Ignite Creation Date:
2024-05-06 @ 8:21 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06340685
Status:
RECRUITING
Last Update Posted:
2024-06-05
First Post:
2024-03-22
Brief Title:
Triheptanoin for Children with Primary-Specific Pyruvate Dehydrogenase Complex PDC Deficiency
Sponsor:
Jirair Krikor Bedoyan
Organization:
University of Pittsburgh
Study Overview
Official Title:
An Open Label Exploratory Proof-of Concept Study of Triheptanoin As Treatment for Patients with Primary-Specific Pyruvate Dehydrogenase Complex PDC Deficiency
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex PDC Deficiency The medication is triheptanoin which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders Previous research suggests that triheptanoin may also be effective in the treatment PDC Deficiency This study will investigate the safety and efficacy how well it works of triheptanoin in patients with PDC Deficiency
Detailed Description:
Participation in the study will require the patient to participate in up to 10 visits over a two-year period Five of those visits must be done at the UPMC Childrens Hospital of Pittsburgh CHP Other visits can take place at CHP or remotely All of these visits will include blood draws
Triheptanoin will be added to the patients diet and administered at least 4 times per day The target dose will be 12-39 g of triheptanoin per kg body weight with a max goal dose of about 4 gkg per day
The triheptanoin will be provided to the patients at no cost All other costs will be billed to the patients insurance
Triheptanoin will be added to the patients diet and administered at least 4 times per day The target dose will be 12-39 g of triheptanoin per kg body weight with a max goal dose of about 4 gkg per day
The triheptanoin will be provided to the patients at no cost All other costs will be billed to the patients insurance
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UX007-IST236 | OTHER_GRANT | Ultragenyx Pharmaceutical Inc | None |