Status:
TERMINATED
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Eligibility:
All Genders
Brief 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 platf...
Detailed 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 b...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Diagnosis of LC-FAOD including: carnitine palmitoyltransferase (CPT I or CPT II) deficiency, very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency, trifunctional protein (TFP) deficiency, or carnitine-acylcarnitine translocase (CACT) deficiency.
- Willing and able to provide consent or, if a minor, provide assent and informed consent by their legally authorized representative.
- Access to the internet and an internet-enabled device.
- Reside in the geographical regions where the platform is supported and approved by applicable IRB/IEC and /or health authority.
- Not receiving triheptanoin through an Ultragenyx-sponsored interventional clinical trial or Ultragenyx Investigator Sponsored Trial (IST). NOTE: Individuals are eligible to participate in this online DMP if they previously participated in UX007 clinical trials, are currently participating in the in clinic UX007-CL401 (NCT04632953), are receiving triheptanoin through Expanded Access or Compassionate Use programs, or are receiving commercially available triheptanoin via prescription.
Exclusion
Key Trial Info
Start Date :
July 25 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 27 2022
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT04812106
Start Date
July 25 2022
End Date
October 27 2022
Last Update
November 18 2022
Active Locations (1)
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1
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224