Status:
COMPLETED
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Lead Sponsor:
University of Florida
Collaborating Sponsors:
FDA Office of Orphan Products Development
Food and Drug Administration (FDA)
Conditions:
Mitochondrial Diseases
Eligibility:
All Genders
12-17 years
Phase:
PHASE3
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 mitochondria...
Detailed Description
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 als...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2013
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT00432744
Start Date
January 1 2007
End Date
May 1 2013
Last Update
September 11 2017
Active Locations (3)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45267
2
Case Western Reserve University
Cleveland, Ohio, United States, 44106
3
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8