Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

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

This study has been completed.
Sponsor:
Collaborator:
Santhera Pharmaceuticals
Information provided by (Responsible Party):
Michio Hirano, Columbia University
ClinicalTrials.gov Identifier:
NCT00887562
First received: April 23, 2009
Last updated: September 20, 2016
Last verified: September 2016
Results First Received: December 17, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Pharmacodynamics Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: MELAS Syndrome
Interventions: Drug: Idebenone
Other: 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
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


Participant Flow:   Overall Study
    Idebenone 900 mg/Day   Idebenone 2250 mg/Day   Placebo
STARTED   10   9   8 
COMPLETED   7   7   7 
NOT COMPLETED   3   2   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.
No text entered.

Reporting Groups
  Description
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

Total Total of all reporting groups

Baseline Measures
   Idebenone 900 mg/Day   Idebenone 2250 mg/Day   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 10   9   8   27 
Age 
[Units: Participants]
       
<=18 years   1   0   0   1 
Between 18 and 65 years   9   9   8   26 
>=65 years   0   0   0   0 
Gender 
[Units: Participants]
       
Female   9   5   6   20 
Male   1   4   2   7 
Race (NIH/OMB) 
[Units: Participants]
       
American Indian or Alaska Native   3   0   0   3 
Asian   0   1   0   1 
Native Hawaiian or Other Pacific Islander   0   0   0   0 
Black or African American   0   0   0   0 
White   7   8   8   23 
More than one race   0   0   0   0 
Unknown or Not Reported   0   0   0   0 
Region of Enrollment 
[Units: Participants]
       
United States   10   9   8   27 


  Outcome Measures
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1.  Primary:   Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy)   [ Time Frame: Up to 4 weeks from baseline ]

2.  Secondary:   Mean Change in Venous Lactate Concentration   [ Time Frame: Up to 4 weeks from baseline ]

3.  Secondary:   Mean Change in Score on the Fatigue Severity Scale (FSS)   [ Time Frame: Baseline and Week 4 ]


  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
Clinical variability in severity of disease among participants


  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI 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
e-mail: mh29@cumc.columbia.edu



Responsible Party: Michio Hirano, Columbia University
ClinicalTrials.gov Identifier: NCT00887562     History of Changes
Other Study ID Numbers: AAAC9240
SNT-II-007 ( Other Identifier: Santhera )
Study First Received: April 23, 2009
Results First Received: December 17, 2015
Last Updated: September 20, 2016
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration