Trial Outcomes & Findings for Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes (NCT NCT00887562)
NCT ID: NCT00887562
Last Updated: 2016-10-26
Results Overview
To compare the efficacy of 1 month treatment with 2 different doses of idebenone with that of placebo on cerebral lactate concentration as measured by magnetic resonance spectroscopy (MRS)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
27 participants
Primary outcome timeframe
Up to 4 weeks from baseline
Results posted on
2016-10-26
Participant Flow
Participant milestones
| Measure |
Idebenone 900 mg/Day
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
Placebo
Placebo: Placebo - No idebenone
|
|---|---|---|---|
|
Overall Study
STARTED
|
10
|
9
|
8
|
|
Overall Study
COMPLETED
|
7
|
7
|
7
|
|
Overall Study
NOT COMPLETED
|
3
|
2
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
Baseline characteristics by cohort
| Measure |
Idebenone 900 mg/Day
n=10 Participants
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
n=9 Participants
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
n=8 Participants
Placebo
Placebo: Placebo - No idebenone
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
9 participants
n=7 Participants
|
8 participants
n=5 Participants
|
27 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Up to 4 weeks from baselineTo compare the efficacy of 1 month treatment with 2 different doses of idebenone with that of placebo on cerebral lactate concentration as measured by magnetic resonance spectroscopy (MRS)
Outcome measures
| Measure |
Idebenone 900 mg/Day
n=7 Participants
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
n=7 Participants
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
n=7 Participants
Placebo
Placebo: Placebo - No idebenone
|
|---|---|---|---|
|
Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy)
|
-0.09 IU
Standard Deviation 0.95
|
0.16 IU
Standard Deviation 1.49
|
-0.49 IU
Standard Deviation 1.18
|
SECONDARY outcome
Timeframe: Up to 4 weeks from baselineTo compare the efficacy of 1 month treatment with 2 different doses of idebenone with that of placebo on venous lactate concentration
Outcome measures
| Measure |
Idebenone 900 mg/Day
n=7 Participants
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
n=7 Participants
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
n=7 Participants
Placebo
Placebo: Placebo - No idebenone
|
|---|---|---|---|
|
Mean Change in Venous Lactate Concentration
|
-0.24 mM/L
Standard Deviation 1.18
|
0.7 mM/L
Standard Deviation 1.31
|
-0.46 mM/L
Standard Deviation 1.07
|
SECONDARY outcome
Timeframe: Baseline and Week 4To assess changes following 1 month treatment with 2 different doses of idebenone with that of placebo in fatigue as assessed by the Fatigue Severity Scale (FSS). Scale score minimum is 9 (least fatigue) and maximum is 63 (maximum fatigue). Scores of 36 or less indicate possibility that patient may not be suffering from fatigue, while scores 36 and over suggest suffering from fatigue
Outcome measures
| Measure |
Idebenone 900 mg/Day
n=7 Participants
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
n=7 Participants
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
n=7 Participants
Placebo
Placebo: Placebo - No idebenone
|
|---|---|---|---|
|
Mean Change in Score on the Fatigue Severity Scale (FSS)
|
-3.8 units on a scale
Standard Deviation 11
|
-1.3 units on a scale
Standard Deviation 10
|
4.3 units on a scale
Standard Deviation 4.5
|
Adverse Events
Idebenone 900 mg/Day
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Idebenone 2250 mg/Day
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Idebenone 900 mg/Day
n=10 participants at risk
Idebenone 900 mg/day
Idebenone: 900 mg/day for 1 month
|
Idebenone 2250 mg/Day
n=9 participants at risk
Idebenone 2250 mg/day
Idebenone: 2250 mg/day for one month
|
Placebo
n=8 participants at risk
Placebo
Placebo: Placebo - No idebenone
|
|---|---|---|---|
|
Gastrointestinal disorders
abdominal discomfort
|
0.00%
0/10 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Gastrointestinal disorders
abdominal distention
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
12.5%
1/8 • Number of events 2 • 6 weeks
|
|
Gastrointestinal disorders
abdominal pain
|
10.0%
1/10 • Number of events 2 • 6 weeks
|
11.1%
1/9 • Number of events 2 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Gastrointestinal disorders
constipation
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Gastrointestinal disorders
diarrhea
|
20.0%
2/10 • Number of events 2 • 6 weeks
|
33.3%
3/9 • Number of events 5 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Gastrointestinal disorders
dispepsia
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Gastrointestinal disorders
dysphagia
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Gastrointestinal disorders
epigastric discomfort
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Gastrointestinal disorders
