Ignite Creation Date:
2024-05-06 @ 3:10 PM
Last Modification Date:
2024-10-26 @ 1:44 PM
Study NCT ID:
NCT04542590
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-07-11
First Post:
2020-08-20
Brief Title:
Natural History of Patients With PH3 and a History of Stone Events
Sponsor:
Dicerna Pharmaceuticals Inc a Novo Nordisk company
Organization:
Novo Nordisk AS
Study Overview
Official Title:
A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and as Applicable Per Age a History of Stone Events
Status:
ACTIVE_NOT_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:
PHYOX-OBX
Brief Summary:
This is a natural history study of adults adolescents and children starting at birth with genetically confirmed primary hyperoxaluria type 3 PH3 who have a history of stone events during the last 3 years andor the presence of pre existing stones detected by renal ultrasound at screening
Detailed Description:
The relationship between the level of Uox and the incidence of kidney stones andor nephrocalcinosis in patients with PH3 has not been established The goal of this study is to record 24-hour Uox levels and the incidence of new stone formation andor the degree of nephrocalcinosis in patients with PH3 over time
Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate number of new stones being formed per year and the change in the degree of nephrocalcinosis if applicable
New stone formation is defined as occurrence of any of the following
Spontaneous stone passage in the absence of pre-existing stones
Stone passage occurring without change in the number of pre-existing stones detected by renal ultrasound
Appearance of new stones on renal ultrasound or 100 or more growth of a pre-existing stone estimated area
Surgical removal of newly formed stones Note Spontaneous passage or surgical removal of pre-existing stones does not meet the criteria for new stone formation
This is a non-interventional study that will last up to 2 years
Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate number of new stones being formed per year and the change in the degree of nephrocalcinosis if applicable
New stone formation is defined as occurrence of any of the following
Spontaneous stone passage in the absence of pre-existing stones
Stone passage occurring without change in the number of pre-existing stones detected by renal ultrasound
Appearance of new stones on renal ultrasound or 100 or more growth of a pre-existing stone estimated area
Surgical removal of newly formed stones Note Spontaneous passage or surgical removal of pre-existing stones does not meet the criteria for new stone formation
This is a non-interventional study that will last up to 2 years
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