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

Description

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.

Conditions

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

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Study Overview

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Study Details

Study overview

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.

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)

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

Condition
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Intervention / Treatment

-

Contacts and Locations

Phoenix

Phoenix Children's Hospital, Phoenix, Arizona, United States, 85016

San Francisco

University of California San Francisco, San Francisco, California, United States, 94158

Aurora

Children's Hospital of Colorado, Aurora, Colorado, United States, 80045

Tampa

University of South Florida, Tampa, Florida, United States, 33606

Atlanta

The Emory Clinic, Atlanta, Georgia, United States, 30322

Chicago

Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, United States, 60611

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02215

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55454

New York

Columbia University, New York, New York, United States, 10032

Columbus

Nationwide Children's Hospital, Columbus, Ohio, United States, 43205

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ultragenyx Pharmaceutical Inc,

Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceutical Inc

Study Record Dates

2035-12