Trial record 1 of 23 for:    coenzyme Q10 AND mitochondrial
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Phase III Trial of Coenzyme Q10 in Mitochondrial Disease

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT00432744
First received: February 6, 2007
Last updated: April 30, 2014
Last verified: April 2014
Results First Received: April 11, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Mitochondrial Diseases
Interventions: Drug: CoenzymeQ10
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Placebo First

Patients will be randomized to receive Placebo in Period #1 (Months 0-6) and CoenzymeQ10 given in Period #2( Months 7-12.)

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.

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.

CoenzymeQ10 Frist

Patients will be randomized to receive CoenzymeQ10 in Period #1 (Months 0-6) and Placebo given in Period #2( Months 7-12.)

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: 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.


Participant Flow for 2 periods

Period 1:   Months 0-6
    Placebo First     CoenzymeQ10 Frist  
STARTED     12     12  
COMPLETED     7     8 [1]
NOT COMPLETED     5     4  
Withdrawal by Subject                 4                 4  
Too disabled to score                 1                 0  
[1] One patient in period 1 "other reason" for loss became too disabled to complete the 6 month test.

Period 2:   Months 7 - 12
    Placebo First     CoenzymeQ10 Frist  
STARTED     7     8  
COMPLETED     6     7  
NOT COMPLETED     1     1  
Withdrawal by Subject                 1                 0  
Too disabled to score                 0                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
This is the set of subjects that met all eligibility requirements

Reporting Groups
  Description
Placebo First

Patients will be randomized to receive Placebo in Period #1 (Months 0-6) and CoenzymeQ10 given in Period #2( Months 7-12.)

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.

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.

CoenzymeQ10 Frist

Patients will be randomized to receive CoenzymeQ10 in Period #1 (Months 0-6) and Placebo given in Period #2( Months 7-12.)

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: 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.

Total Total of all reporting groups

Baseline Measures
    Placebo First     CoenzymeQ10 Frist     Total  
Number of Participants  
[units: participants]
  12     12     24  
Age  
[units: years]
Mean ± Standard Deviation
  9.0  ± 6.3     7.5  ± 4.5     8.3  ± 5.4  
Age  
[units: participants]
     
<=18 years     12     12     24  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     6     8     14  
Male     6     4     10  
Region of Enrollment  
[units: participants]
     
United States     10     9     19  
Canada     2     3     5  



  Outcome Measures
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1.  Primary:   McMaster Gross Motor Function (GMFM 88)   [ Time Frame: Taken at 6 and 12 Months ]

2.  Primary:   Pediatric Quality of Life Scale   [ Time Frame: At 6 and 12 Months ]

3.  Primary:   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. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The trial intended to accrual 40 subjects, but accrual was below expectations and the granting agency turned down a request for extended accrual.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Peter W Stacpoole
Organization: University of Florida
phone: 352-273-9023
e-mail: pws@ufl.edu


Publications:
Kerr DS. Treatment of congenital lactic acidosis: a review. Intern Pediatr, 1995;10:75-81.


Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT00432744     History of Changes
Other Study ID Numbers: 1 R01 FD003032-01A1
Study First Received: February 6, 2007
Results First Received: April 11, 2014
Last Updated: April 30, 2014
Health Authority: United States: Food and Drug Administration