Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Clinical Trial

Official title: Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) Online Disease Monitoring Program (DMP)

Status Terminated

Clinical Trial Summary

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.

Clinical Trial Description

The online LC-FAOD DMP is a non-interventional, prospective, observational disease-monitoring program conducted through a mobile/web-based application developed by the Sponsor. Participant data will be captured via the mobile/web-based application and entered into the database designed and maintained by the Sponsor and/or its designee. Additional objectives of the study are to benefit the LC-FAOD community by developing a comprehensive database for use by the LC-FAOD community to characterize patient experience, inform disease management, and enable further research, utilize the digital platform to notify patients, caregivers, and relevant patient advocacy groups of research studies and clinical trials and share aggregated and de-identified outputs to facilitate the exchange of information with patients and/or caregivers.

Participants can be on any treatment in order to participate. Medical management of the disease should continue as directed by the patient's physician(s). Specifically, no treatments, investigational agents, or experimental interventions will be provided as part of this online LC-FAOD DMP. ;

Related Conditions & MeSH terms

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

• NCT number: NCT04812106
• Study type: Observational
• Source: Ultragenyx Pharmaceutical Inc
• Status: Terminated
• Start date: July 25, 2022
• Completion date: October 27, 2022