Ignite Creation Date:
2024-05-06 @ 3:12 PM
Last Modification Date:
2024-10-26 @ 1:45 PM
Study NCT ID:
NCT04555486
Status:
COMPLETED
Last Update Posted:
2024-07-11
First Post:
2020-09-02
Brief Title:
Study to Evaluate Safety Tolerability PK and PD of DCR-PHXC in PH Type 3 Patients
Sponsor:
Dicerna Pharmaceuticals Inc a Novo Nordisk company
Organization:
Novo Nordisk AS
Study Overview
Official Title:
A Phase 1 Placebo-Controlled Double-Blind Multicenter Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of a Single Dose of DCR-PHXC in Patients With Primary Hyperoxaluria Type 3
Status:
COMPLETED
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:
PHYOX4
Brief Summary:
The DCR-PHXC-104 study is designed to assess the safety tolerability and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 PH3 Participants should have had at least one stone event within 12 months of screening and intact renal function
Detailed Description:
Potential participants are screened over an up-to-35-day period with an extra 7-day period for participants who are required to repeat screening 24-hour urine collections or initially unanalyzable screening laboratory assessment samples prior to randomization Eligible participants will receive a single dose of DCR-PHXC or placebo on Day 1
In order to maintain the treatment blind 24-hour urine oxalate Uox results that could unblind the study will not be reported to investigative sites or other blinded personnel until the study has been unblinded
It is expected that approximately 10 participants will be screened in order to randomize 6 participants 21 randomization 4 nedosiran2 placebo to the study
Following the up-to-6-week screening period participants will return to the clinic for interim visits up to Day 85 Visits occurring between the Day 1 and the Day 85 visit may be conducted as at-home telemedicine visits at the discretion of the Investigator The total time on study for each participant is approximately 18 weeks
In order to maintain the treatment blind 24-hour urine oxalate Uox results that could unblind the study will not be reported to investigative sites or other blinded personnel until the study has been unblinded
It is expected that approximately 10 participants will be screened in order to randomize 6 participants 21 randomization 4 nedosiran2 placebo to the study
Following the up-to-6-week screening period participants will return to the clinic for interim visits up to Day 85 Visits occurring between the Day 1 and the Day 85 visit may be conducted as at-home telemedicine visits at the discretion of the Investigator The total time on study for each participant is approximately 18 weeks
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