Trial Outcomes & Findings for Clinical Trial of High Dose CoQ10 in ALS (NCT NCT00243932)
NCT ID: NCT00243932
Last Updated: 2024-05-07
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
185 participants
Primary outcome timeframe
9 months
Results posted on
2024-05-07
Participant Flow
Subjects were randomized between April, 2005 and May, 2007. The last participant completed the trial March 2008.
Exclusion criteria were forced vital capacity (FVC) of less than 60%, severe medical illness, or disease onset more than 5 years before study entry.
Participant milestones
| Measure |
2700mg CoQ10
35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75.
|
Placebo
35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75.
|
1,800 mg CoQ10
35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
|
|---|---|---|---|
|
Stage 1: Dose Selection
STARTED
|
35
|
35
|
35
|
|
Stage 1: Dose Selection
COMPLETED
|
33
|
35
|
35
|
|
Stage 1: Dose Selection
NOT COMPLETED
|
2
|
0
|
0
|
|
Stage 2: Efficacy
STARTED
|
40
|
40
|
0
|
|
Stage 2: Efficacy
COMPLETED
|
40
|
40
|
0
|
|
Stage 2: Efficacy
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
2700mg CoQ10
35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75.
|
Placebo
35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75.
|
1,800 mg CoQ10
35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
|
|---|---|---|---|
|
Stage 1: Dose Selection
Lost to Follow-up
|
2
|
0
|
0
|
Baseline Characteristics
Clinical Trial of High Dose CoQ10 in ALS
Baseline characteristics by cohort
| Measure |
2700mg CoQ10
n=75 Participants
35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75.
|
Placebo
n=75 Participants
35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75.
|
1,800 mg CoQ10
n=35 Participants
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
n=185 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
62 Participants
n=5 Participants
|
62 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
147 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
38 Participants
n=4 Participants
|
|
Age, Continuous
|
56 years
STANDARD_DEVIATION 10.8 • n=5 Participants
|
56.9 years
STANDARD_DEVIATION 11 • n=7 Participants
|
58.9 years
STANDARD_DEVIATION 12.5 • n=5 Participants
|
56.9 years
STANDARD_DEVIATION 11.2 • n=4 Participants
|
|
Sex: Female, Male
Female
|
35 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
74 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
40 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
111 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
75 participants
n=5 Participants
|
75 participants
n=7 Participants
|
35 participants
n=5 Participants
|
185 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 9 monthsThe 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.
Outcome measures
| Measure |
2700mg CoQ10
n=73 Participants
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.
|
Placebo
n=75 Participants
40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
|
1,800 mg CoQ10
n=35 Participants
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.
|
|---|---|---|---|
|
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
|
8.80 units on a scale
Standard Deviation 7.34
|
9.44 units on a scale
Standard Deviation 8.82
|
10.9 units on a scale
Standard Deviation 9.3
|
SECONDARY outcome
Timeframe: 9 monthsPopulation: 1,800 mg CoQ10 group was not included in the efficacy analysis because the 1,800 mg dose was discontinued after stage 1.
The change over 9 months in fatigue severity scale. A 9-item scale measuring the impact of fatigue. Scores range from 7 (strongly disagree)-63 (strongly agree) with higher scores indicating a worse outcome.
Outcome measures
| Measure |
2700mg CoQ10
n=75 Participants
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.
|
Placebo
n=75 Participants
40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
|
1,800 mg CoQ10
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.
|
|---|---|---|---|
|
Change in Fatigue Severity Scale
|
0.71 score on a scale
Standard Deviation 1.21
|
0.88 score on a scale
Standard Deviation 1.51
|
—
|
SECONDARY outcome
Timeframe: 9 monthsPopulation: 1,800 mg CoQ10 group was not included in the efficacy analysis because the 1,800 mg dose was discontinued after stage 1.
The change over 9 months in forced vital capacity is the volume (liters) of gas that can be exhaled by maximum voluntary effort following deep inspiration. The best of three trials will be recorded. The result is recorded as percentage of predicted for age, height and weight.
Outcome measures
| Measure |
2700mg CoQ10
n=75 Participants
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.
|
Placebo
n=75 Participants
40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
|
1,800 mg CoQ10
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.
|
|---|---|---|---|
|
Change in Forced Vital Capacity
|
0.20 Percent of Predicted
Standard Deviation 0.15
|
0.17 Percent of Predicted
Standard Deviation 0.18
|
—
|
SECONDARY outcome
Timeframe: 9 monthsPopulation: 1,800 mg CoQ10 group was not included in the efficacy analysis because the 1,800 mg dose was discontinued after stage 1.
The change over 9 months in SF-36 score, which is a quality of life measure. Scores range from 0-100 with a higher score indicating a better outcome.
Outcome measures
| Measure |
2700mg CoQ10
n=75 Participants
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.
|
Placebo
n=75 Participants
40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
|
1,800 mg CoQ10
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.
|
|---|---|---|---|
|
Change in Short Form (SF)-36 Score (Physical)
|
4.22 score on a scale
Standard Deviation 8.02
|
6.04 score on a scale
Standard Deviation 6.85
|
—
|
SECONDARY outcome
Timeframe: 9 monthsPopulation: 1,800 mg CoQ10 group was not included in the efficacy analysis because the 1,800 mg dose was discontinued after stage 1.
The change over 9 months in SF-36 score, which is a quality of life measure. Scores range from 0-100 with a higher score indicating a better outcome.
