Ignite Creation Date:
2024-05-05 @ 9:40 PM
Last Modification Date:
2024-10-26 @ 10:08 AM
Study NCT ID:
NCT00947960
Status:
COMPLETED
Last Update Posted:
2018-03-05
First Post:
2009-07-24
Brief Title:
Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
Sponsor:
Baylor Research Institute
Organization:
Baylor Research Institute
Study Overview
Official Title:
A Treatment Trial of Triheptanoin in Patients With Adult Polyglucosan Body Disease - A Randomized Controlled Study
Status:
COMPLETED
Status Verified Date:
2018-03
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 purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease
Detailed Description:
Adult polyglucosan disease is a progressive neurogenetic disorder characterized by neurogenic bladder progressive difficulty with walking and sensory abnormalities in the lower extremities which typically present in the 4th or 5th decade of life The pathogenesis of the disease includes the accumulation of intracellular polyglucosan bodies amylopectin-like polysaccharides in the peripheral nerves as well as the central nervous system cells and is often associated with brancher enzyme deficiency which causes improper glycogen formation It is hypothesized that decreased glycogen degradation leads to energy deficit in the nervous system cells Therefore anaplerotic therapy may supply needed substrate to the citric acid cycle to correct the energy deficit This intervention may slow halt or reverse the progression of the disease for which there is no effective treatment The trial involves 18 subjects ingesting a diet supplemented with triheptanoin a 7 carbon triglyceride or a placebo of vegetable oil at a dose of 1-2 gkg24 hours in a randomized crossover controlled double blind study The study lasts one year with patients receiving triheptanoin for 6 mo and the placebo oil for 6 mo Safety monitoring includes urine organic acids and acyl carnitine profile
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None