Ignite Creation Date:
2024-05-06 @ 7:36 PM
Last Modification Date:
2024-10-26 @ 3:10 PM
Study NCT ID:
NCT06067802
Status:
RECRUITING
Last Update Posted:
2024-05-03
First Post:
2023-08-10
Brief Title:
Study of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency MCADD
Sponsor:
Jerry Vockley MD PhD
Organization:
University of Pittsburgh
Study Overview
Official Title:
A Phase II Escalating Dose Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency MCADD
Status:
RECRUITING
Status Verified Date:
2024-09
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:
This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase deficiency MCADD 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 MCADD This study will investigate the safety and efficacy how well it works of triheptanoin in patients with MCADD
Detailed Description:
Participation in the study will require three overnight admissions at the Clinical and Translational Research Center at the UPMC Childrens Hospital of Pittsburgh also called the PCTRC The total length of the study is 10 weeks
Subjects will have blood work and an intravenous access line IV placed for several blood draws during the visit Subjects will begin fasting during the admission which means they may consume only non-caloric fluids water unsweetened black coffee or tea or sugar-free beverages Bloodwork will be collected during the fast Following the completion of the fast the subject will eat a meal and will receive the study drug triheptanoin The total time of fasting will be up to 24 hours
Dosing for this study will begin at 02 gmkgday up to a dose of 10 gmkgday The dose will be increased gradually to avoid gastric upset The dose should be divided into 3 or 4 daily doses and given with food or liquid The dose can be decreased if a subject experiences any gastric upset that indicates that they cannot tolerate the higher dose
Subjects will return two more times at Weeks 5 and 9 to undergo the overnight admission and 24-hour fasting procedures outlined above After the Week 9 admission they will no longer take the triheptanoin Study staff will contact them by phone one week later Week 10 to make sure they are not experiencing any adverse effects
All study procedures will be done at no cost to the subjects
Subjects will have blood work and an intravenous access line IV placed for several blood draws during the visit Subjects will begin fasting during the admission which means they may consume only non-caloric fluids water unsweetened black coffee or tea or sugar-free beverages Bloodwork will be collected during the fast Following the completion of the fast the subject will eat a meal and will receive the study drug triheptanoin The total time of fasting will be up to 24 hours
Dosing for this study will begin at 02 gmkgday up to a dose of 10 gmkgday The dose will be increased gradually to avoid gastric upset The dose should be divided into 3 or 4 daily doses and given with food or liquid The dose can be decreased if a subject experiences any gastric upset that indicates that they cannot tolerate the higher dose
Subjects will return two more times at Weeks 5 and 9 to undergo the overnight admission and 24-hour fasting procedures outlined above After the Week 9 admission they will no longer take the triheptanoin Study staff will contact them by phone one week later Week 10 to make sure they are not experiencing any adverse effects
All study procedures will be done at no cost to the subjects
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-IST237 | OTHER_GRANT | Ultragenyx Pharmaceutical Inc | None |