Ignite Creation Date:
2024-05-06 @ 4:04 PM
Last Modification Date:
2024-10-26 @ 2:03 PM
Study NCT ID:
NCT04868578
Status:
RECRUITING
Last Update Posted:
2024-05-17
First Post:
2021-03-30
Brief Title:
PPI Supplementation to Fight ECtopIc Calcification in PXE
Sponsor:
Centre Hospitalier Universitaire de Nice
Organization:
Centre Hospitalier Universitaire de Nice
Study Overview
Official Title:
Efficacy of PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum - PROPHECI-PPI Study
Status:
RECRUITING
Status Verified Date:
2024-05
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:
PROPHECI-PPI
Brief Summary:
Pseudoxanthoma elasticum PXE is a rare inherited metabolic disorder OMIM 264800 frequency 125000 characterized by progressive ectopic calcification of connective tissues PXE mainly affects the skin inesthetic papules and plaques in the skin folds the retina central blindness the vasculature peripheral arterial occlusive disease and stroke and the renal system renal lithiasis in adulthood Although rarely early lethal forms have been reported This chronic and highly disabling condition results from a loss of function of the gene encoding for the ABCC6 membrane transporter primarily expressed in the hepatocytes and renal tubular cells Recently it has been reported that PXE was characterized by a 50-60 decrease in the plasma level of inorganic pyrophosphate PPi a major physiological anti-calcifying factor PXE is an incurable disease which therapeutic options are limited to symptomatic treatments to stem the devastating effect of the ectopic calcifications Recently encouraging proof of concept studies with animals PXE models and healthy volunteers have shown that contrary to what was initially reported and thought the oral administration of PPi salts are able to increase PPi plasma levels opening up new therapeutic perspectives in PXE Therefore we propose to perform the first Phase II randomized controlled trial RCT to evaluate the safety and efficacy of a daily and oral administration of PPi salts against placebo in PXE patients
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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