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Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes (MELAS)

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ClinicalTrials.gov Identifier: NCT00887562
Recruitment Status : Completed
First Posted : April 24, 2009
Results First Posted : August 10, 2016
Last Update Posted : October 26, 2016
Sponsor:
Collaborator:
Santhera Pharmaceuticals
Information provided by (Responsible Party):
Michio Hirano, Columbia University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition MELAS Syndrome
Interventions Drug: Idebenone
Other: Placebo
Enrollment 27
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Hide Arm/Group Description

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

Period Title: Overall Study
Started 10 9 8
Completed 7 7 7
Not Completed 3 2 1
Arm/Group Title Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo Total
Hide Arm/Group Description

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

Total of all reporting groups
Overall Number of Baseline Participants 10 9 8 27
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 8 participants 27 participants
<=18 years
1
  10.0%
0
   0.0%
0
   0.0%
1
   3.7%
Between 18 and 65 years
9
  90.0%
9
 100.0%
8
 100.0%
26
  96.3%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 8 participants 27 participants
Female
9
  90.0%
5
  55.6%
6
  75.0%
20
  74.1%
Male
1
  10.0%
4
  44.4%
2
  25.0%
7
  25.9%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 8 participants 27 participants
American Indian or Alaska Native
3
  30.0%
0
   0.0%
0
   0.0%
3
  11.1%
Asian
0
   0.0%
1
  11.1%
0
   0.0%
1
   3.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
7
  70.0%
8
  88.9%
8
 100.0%
23
  85.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 9 participants 8 participants 27 participants
10 9 8 27
1.Primary Outcome
Title Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy)
Hide Description 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)
Time Frame Up to 4 weeks from baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Hide Arm/Group Description:

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

Overall Number of Participants Analyzed 7 7 7
Mean (Standard Deviation)
Unit of Measure: IU
-0.09  (0.95) 0.16  (1.49) -0.49  (1.18)
2.Secondary Outcome
Title Mean Change in Venous Lactate Concentration
Hide Description To compare the efficacy of 1 month treatment with 2 different doses of idebenone with that of placebo on venous lactate concentration
Time Frame Up to 4 weeks from baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Hide Arm/Group Description:

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

Overall Number of Participants Analyzed 7 7 7
Mean (Standard Deviation)
Unit of Measure: mM/L
-0.24  (1.18) 0.7  (1.31) -0.46  (1.07)
3.Secondary Outcome
Title Mean Change in Score on the Fatigue Severity Scale (FSS)
Hide Description

To 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

Time Frame Baseline and Week 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 900 mg/Day 2250 mg/Day Placebo
Hide Arm/Group Description:

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

Overall Number of Participants Analyzed 7 7 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
-3.8  (11) -1.3  (10) 4.3  (4.5)
Time Frame 6 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Hide Arm/Group Description

