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Clinical Trial Details — Status: Terminated

Administrative data

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

Study information

Verified date November 2022
Source Ultragenyx Pharmaceutical Inc
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

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.


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.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms

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

Intervention

Other:
No Intervention
No Intervention

Locations

Country Name City State
United States Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania

Sponsors (1)

Lead Sponsor Collaborator
Ultragenyx Pharmaceutical Inc

Country where clinical trial is conducted

United States, 

Outcome

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
See also
  Status Clinical Trial Phase
Recruiting NCT04632953 - Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Completed NCT03768817 - Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
Completed NCT01886378 - A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Phase 2
Recruiting NCT05933200 - A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD Phase 3