Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Clinical Trial of High Dose CoQ10 in ALS

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.
 
ClinicalTrials.gov Identifier: NCT00243932
Recruitment Status : Completed
First Posted : October 25, 2005
Results First Posted : March 17, 2011
Last Update Posted : February 28, 2014
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Hiroshi Mitsumoto, Columbia University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Amyotrophic Lateral Sclerosis
Lou Gehrig's Disease
Interventions Drug: coenzyme Q10
Drug: Placebo
Enrollment 185
Recruitment Details Subjects were randomized between April, 2005 and May, 2007. The last participant completed the trial March 2008.
Pre-assignment Details Exclusion criteria were forced vital capacity (FVC) of less than 60%, severe medical illness, or disease onset more than 5 years before study entry.
Arm/Group Title 2700mg CoQ10 Placebo 1,800 mg CoQ10
Hide Arm/Group Description 35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75. 35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75. 35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
Period Title: Stage 1: Dose Selection
Started 35 35 35
Completed 33 35 35
Not Completed 2 0 0
Reason Not Completed
Lost to Follow-up             2             0             0
Period Title: Stage 2: Efficacy
Started 40 [1] 40 [1] 0 [2]
Completed 40 40 0
Not Completed 0 0 0
[1]
40 newly randomized pts. Efficacy analysis included Stage 1 + Stage 2 patients
[2]
No new pts. randomized because selection procedure in Stage 1 selected the 2,700 mg dose.
Arm/Group Title 2700mg CoQ10 Placebo 1,800 mg CoQ10 Total
Hide Arm/Group Description 35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75. 35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75. 35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis. Total of all reporting groups
Overall Number of Baseline Participants 75 75 35 185
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 75 participants 35 participants 185 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
62
  82.7%
62
  82.7%
23
  65.7%
147
  79.5%
>=65 years
13
  17.3%
13
  17.3%
12
  34.3%
38
  20.5%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 75 participants 75 participants 35 participants 185 participants
56  (10.8) 56.9  (11) 58.9  (12.5) 56.9  (11.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 75 participants 75 participants 35 participants 185 participants
Female
35
  46.7%
29
  38.7%
10
  28.6%
74
  40.0%
Male
40
  53.3%
46
  61.3%
25
  71.4%
111
  60.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 75 participants 75 participants 35 participants 185 participants
75 75 35 185
1.Primary Outcome
Title Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
Hide Description The ALSFRSr, a questionnaire-based scale assessing daily living function ranging from 48 (best score) to 0 (worst), was administered to the patient, or to a proxy if the patient could not communicate effectively. Decline was defined as ALSFRSr at baseline minus ALSFRSr at month 9. Thus a positive value indicates worsening.
Time Frame 9 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 2700mg CoQ10 Placebo 1,800 mg CoQ10
Hide Arm/Group Description:

40 new subjects and 35 subjects from stage 1 all taking 2,700mg CoQ10 - total 75.

Stage 1 - 35 participants per group compared CoQ10 doses of 1,800mg and 2,700 mg/day and placebo.