Outcome measures
| Measure |
2700mg CoQ10
n=75 Participants
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.
|
Placebo
n=75 Participants
40 new subjects plus 35 subjects from stage 1 receiving placebo - total 75.
|
1,800 mg CoQ10
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.
|
|---|---|---|---|
|
Change in Short Form (SF)-36 Score (Mental)
|
2.87 score on a scale
Standard Deviation 11.78
|
5.01 score on a scale
Standard Deviation 11.69
|
—
|
Adverse Events
2700mg CoQ10
Serious events: 18 serious events
Other events: 63 other events
Deaths: 0 deaths
Placebo
Serious events: 19 serious events
Other events: 64 other events
Deaths: 0 deaths
1,800 mg CoQ10
Serious events: 10 serious events
Other events: 35 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
2700mg CoQ10
n=75 participants at risk
35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75.
|
Placebo
n=75 participants at risk
35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75.
|
1,800 mg CoQ10
n=35 participants at risk
35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
|
|---|---|---|---|
|
Surgical and medical procedures
Peg placement
|
6.7%
5/75 • Number of events 5
|
5.3%
4/75 • Number of events 4
|
5.7%
2/35 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulmonary infiltrates
|
4.0%
3/75 • Number of events 4
|
2.7%
2/75 • Number of events 3
|
2.9%
1/35 • Number of events 1
|
|
General disorders
Pain
|
4.0%
3/75 • Number of events 4
|
0.00%
0/75
|
5.7%
2/35 • Number of events 2
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
2.7%
2/75 • Number of events 2
|
2.7%
2/75 • Number of events 2
|
0.00%
0/35
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other
|
2.7%
2/75 • Number of events 2
|
4.0%
3/75 • Number of events 3
|
2.9%
1/35 • Number of events 1
|
|
General disorders
Other
|
18.7%
14/75 • Number of events 16
|
12.0%
9/75 • Number of events 9
|
2.9%
1/35 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Syndrome (ARDS)
|
0.00%
0/75
|
2.7%
2/75 • Number of events 2
|
0.00%
0/35
|
|
Gastrointestinal disorders
Dehydration
|
0.00%
0/75
|
2.7%
2/75 • Number of events 2
|
0.00%
0/35
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
0.00%
0/75
|
0.00%
0/75
|
2.9%
1/35 • Number of events 1
|
|
Cardiac disorders
Cardiac ischemia/infarction
|
0.00%
0/75
|
0.00%
0/75
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Fracture
|
0.00%
0/75
|
0.00%
0/75
|
2.9%
1/35 • Number of events 1
|
|
Blood and lymphatic system disorders
Hemorrhage, GI
|
0.00%
0/75
|
0.00%
0/75
|
2.9%
1/35 • Number of events 1
|
Other adverse events
| Measure |
2700mg CoQ10
n=75 participants at risk
35 subjects from stage 1 + 40 new subjects; all taking 2,700mg CoQ10 - total 75.
|
Placebo
n=75 participants at risk
35 subjects from stage 1 + 40 new subjects; all receiving placebo - total 75.
|
1,800 mg CoQ10
n=35 participants at risk
35 patients. This dose group was discontinued after stage 1; only the 2,700 mg group was compared with placebo in the efficacy analysis.
|
|---|---|---|---|
|
Nervous system disorders
Neurology - Other
|
38.7%
29/75 • Number of events 68
|
24.0%
18/75 • Number of events 40
|
22.9%
8/35 • Number of events 16
|
|
General disorders
Pain
|
28.0%
21/75 • Number of events 42
|
26.7%
20/75 • Number of events 21
|
28.6%
10/35 • Number of events 14
|
|
Gastrointestinal disorders
Nausea
|
17.3%
13/75 • Number of events 18
|
21.3%
16/75 • Number of events 19
|
11.4%
4/35 • Number of events 4
|
|
Gastrointestinal disorders
Constipation
|
22.7%
17/75 • Number of events 19
|
13.3%
10/75 • Number of events 10
|
11.4%
4/35 • Number of events 6
|
|
Gastrointestinal disorders
Diarrhea
|
10.7%
8/75 • Number of events 9
|
24.0%
18/75 • Number of events 22
|
17.1%
6/35 • Number of events 8
|
|
Psychiatric disorders
Mood alteration
|
20.0%
15/75 • Number of events 20
|
9.3%
7/75 • Number of events 8
|
20.0%
7/35 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other
|
9.3%
7/75 • Number of events 11
|
14.7%
11/75 • Number of events 15
|
17.1%
6/35 • Number of events 6
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other
|
13.3%
10/75 • Number of events 13
|
8.0%
6/75 • Number of events 6
|
14.3%
5/35 • Number of events 5
|
|
Surgical and medical procedures
BIPAP/NIPPV
|
10.7%
8/75 • Number of events 8
|
8.0%
6/75 • Number of events 6
|
8.6%
3/35 • Number of events 3
|
|
Gastrointestinal disorders
Gastrointestinal - Other
|
10.7%
8/75 • Number of events 11
|
8.0%
6/75 • Number of events 6
|
14.3%
5/35 • Number of events 5
|
|
Cardiac disorders
Edema: limb
|
10.7%
8/75 • Number of events 12
|
6.7%
5/75 • Number of events 5
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal/Soft Tissue - Other
|
8.0%
6/75 • Number of events 8
|
8.0%
6/75 • Number of events 8
|
5.7%
2/35 • Number of events 2
|
|
Renal and urinary disorders
Urinary frequency/urgency
|
8.0%
6/75 • Number of events 7
|
6.7%
5/75 • Number of events 5
|
5.7%
2/35 • Number of events 2
|
|
Infections and infestations
Infection with unknown ANC
|
6.7%
5/75 • Number of events 6
|
8.0%
6/75 • Number of events 8
|
0.00%
0/35
|
|
General disorders
Other
|
66.7%
50/75 • Number of events 244
|
68.0%
51/75 • Number of events 231
|
5.7%
2/35 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place