Idebenone 900 mg/day

Idebenone: 900 mg/day for 1 month

Idebenone 2250 mg/day

Idebenone: 2250 mg/day for one month

Placebo

Placebo: Placebo - No idebenone

All-Cause Mortality
Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/10 (0.00%)      0/9 (0.00%)      0/8 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Idebenone 900 mg/Day Idebenone 2250 mg/Day Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   10/10 (100.00%)      9/9 (100.00%)      8/8 (100.00%)    
Blood and lymphatic system disorders       
lymphadenopathy   1/10 (10.00%)  3 0/9 (0.00%)  0 0/8 (0.00%)  0
bilirubin decreased   2/10 (20.00%)  2 1/9 (11.11%)  2 1/8 (12.50%)  1
chloride decreased   0/10 (0.00%)  0 0/9 (0.00%)  0 2/8 (25.00%)  2
creatine phosphokinase increased   1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
glucose decreased   2/10 (20.00%)  3 0/9 (0.00%)  0 0/8 (0.00%)  0
glucose increase   0/10 (0.00%)  0 1/9 (11.11%)  1 1/8 (12.50%)  1
lactic acid increase   3/10 (30.00%)  4 2/9 (22.22%)  2 2/8 (25.00%)  2
carbon dioxide decrease   3/10 (30.00%)  3 0/9 (0.00%)  0 2/8 (25.00%)  2
hematocrit decrease   1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
hemoglobin decrease   1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
high density lipoprotein decrease   1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
monocyte increased   0/10 (0.00%)  0 3/9 (33.33%)  3 0/8 (0.00%)  0
red blood cell decreased   1/10 (10.00%)  1 0/9 (0.00%)  0 2/8 (25.00%)  2
lymphadenopathy *  1/10 (10.00%)  3 0/9 (0.00%)  0 0/8 (0.00%)  0
Cardiac disorders       
EKG abnormal   0/10 (0.00%)  0 2/9 (22.22%)  2 2/8 (25.00%)  2
Eye disorders       
visual impairment   0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  3
Gastrointestinal disorders       
abdominal discomfort * 1  0/10 (0.00%)  0 0/9 (0.00%)  0 2/8 (25.00%)  2
abdominal distention * 2  1/10 (10.00%)  1 2/9 (22.22%)  2 1/8 (12.50%)  2
abdominal pain *  1/10 (10.00%)  2 1/9 (11.11%)  2 0/8 (0.00%)  0
constipation *  1/10 (10.00%)  1 0/9 (0.00%)  0 0/8 (0.00%)  0
diarrhea *  2/10 (20.00%)  2 3/9 (33.33%)  5 1/8 (12.50%)  1
dispepsia *  1/10 (10.00%)  1 0/9 (0.00%)  0 0/8 (0.00%)  0
dysphagia *  1/10 (10.00%)  1 0/9 (0.00%)  0 0/8 (0.00%)  0
epigastric discomfort *  1/10 (10.00%)  1 0/9 (0.00%)  0 0/8 (0.00%)  0
flatulence *  0/10 (0.00%)  0 1/9 (11.11%)  1 1/8 (12.50%)  1
nausea *  3/10 (30.00%)  5 0/9 (0.00%)  0 0/8 (0.00%)  0
General disorders       
chest pain *  0/10 (0.00%)  0 1/9 (11.11%)  1 1/8 (12.50%)  1
fatigue *  5/10 (50.00%)  8 1/9 (11.11%)  2 3/8 (37.50%)  3
feeling abnormal *  1/10 (10.00%)  1 1/9 (11.11%)  1 1/8 (12.50%)  1
irratibility *  0/10 (0.00%)  0 0/9 (0.00%)  0 1/8 (12.50%)  2
Infections and infestations       
influenza *  1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
sinusitus *  1/10 (10.00%)  1 1/9 (11.11%)  1 1/8 (12.50%)  1
urinary tract infection *  1/10 (10.00%)  1 0/9 (0.00%)  0 1/8 (12.50%)  1
Musculoskeletal and connective tissue disorders       
arthralgia *  1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
muscle weakness *  1/10 (10.00%)  2 0/9 (0.00%)  0 0/8 (0.00%)  0
myalgia *  2/10 (20.00%)  2 0/9 (0.00%)  0 1/8 (12.50%)  1
pain in extremity *  1/10 (10.00%)  1 1/9 (11.11%)  1 0/8 (0.00%)  0
Nervous system disorders       
dizziness *  1/10 (10.00%)  2 0/9 (0.00%)  0 1/8 (12.50%)  2
headache *  3/10 (30.00%)  6 2/9 (22.22%)  2 4/8 (50.00%)  14
hypoaesthesia *  1/10 (10.00%)  2 1/9 (11.11%)  2 0/8 (0.00%)  0
migraine *  3/10 (30.00%)  3 0/9 (0.00%)  0 3/8 (37.50%)  7
petit mal eplipsey *  2/10 (20.00%)  6 0/9 (0.00%)  0 1/8 (12.50%)  1
somnolence *  0/10 (0.00%)  0 1/9 (11.11%)  1 1/8 (12.50%)  1
Psychiatric disorders       
nervousness *  1/10 (10.00%)  2 0/9 (0.00%)  0 1/8 (12.50%)  1
Renal and urinary disorders       
albumin in urine   1/10 (10.00%)  1 1/9 (11.11%)  1 1/8 (12.50%)  1
glucose in urine increased   1/10 (10.00%)  1 2/9 (22.22%)  2 0/8 (0.00%)  0
ketone in urine increased   0/10 (0.00%)  0 2/9 (22.22%)  2 0/8 (0.00%)  0
urine leukocyte esterase increase   4/10 (40.00%)  4 1/9 (11.11%)  1 1/8 (12.50%)  1
pollakiuria *  0/10 (0.00%)  0 2/9 (22.22%)  2 0/8 (0.00%)  0
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, not serious AE
2
Term from vocabulary, not serious
Clinical variability in severity of disease among participants
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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: Michio Hirano, MD
Organization: Columbia University
Phone: 12123051048
Responsible Party: Michio Hirano, Columbia University
ClinicalTrials.gov Identifier: NCT00887562     History of Changes
Other Study ID Numbers: AAAC9240
SNT-II-007 ( Other Identifier: Santhera )
First Submitted: April 23, 2009
First Posted: April 24, 2009
Results First Submitted: December 17, 2015
Results First Posted: August 10, 2016
Last Update Posted: October 26, 2016