Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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Brief Summary
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Detailed Description
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Conditions
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Interventions
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EPI-743
EPI-743 (oral solution \[100 mg/mL\] or liquid-filled capsules \[100 mg or 200 mg\]) will be administered per dose and schedule specified in the arm.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Participants with clinical diagnosis: Diagnosis of inherited mitochondrial disease absent genetic confirmation; Specifically, participants must meet the diagnostic criteria of "definite" or "probable" mitochondrial disease as defined by Bernier et al., 2002
3. Deemed by principal investigator to be within 90 days of end-of-life hospice/terminal care
4. Male or female age \> one year
5. Hematocrit within normal range for age group
6. Agreement to use contraception if within reproductive years
7. Participant or participant's guardian able to consent and comply with protocol requirements
8. Presence of caregiver to ensure study compliance
9. Abstention from use of all pill-form dietary supplements and non-prescribed medications (except as allowed by the investigator)
10. Abstention from foods or beverages or bars fortified with Coenzyme Q10, vitamin E, super-fortified "functional" foods or beverages
11. Abstention from use of idebenone
12. Clinically staged with a Mitochondrial Disease Scale such as the Newcastle Score
Exclusion Criteria
2. Clinical history of bleeding or abnormal prothrombin time (PT)/partial thromboplastin time (PTT) (excluding anticoagulation Rx)
3. Hepatic insufficiency with liver function tests (LFTs) greater than two times normal
4. Renal insufficiency requiring dialysis
5. Fat malabsorption syndromes precluding drug absorption
6. Any other concurrent inborn errors of metabolism
7. Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis
8. Pregnancy
1 Year
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Gregory Enns, MB, ChB
Role: PRINCIPAL_INVESTIGATOR
Stanford University
Locations
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UCLA
Los Angeles, California, United States
CHOC Children's Clinic
Orange, California, United States
Lucille Packard Children's Hospital
Palo Alto, California, United States
UCSD
San Diego, California, United States
Stanford Children's Health
Stanford, California, United States
Emory University
Decatur, Georgia, United States
CUMC (Columbia University)
New York, New York, United States
Carolinas Medical Center
Charlotte, North Carolina, United States
Akron Children's Hospital
Akron, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
MUSC
North Charleston, South Carolina, United States
Baylor College of Medicine
Houston, Texas, United States
UTH
Houston, Texas, United States
Naval Hospital, Bremerton
Bremerton, Washington, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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Other Identifiers
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EPI-2009-1
Identifier Type: -
Identifier Source: org_study_id
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