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 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.
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 not affected by LC-FAOD carrying a fetus affected by LC-FAOD with confirmed pre-natal diagnosis of LC-FAOD.

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:

200

Study ID:

NCT04632953

Recruitment Status:

Recruiting

Sponsor:

Ultragenyx Pharmaceutical Inc

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There are 16 Locations for this study

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Phoenix Children's Hospital
Phoenix Arizona, 85016, United States
University of California San Francisco
San Francisco California, 94158, United States
Children's Hospital of Colorado
Aurora Colorado, 80045, United States
University of South Florida
Tampa Florida, 33606, United States
The Emory Clinic
Atlanta Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital
Chicago Illinois, 60611, United States
Boston Children's Hospital
Boston Massachusetts, 02215, United States
University of Minnesota
Minneapolis Minnesota, 55454, United States
Columbia University
New York New York, 10032, United States
Nationwide Children's Hospital
Columbus Ohio, 43205, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh Pennsylvania, 15224, United States
University of Utah
Salt Lake City Utah, 84108, United States
Seattle Children's Hospital
Seattle Washington, 98020, United States
University of Alberta
Edmonton Alberta, T6G 1, Canada
CHEO (Children's Hospital Eastern Ontario)
Ottawa Ontario, K1H 8, Canada
SickKids (The Hospital for Sick Children)
Toronto Ontario, M5G 1, Canada

How clear is this clinincal trial information?

Study is for people with:

Colon Cancer

Estimated Enrollment:

200

Study ID:

NCT04632953

Recruitment Status:

Recruiting

Sponsor:


Ultragenyx Pharmaceutical Inc

How clear is this clinincal trial information?

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