total disability is assigned plus or minus infinity depending on when it occurs. Such a rule can materially affect the ordering of observations used by rank-based methods. Clear reporting of the rule is essential for understanding what the estimate represents.
The reported test and effect measure also illustrate why a p-value should not be read in isolation. The Wilcoxon (Mann-Whitney) p-value is 0.95, while the reported Kendall’s Tau B is .015 and its interval spans zero in both directions. The estimate, uncertainty, and test result together describe weak separation in the posted analysis with substantial uncertainty. They do not prove equivalence, and they do not provide a clinically meaningful threshold.
The distinction between treatment and sequence is another important statistical point. In a crossover trial, participants receive treatments in order, and period effects or carryover may influence observed outcomes. The registry record identifies a comparison of sequences but does not report how those potential features were handled analytically. Readers should not treat the reported sequence groups as parallel groups assigned only to one treatment.
Finally, enrollment and the primary analysis group sizes are reported but not reconciled, and missing-data and analysis-population details are not reported. These omissions affect evaluation of precision and representativeness. The record is useful for seeing how the endpoint and primary analysis were described, while also showing why complete reporting of participant flow and crossover-specific methods matters.
15. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
Several trial-specific reporting limitations make the primary result difficult to evaluate beyond the reported statistic. The registry record describes randomized treatment sequences and a crossover outcome, but does not report a washout interval or explain whether possible period and carryover effects were evaluated. The test is named as Wilcoxon (Mann-Whitney), yet further details on how it was applied to the crossover design are not reported in the registry record.
The record reports enrollment of 24, while the primary sequence-group summaries list 6 for Placebo First and 8 for CoenzymeQ10 Frist. The reason for the difference is not reported in the registry record. The analysis population, participant flow, and handling of missing outcome data are also not reported. Without those details, the analysis groups cannot be related confidently to everyone enrolled or randomized.
The primary endpoint description includes an unusual total-disability rule, but the registry record does not state how many observations were affected by it. The arm-level summaries provide medians and bounds, but do not explicitly label the bounds in those fields. The reported rank-based effect cannot be independently reconstructed from those summary values.
The registry record lists 3 outcome measures and 3 analyses, but does not report whether multiplicity was addressed or how the analyses were prespecified. It does not report a clinically meaningful threshold for GMFM 88 or an equivalence margin. These omissions limit interpretation of the clinical importance of the estimate and the overall inferential context. The adverse-event summary likewise lacks treatment attribution for the listed serious-event terms and deaths are not reported in the registry record.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Phase III Trial of Coenzyme Q10 in Mitochondrial Disease — NCT00432744.
- Registry record last updated 2017-09-11; status last verified 2017-09.
- Results first posted 2014-05-01.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
The registry record describes a completed, randomized, triple-masked Phase 3 crossover trial of CoenzymeQ10 and placebo for mitochondrial disease, sponsored by the University of Florida. Enrollment is recorded as 24. The start is 2007-01 and primary completion is 2013-05.
The primary outcome is McMaster Gross Motor Function (GMFM 88), measured at 6 and 12 Months and analyzed using the record’s period-difference and disability-scoring rules. The reported method is Wilcoxon (Mann-Whitney), with Kendall’s Tau B of .015, a 95% confidence interval from -0.44 to 0.48, and p = 0.95.
The record does not report the analysis population, missing-data handling, crossover washout details, or a multiplicity approach. The posted result does not establish equivalence, and the available safety summaries do not provide a definitive comparative safety assessment.