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)
NCT number | NCT04812106 |
Other study ID # | UX007-CL402 |
Secondary ID | |
Status | Terminated |
Phase | |
First received | |
Last updated | |
Start date | July 25, 2022 |
Est. completion date | October 27, 2022 |
Verified date | November 2022 |
Source | Ultragenyx Pharmaceutical Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Terminated |
Enrollment | 8 |
Est. completion date | October 27, 2022 |
Est. primary completion date | October 27, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Ultragenyx Pharmaceutical Inc |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | LC-FAOD Management: Nutrition and Dosing Utilized to Control LC-FAOD | 10 years | ||
Primary | LC-FAOD At-home Interventions: Types of Interventions Used | Interventions used to prevent or mitigate Major Clinical Events (MCEs) | 10 years | |
Primary | LC-FAOD MCEs: Annualized Event Rate of all MCEs | MCEs include skeletal myopathy (rhabdomyolysis), hepatic (hypoglycemia, liver dysfunction), and cardiac disease (cardiomyopathy) events. An MCE is defined as any visit to the ED/acute care, hospitalization, emergency intervention (ie, any unscheduled administration of therapeutics in the clinic), or any similar event whether caused primarily by LC-FAOD or by an intercurrent illness complicated by LC-FAOD | 10 years | |
Primary | LC-FAOD MCEs: Annualized Event Days in the Emergency Department (ED)/Hospital for all MCEs | 10 years | ||
Primary | LC-FAOD MCEs: Time to First MCE in all Newborn Patients (<=1 Year) | 10 years | ||
Primary | Disease Management as Assessed by Physical Activity | 10 years | ||
Primary | Patient Reported Outcomes: Infant and Toddler Quality of Life Questionnaire (ITQOL) Short Form | 10 years | ||
Primary | Patient Reported Outcomes: Short-Form 10 (SF-10) Health Survey for Children | 10 years | ||
Primary | Patient Reported Outcomes: Medical Outcomes Study 12-Item Short Form Version 2 (SF-12v2) for Adults | 10 years | ||
Primary | Participant Survey: Patient and Caregiver-Reported Outcomes | 10 years | ||
Primary | Participant Survey: Disease Symptoms | 10 years | ||
Primary | Participant Survey: Schooling and Work Status | 10 years | ||
Primary | Participant Survey: Absenteeism/Presenteeism | 10 years | ||
Primary | Participant Survey: EuroQol Group 5-dimension 5-level (EQ-5D-5L) Instrument | 10 years | ||
Primary | Healthcare Resource Utilization: LC-FAOD-related Medical Resource Use Over Time | 10 years |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04632953 -
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
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Completed |
NCT03768817 -
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
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Completed |
NCT01886378 -
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
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Phase 2 | |
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A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD
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