Trials / Completed
CompletedNCT03768817
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 20 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- —
Summary
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.
Detailed description
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | No Intervention | This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol. |
Timeline
- Start date
- 2019-01-30
- Primary completion
- 2020-06-08
- Completion
- 2020-06-08
- First posted
- 2018-12-07
- Last updated
- 2020-08-28
Locations
15 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT03768817. Inclusion in this directory is not an endorsement.