Colon Cancer Clinical Trial

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

Summary

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.

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Full Description

The LC-FAOD DMP is a global observational long-term prospective outcomes study aiming to collect information for up to 10 years from adult and pediatric patients with LC-FAOD, regardless of disease management, including treatment with triheptanoin, and those who have previously participated in triheptanoin clinical trials or 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 only have access to triheptanoin through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

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Eligibility Criteria

Inclusion Criteria:

Confirmed diagnosis of any LC-FAOD sub-type. Diagnosis must be confirmed by results of acylcarnitine profiles, and/or mutation analysis obtained from medical records or equivalent documentation.
Willing and able to comply with all study procedures.
Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures. For minors (<16 or<18 years of age, as defined by region) willing and able (if possible) to provide written assent have a legally authorized representative informed consent after the nature study has been explained, prior any research-related procedures.
Female of child-bearing 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.
Pregnant females carrying affected fetus(es) with confirmed pre-natal diagnosis of LC-FAOD, will also 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.

Study is for people with:

Colon Cancer

Estimated Enrollment:

300

Study ID:

NCT04632953

Recruitment Status:

Recruiting

Sponsor:

Ultragenyx Pharmaceutical Inc

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How clear is this clinincal trial information?

Study is for people with:

Colon Cancer

Estimated Enrollment:

300

Study ID:

NCT04632953

Recruitment Status:

Recruiting

Sponsor:


Ultragenyx Pharmaceutical Inc

How clear is this clinincal trial information?

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