Stopped: Sponsor decision not related to safety concerns
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or caregivers) to self-report information.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
LC-FAOD Management: Nutrition and Dosing Utilized to Control LC-FAOD
Timeframe: 10 years
LC-FAOD At-home Interventions: Types of Interventions Used
Timeframe: 10 years
LC-FAOD MCEs: Annualized Event Rate of all MCEs
Timeframe: 10 years
LC-FAOD MCEs: Annualized Event Days in the Emergency Department (ED)/Hospital for all MCEs
Timeframe: 10 years
LC-FAOD MCEs: Time to First MCE in all Newborn Patients (<=1 Year)
Timeframe: 10 years
Disease Management as Assessed by Physical Activity
Timeframe: 10 years
Patient Reported Outcomes: Infant and Toddler Quality of Life Questionnaire (ITQOL) Short Form
Timeframe: 10 years
Patient Reported Outcomes: Short-Form 10 (SF-10) Health Survey for Children
Timeframe: 10 years
Patient Reported Outcomes: Medical Outcomes Study 12-Item Short Form Version 2 (SF-12v2) for Adults
Timeframe: 10 years
Participant Survey: Patient and Caregiver-Reported Outcomes
Timeframe: 10 years
Participant Survey: Disease Symptoms
Timeframe: 10 years
Participant Survey: Schooling and Work Status
Timeframe: 10 years
Participant Survey: Absenteeism/Presenteeism
Timeframe: 10 years
Participant Survey: EuroQol Group 5-dimension 5-level (EQ-5D-5L) Instrument
Timeframe: 10 years
Healthcare Resource Utilization: LC-FAOD-related Medical Resource Use Over Time
Timeframe: 10 years