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The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.
The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients treated with triheptanoin | Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Duration of Hospitalization for Trigger Event | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | |
| Disposition on Discharge of Trigger Event | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | |
| Duration of Important Interventions for Trigger Event | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | |
| Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Duration of Hospitalization for Other Major Clinical Events | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | |
| Disposition on Discharge of Other Major Clinical Events | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment |
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Inclusion Criteria:
Exclusion Criteria:
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Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | United States | ||
| Kaiser Permanente |
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| Number of Important Medical Interventions for Other Major Clinical Events | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment |
| Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment |
| Los Angeles |
| California |
| 90027 |
| United States |
| Children's Hospital of Orange County | Orange | California | 92868 | United States |
| Stanford University | Palo Alto | California | 94304 | United States |
| Yale University | New Haven | Connecticut | 06511 | United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| University of South Florida | Tampa | Florida | 33606 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55455 | United States |
| Duke University | Durham | North Carolina | 27710 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |