EPI-743 for Metabolism or Mitochondrial Disorders
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ClinicalTrials.gov Identifier: NCT01642056 |
Recruitment Status :
Completed
First Posted : July 17, 2012
Results First Posted : April 14, 2021
Last Update Posted : April 14, 2021
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Sponsor:
National Human Genome Research Institute (NHGRI)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
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Study Type | Interventional |
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Study Design | Allocation: Non-Randomized; Intervention Model: Crossover Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment |
Conditions |
Mitochondrial Disease Neurology Myopathy |
Interventions |
Drug: EPI-743 Drug: Placebo |
Enrollment | 20 |
Participant Flow
Recruitment Details | |
Pre-assignment Details | 20 subjects were consented. One subject died during first intervention due to progression of disease. Three subjects did not complete the second intervention. |
Arm/Group Title | EPI-743, Then Placebo | Placebo, Then EPI-743 |
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Received EPI-743, 15mg/kg up to a maximum dose of 200 mg orally or via gastric tube three times daily with meals, then placebo three times daily orally or via gastric tube with meals. EPI-743 is structurally related to Vitamin E and can be broadly classified as an antioxidant. | Received Placebo three times daily orally or via gastric tube with meals, then EPI-743, 15mg/kg up to a maximum dose of 200 mg three times daily orally or via gastric tube with meals. EPI-743 is structurally related to Vitamin E and can be broadly classified as an antioxidant. |
Period Title: First Intervention | ||
Started | 10 | 10 |
Completed | 9 | 10 |
Not Completed | 1 | 0 |
Period Title: Second Intervention | ||
Started | 9 | 10 |
Completed | 8 | 8 |
Not Completed | 1 | 2 |
Baseline Characteristics
Arm/Group Title | EPI-743, Then Placebo | Placebo, Then EPI-743 | Total | |
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Received EPI-743, 15mg/kg up to a maximum dose of 200 mg orally or via gastric tube three times daily with meals, then placebo three times daily orally or via gastric tube with meals. EPI-743 is structurally related to Vitamin E and can be broadly classified as an antioxidant. | Received Placebo three times daily orally or via gastric tube with meals, then EPI-743, 15mg/kg up to a maximum dose of 200 mg three times daily orally or via gastric tube with meals. EPI-743 is structurally related to Vitamin E and can be broadly classified as an antioxidant. | Total of all reporting groups | |
Overall Number of Baseline Participants | 10 | 10 | 20 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 10 participants | 10 participants | 20 participants | |
<=18 years |
10 100.0%
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10 100.0%
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20 100.0%
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Between 18 and 65 years |
0 0.0%
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0 0.0%
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0 0.0%
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>=65 years |
0 0.0%
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0 0.0%
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0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 10 participants | 10 participants | 20 participants | |
Female |
3 30.0%
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5 50.0%
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8 40.0%
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Male |
7 70.0%
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5 50.0%
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12 60.0%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 10 participants | 10 participants | 20 participants | |
Hispanic or Latino |
0 0.0%
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1 10.0%
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1 5.0%
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Not Hispanic or Latino |
9 90.0%
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9 90.0%
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18 90.0%
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Unknown or Not Reported |
1 10.0%
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0 0.0%
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1 5.0%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 10 participants | 10 participants | 20 participants | |
American Indian or Alaska Native |
0 0.0%
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0 0.0%
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0 0.0%
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Asian |
3 30.0%
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1 10.0%
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4 20.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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0 0.0%
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0 0.0%
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Black or African American |
1 10.0%
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0 0.0%
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1 5.0%
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White |
4 40.0%
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9 90.0%
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13 65.0%
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More than one race |
1 10.0%
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0 0.0%
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1 5.0%
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Unknown or Not Reported |
1 10.0%
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0 0.0%
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1 5.0%
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Outcome Measures
Adverse Events
Limitations and Caveats
All 20 randomized patients were included in the model to assess baseline characteristics. Of the 20 randomized, 19 provided 6-month outcomes (10 placebo, 9 treatment), who then crossed over their treatments and provided 8-month baseline scores. Of these, 16 provided 14-month outcomes (8 who had crossed over to treatment, and 8 who had crossed over to placebo).
More Information
Results Point of Contact
Name/Title: | Gahl, William |
Organization: | National Human Genome Research Institute |
Phone: | +1 301 402 2739 |
EMail: | gahlw@mail.nih.gov |
Publications:
Responsible Party: | National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) ) |
ClinicalTrials.gov Identifier: | NCT01642056 |
Other Study ID Numbers: |
120161 12-HG-0161 |
First Submitted: | July 14, 2012 |
First Posted: | July 17, 2012 |
Results First Submitted: | February 19, 2021 |
Results First Posted: | April 14, 2021 |
Last Update Posted: | April 14, 2021 |