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Phase III Trial of Coenzyme Q10 in Mitochondrial Disease

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00432744
First Posted: February 8, 2007
Last Update Posted: September 11, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
FDA Office of Orphan Products Development
Food and Drug Administration
Information provided by (Responsible Party):
University of Florida
Results First Submitted: April 11, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, 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 
Overall Participants Analyzed 
[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]
Count of Participants
     
<=18 years      12 100.0%      12 100.0%      24 100.0% 
Between 18 and 65 years      0   0.0%      0   0.0%      0   0.0% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      6  50.0%      8  66.7%      14  58.3% 
Male      6  50.0%      4  33.3%      10  41.7% 
Region of Enrollment 
[Units: Participants]
     
United States   10   9   19 
Canada   2   3   5 


  Outcome Measures
  Show All Outcome Measures

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: 1R01FD003032-01A1 ( U.S. FDA Grant/Contract )
R01FD003032-01A1 ( U.S. FDA Grant/Contract )
First Submitted: February 6, 2007
First Posted: February 8, 2007
Results First Submitted: April 11, 2014
Results First Posted: May 1, 2014
Last Update Posted: September 11, 2017