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Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

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NCT04632953 · readout ≤ 3,420 d

Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.

Phase
Status
Active, not recruiting
Study type
Observational
Enrollment
150actual
Sites
16
Countries
Canada, United States

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis
DateFiledBasisWhat it is
Start2021-11-30actualWhen the study began enrolling
Primary completionDec 2035estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionDec 2035estimatedThe whole study's end, after follow-up
First posted2020-11-17actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-06-03actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Intervention

  • Other: No Intervention

Primary outcomes

what the primary-completion date above is the date OF
Long-Term Safety of Patients With LC-FAOD as Assessed by Incidence, Severity, and Frequency of Serious Adverse Events (SAEs) and Adverse Events (AEs) in Pregnant and Lactating Patients with LC-FAOD
measured 10 Years
Long-Term Safety of Patients With LC-FAOD as Assessed by Outcomes of Pregnancy in Patients with LC-FAOD
measured 10 Years
Long-Term Safety of Patients With LC-FAOD as Assessed by Incidence, Frequency, and Severity of SAEs and AEs During the First Year of Life in Infants Born to Study Participants
measured 10 Years
Long-Term Safety of Patients With LC-FAOD as Assessed by Incidence of SAEs Assessed as Related to Triheptanoin Treatment by Study Investigator
measured 10 Years
Long-Term Safety of Patients With LC-FAOD as Assessed by Incidence of All Colon Cancer or Gastrointestinal (GI) Cancer, GI Dysplasia, and GI Neoplasia, SAEs and AEs Reported for All Patients With LC-FAOD
measured 10 Years

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