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Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

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ClinicalTrials.gov Identifier: NCT03768817
Recruitment Status : Active, not recruiting
First Posted : December 7, 2018
Last Update Posted : August 5, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.

Condition or disease Intervention/treatment
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Other: No Intervention

Detailed Description:
The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted.

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Study Type : Observational
Estimated Enrollment : 22 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
Actual Study Start Date : January 28, 2019
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : November 2019

Group/Cohort Intervention/treatment
Patients treated with triheptanoin
Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND
Other: No Intervention
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.




Primary Outcome Measures :
  1. Duration of Hospitalization for Trigger Event [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  2. Disposition on Discharge of Trigger Event [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  3. Duration of Important Interventions for Trigger Event [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  4. Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]

Secondary Outcome Measures :
  1. Duration of Hospitalization for Other Major Clinical Events [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  2. Disposition on Discharge of Other Major Clinical Events [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  3. Number of Important Medical Interventions for Other Major Clinical Events [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]
  4. Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events [ Time Frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment ]


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018.
Criteria

Inclusion Criteria:

  • Male or female patients of any age with confirmed diagnosis of one of the LC-FAOD disorders including: mitochondrial trifunctional protein (TFP) deficiency, carnitine palmitoyltransferase deficiencies (CPT I and CPT II), very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD) deficiency, and carnitine acylcarnitine translocase (CATR) deficiency
  • Treatment initiation with triheptanoin was via eIND before 01 September 2018
  • Treated with triheptanoin in the USA
  • Willing and able to provide written, signed informed consent, or where appropriate for subjects under the age of 18, or provide written assent and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. To obtain and review medical records of deceased individuals, informed consent from next of kin or appropriate legal entity will be obtained, as applicable.

Exclusion Criteria:

  • Unwilling to sign informed consent or assent to release of medical records

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03768817


Locations
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United States, California
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Kaiser Permanente
Los Angeles, California, United States, 90027
Children's Hospital of Orange County
Orange, California, United States, 92868
Stanford University
Palo Alto, California, United States, 94304
United States, District of Columbia
Children's National Hospital
Washington, District of Columbia, United States, 20010
United States, Florida
University of South Florida
Tampa, Florida, United States, 33606
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55455
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27710
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc

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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03768817     History of Changes
Other Study ID Numbers: UX007-CL003
First Posted: December 7, 2018    Key Record Dates
Last Update Posted: August 5, 2019
Last Verified: August 2019

Keywords provided by Ultragenyx Pharmaceutical Inc:
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Triheptanoin

Additional relevant MeSH terms:
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Disease
Pathologic Processes