ACTIVE_NOT_RECRUITING

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

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Conditions

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)CACT DeficiencyCarnitine Acylcarnitine Translocase DeficiencyCPT1CPT2Carnitine Palmitoyltransferase DeficienciesVLCADVery Long Chain Acyl Coa Dehydrogenase DeficiencyLCHAD DeficiencyLong-chain 3-hydroxyacyl-CoA Dehydrogenase DeficiencyTFP DeficiencyTrifunctional Protein Deficiency

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-11-30
Study Completion:2035-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04632953

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * 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.
  2. * Willing and able to comply with all study procedures.
  3. * Willing and able to provide consent or, if a minor, provide assent and informed consent by their legally authorized representative.
  4. * 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.
  1. * Presence of a concurrent disease or condition that would interfere with study participation or affect patient's safety in the opinion of the Investigator.
  2. * 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.

Contacts and Locations

Principal Investigator

Medical Director
STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc

Study Locations (Sites)

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

Collaborators and Investigators

Sponsor: Ultragenyx Pharmaceutical Inc

  • Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceutical Inc

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-11-30
Study Completion Date2035-12

Study Record Updates

Study Start Date2021-11-30
Study Completion Date2035-12

Terms related to this study

Keywords Provided by Researchers

  • CACT Deficiency
  • Carnitine Acylcarnitine Translocase Deficiency
  • CPT1
  • CPT2
  • Carnitine Palmitoyltransferase Deficiencies
  • VLCAD
  • Very Long Chain Acyl Coa Dehydrogenase Deficiency
  • LCHAD Deficiency
  • Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
  • TFP Deficiency
  • Trifunctional Protein Deficiency

Additional Relevant MeSH Terms

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