Phase 3
Completed N=24
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Mitochondrial Diseases
Source: ClinicalTrials.gov NCT00432744 ↗
Enrolled (actual)
24
Serious AEs
7.7%
Results posted
May 2014
Primary outcomePrimary: McMaster Gross Motor Function (GMFM 88) — -0.002; -0.12 units on a scale — p=0.95
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY McMaster Gross Motor Function (GMFM 88) |
-0.002; -0.12 | 0.95 |
| PRIMARY Pediatric Quality of Life Scale |
-1.1; -11.9 | 0.18 |
| PRIMARY Non-parametric Hotelling T-square Bivariate Analysis of GMGF 88 and OPeds QOL. |
7; 8 | 0.42 |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00432744). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.