Clinical Trial Details
— Status: Active, not recruiting
Administrative data
NCT number |
NCT04542590 |
Other study ID # |
DCR-PHXC-502 |
Secondary ID |
|
Status |
Active, not recruiting |
Phase |
|
First received |
|
Last updated |
|
Start date |
September 9, 2021 |
Est. completion date |
August 5, 2024 |
Study information
Verified date |
March 2024 |
Source |
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Observational [Patient Registry]
|
Clinical Trial 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 and/or the presence of pre existing stones detected by renal
ultrasound at screening.
Description:
The relationship between the level of Uox and the incidence of kidney stones and/or
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 and/or 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.