TerminatedNot Applicable

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Stopped: Sponsor decision not related to safety concerns

United States8 participantsStarted 2022-07-25

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Trial details

NCT IDNCT04812106

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-10-27

View on ClinicalTrials.gov More Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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