Ignite Creation Date:
2024-05-06 @ 3:26 PM
Last Modification Date:
2024-10-26 @ 1:49 PM
Study NCT ID:
NCT04632953
Status:
RECRUITING
Last Update Posted:
2024-06-25
First Post:
2020-11-09
Brief Title:
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Ultragenyx Pharmaceutical Inc
Study Overview
Official Title:
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program LC-FAOD DMP
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed Description:
The LC-FAOD Disease Monitoring Program DMP is an international long-term retrospective and prospective outcomes study aiming to collect safety and effectiveness data for triheptanoin and the natural history of LC-FAOD for a study duration of up to 10 years from adult and pediatric patients with LC-FAOD with any previous disease management regardless of prior treatment with triheptanoin those who have previously participated in triheptanoin clinical trials and those who received triheptanoin through an 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 have access to triheptanoin only through authorized commercial use if approved in their country or available EAP but not from the LC-FAOD DMP itself
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 have access to triheptanoin only through authorized commercial use if approved in their country or available EAP but not from the LC-FAOD DMP itself
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None