CompletedNot Applicable

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

United States20 participantsStarted 2019-01-30

Plain-language 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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Duration of Hospitalization for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Disposition on Discharge of Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Duration of Important Interventions for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Trial details

NCT IDNCT03768817

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2020-06-08

View on ClinicalTrials.gov More Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) trials

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Duration of Hospitalization for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Disposition on Discharge of Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Duration of Important Interventions for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event

Timeframe: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment