Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Ultragenyx

Status: Enrolling

Conditions: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT04632953

UX007-CL401

Details and patient eligibility

About

The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.

Full description

The LC-FAOD Disease Monitoring Program (DMP) is an international, long-term, retrospective and prospective outcomes study aiming to collect safety and effectiveness data for triheptanoin and the natural history of LC-FAOD for a study duration of up to 10 years from adult and pediatric patients with LC-FAOD, with any previous disease management, regardless of prior treatment with triheptanoin, those who have previously participated in triheptanoin clinical trials, and those who received triheptanoin through an Expanded Access Program (EAP).

Patients enrolling in the LC-FAOD DMP will be managed at the discretion of their physicians and may or may not be treated with triheptanoin during the course of the study. Patients will have access to triheptanoin only through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

Enrollment: 150 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of any LC-FAOD subtype. Diagnosis must be confirmed by results of acylcarnitine profiles and/or genetic testing results obtained from medical records or equivalent documentation.
• Willing and able to comply with all study procedures.
• Willing and able to provide consent or, if a minor, provide assent and informed consent by their legally authorized representative.
• Females of childbearing potential who become pregnant during the study will be invited to remain in the study. Pregnant females with LC-FAOD will be informed of the study and invited to enroll.

Exclusion criteria

• Presence of a concurrent disease or condition that would interfere with study participation or affect patient's safety in the opinion of the Investigator.
• Presence or history of any condition that, in the view of the Investigator, places the patient at high risk of not completing the study or would affect the interpretation of study results.

Trial design

150 participants in 3 patient groups

Cohort 1: Previously Treated with Triheptanoin

Description:

Patients who have been previously treated with triheptanoin in clinical studies: UX007-CL201 (NCT01886378), UX007-CL202 (NCT02214160), UX007-CL302 (2022-001539-10), Investigator Sponsored Trials (ISTs), or UX007-EAP (NCT03773770).

Treatment:

Other: No Intervention

Cohort 2: Currently or Previously Treated with Triheptanoin

Description:

New patients enrolling into the DMP currently or previously treated with triheptanoin (excluding those in Cohort 1).

Treatment:

Other: No Intervention

Cohort 3: Triheptanoin Naïve

Description:

New patients enrolling into the DMP with no exposure to triheptanoin (naïve).

Treatment:

Other: No Intervention