Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
NCT ID: NCT03768817
Last Updated: 2020-08-28
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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COMPLETED
20 participants
OBSERVATIONAL
2019-01-30
2020-06-08
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
RETROSPECTIVE
Study Groups
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Patients treated with triheptanoin
Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND
No Intervention
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
Interventions
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No Intervention
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
Eligibility Criteria
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Inclusion Criteria
* Treatment initiation with triheptanoin was via eIND before 01 September 2018
* Treated with triheptanoin in the USA
* Willing and able to provide written, signed informed consent, or where appropriate for subjects under the age of 18, or provide written assent and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. To obtain and review medical records of deceased individuals, informed consent from next of kin or appropriate legal entity will be obtained, as applicable.
Exclusion Criteria
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc
Locations
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Children's Hospital of Los Angeles
Los Angeles, California, United States
Kaiser Permanente
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
Stanford University
Palo Alto, California, United States
Yale University
New Haven, Connecticut, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
University of South Florida
Tampa, Florida, United States
Emory University
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
Duke University
Durham, North Carolina, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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Other Identifiers
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UX007-CL003
Identifier Type: -
Identifier Source: org_study_id
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