flatulence
|
0.00%
0/10 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Gastrointestinal disorders
nausea
|
30.0%
3/10 • Number of events 5 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
General disorders
chest pain
|
0.00%
0/10 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
General disorders
fatigue
|
50.0%
5/10 • Number of events 8 • 6 weeks
|
11.1%
1/9 • Number of events 2 • 6 weeks
|
37.5%
3/8 • Number of events 3 • 6 weeks
|
|
General disorders
feeling abnormal
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
General disorders
irratibility
|
0.00%
0/10 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 2 • 6 weeks
|
|
Blood and lymphatic system disorders
lymphadenopathy
|
10.0%
1/10 • Number of events 3 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Eye disorders
visual impairment
|
0.00%
0/10 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 3 • 6 weeks
|
|
Infections and infestations
influenza
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Infections and infestations
sinusitus
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Infections and infestations
urinary tract infection
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Renal and urinary disorders
albumin in urine
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
bilirubin decreased
|
20.0%
2/10 • Number of events 2 • 6 weeks
|
11.1%
1/9 • Number of events 2 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
chloride decreased
|
0.00%
0/10 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Blood and lymphatic system disorders
creatine phosphokinase increased
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
glucose decreased
|
20.0%
2/10 • Number of events 3 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Blood and lymphatic system disorders
glucose increase
|
0.00%
0/10 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
lactic acid increase
|
30.0%
3/10 • Number of events 4 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Blood and lymphatic system disorders
carbon dioxide decrease
|
30.0%
3/10 • Number of events 3 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Cardiac disorders
EKG abnormal
|
0.00%
0/10 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Renal and urinary disorders
glucose in urine increased
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Blood and lymphatic system disorders
hematocrit decrease
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Blood and lymphatic system disorders
hemoglobin decrease
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Blood and lymphatic system disorders
high density lipoprotein decrease
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Blood and lymphatic system disorders
monocyte increased
|
0.00%
0/10 • 6 weeks
|
33.3%
3/9 • Number of events 3 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Blood and lymphatic system disorders
red blood cell decreased
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
25.0%
2/8 • Number of events 2 • 6 weeks
|
|
Renal and urinary disorders
ketone in urine increased
|
0.00%
0/10 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Renal and urinary disorders
urine leukocyte esterase increase
|
40.0%
4/10 • Number of events 4 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Musculoskeletal and connective tissue disorders
muscle weakness
|
10.0%
1/10 • Number of events 2 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
20.0%
2/10 • Number of events 2 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Musculoskeletal and connective tissue disorders
pain in extremity
|
10.0%
1/10 • Number of events 1 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Nervous system disorders
dizziness
|
10.0%
1/10 • Number of events 2 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 2 • 6 weeks
|
|
Nervous system disorders
headache
|
30.0%
3/10 • Number of events 6 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
50.0%
4/8 • Number of events 14 • 6 weeks
|
|
Nervous system disorders
hypoaesthesia
|
10.0%
1/10 • Number of events 2 • 6 weeks
|
11.1%
1/9 • Number of events 2 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
|
Nervous system disorders
migraine
|
30.0%
3/10 • Number of events 3 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
37.5%
3/8 • Number of events 7 • 6 weeks
|
|
Nervous system disorders
petit mal eplipsey
|
20.0%
2/10 • Number of events 6 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Nervous system disorders
somnolence
|
0.00%
0/10 • 6 weeks
|
11.1%
1/9 • Number of events 1 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Psychiatric disorders
nervousness
|
10.0%
1/10 • Number of events 2 • 6 weeks
|
0.00%
0/9 • 6 weeks
|
12.5%
1/8 • Number of events 1 • 6 weeks
|
|
Renal and urinary disorders
pollakiuria
|
0.00%
0/10 • 6 weeks
|
22.2%
2/9 • Number of events 2 • 6 weeks
|
0.00%
0/8 • 6 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60