40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
35 patients. Mean and SD are presented but this group was not included in the efficacy analysis because the 1,800 mg dose was discontinued after stage 1.
Overall Number of Participants Analyzed 73 75 35
Mean (Standard Deviation)
Unit of Measure: units on a scale
8.80  (7.34) 9.44  (8.82) 10.9  (9.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 2700mg CoQ10, Placebo
Comments Null hypothesis: 2,700mg CoQ10 is at least 20% superior to placebo.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.14
Comments [Not Specified]
Method futility (non-superiority)
Comments [Not Specified]
2.Secondary Outcome
Title The Change Over 9 Months in Forced Vital Capacity; Fatigue Severity Scale; Short Form-36; and 8OH2dG (a Biomarker of Oxidative Stress Measured in a Blood Sample).
Hide Description [Not Specified]
Time Frame 9 months
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 2700mg CoQ10 Placebo 1,800 mg CoQ10
Hide Arm/Group Description 35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75. 35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75. 35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
All-Cause Mortality
2700mg CoQ10 Placebo 1,800 mg CoQ10
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Hide Serious Adverse Events
2700mg CoQ10 Placebo 1,800 mg CoQ10
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/75 (24.00%)      19/75 (25.33%)      10/35 (28.57%)    
Blood and lymphatic system disorders       
Hemorrhage, GI *  0/75 (0.00%)  0 0/75 (0.00%)  0 1/35 (2.86%)  1
Cardiac disorders       
Cardiac ischemia/infarction *  0/75 (0.00%)  0 0/75 (0.00%)  0 1/35 (2.86%)  1
Gastrointestinal disorders       
Dehydration *  0/75 (0.00%)  0 2/75 (2.67%)  2 0/35 (0.00%)  0
General disorders       
Pain *  3/75 (4.00%)  4 0/75 (0.00%)  0 2/35 (5.71%)  2
Other *  14/75 (18.67%)  16 9/75 (12.00%)  9 1/35 (2.86%)  2
Musculoskeletal and connective tissue disorders       
Fracture *  0/75 (0.00%)  0 0/75 (0.00%)  0 1/35 (2.86%)  1
Respiratory, thoracic and mediastinal disorders       
Pneumonitis/pulmonary infiltrates *  3/75 (4.00%)  4 2/75 (2.67%)  3 1/35 (2.86%)  1
Pulmonary/Upper Respiratory - Other *  2/75 (2.67%)  2 3/75 (4.00%)  3 1/35 (2.86%)  1
Adult Respiratory Distress Syndrome (ARDS) *  0/75 (0.00%)  0 2/75 (2.67%)  2 0/35 (0.00%)  0
Aspiration *  0/75 (0.00%)  0 0/75 (0.00%)  0 1/35 (2.86%)  1
Surgical and medical procedures       
Peg placement *  5/75 (6.67%)  5 4/75 (5.33%)  4 2/35 (5.71%)  2
Vascular disorders       
Thrombosis/thrombus/embolism *  2/75 (2.67%)  2 2/75 (2.67%)  2 0/35 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
2700mg CoQ10 Placebo 1,800 mg CoQ10
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   63/75 (84.00%)      64/75 (85.33%)      35/35 (100.00%)    
Cardiac disorders       
Edema: limb *  8/75 (10.67%)  12 5/75 (6.67%)  5 1/35 (2.86%)  1
Gastrointestinal disorders       
Nausea *  13/75 (17.33%)  18 16/75 (21.33%)  19 4/35 (11.43%)  4
Constipation *  17/75 (22.67%)  19 10/75 (13.33%)  10 4/35 (11.43%)  6
Diarrhea *  8/75 (10.67%)  9 18/75 (24.00%)  22 6/35 (17.14%)  8
Gastrointestinal - Other *  8/75 (10.67%)  11 6/75 (8.00%)  6 5/35 (14.29%)  5
General disorders       
Pain *  21/75 (28.00%)  42 20/75 (26.67%)  21 10/35 (28.57%)  14
Other *  50/75 (66.67%)  244 51/75 (68.00%)  231 2/35 (5.71%)  2
Infections and infestations       
Infection with unknown ANC *  5/75 (6.67%)  6 6/75 (8.00%)  8 0/35 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Musculoskeletal/Soft Tissue - Other *  6/75 (8.00%)  8 6/75 (8.00%)  8 2/35 (5.71%)  2
Nervous system disorders       
Neurology - Other *  29/75 (38.67%)  68 18/75 (24.00%)  40 8/35 (22.86%)  16
Psychiatric disorders       
Mood alteration *  15/75 (20.00%)  20 7/75 (9.33%)  8 7/35 (20.00%)  7
Renal and urinary disorders       
Urinary frequency/urgency *  6/75 (8.00%)  7 5/75 (6.67%)  5 2/35 (5.71%)  2
Respiratory, thoracic and mediastinal disorders       
Pulmonary/Upper Respiratory - Other *  7/75 (9.33%)  11 11/75 (14.67%)  15 6/35 (17.14%)  6
Skin and subcutaneous tissue disorders       
Dermatology/Skin - Other *  10/75 (13.33%)  13 6/75 (8.00%)  6 5/35 (14.29%)  5
Surgical and medical procedures       
BIPAP/NIPPV *  8/75 (10.67%)  8 6/75 (8.00%)  6 3/35 (8.57%)  3
*
Indicates events were collected by non-systematic assessment
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
Layout table for Results Point of Contact information
Name/Title: Petra Kaufmann, MD, MSc
Organization: Columbia University
Phone: (212) 342-1252
EMail: pk88@columbia.edu
Layout table for additonal information
Responsible Party: Hiroshi Mitsumoto, Columbia University
ClinicalTrials.gov Identifier: NCT00243932    
Other Study ID Numbers: AAAA1536
R01NS048125 ( U.S. NIH Grant/Contract )
First Submitted: October 24, 2005
First Posted: October 25, 2005
Results First Submitted: March 29, 2010
Results First Posted: March 17, 2011
Last Update Posted: February 28, 2014