A Phase 1 interventional study of DCR-PHXC and Sterile Normal Saline (0.9% NaCl) in Primary Hyperoxaluria Type 3, sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company. Completed at 6 sites in 4 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (part of Novo Nordisk A/S) · Phase 1, Interventional, and Treatment
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.
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 (2:1 randomization; 4 nedosiran:2 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.
42 studies on the registry are indexed under Hyperoxaluria, Primary; 11 are open to participants now.
This study's enrollment of 6 is below the median of 24 across 26 interventional studies indexed under Hyperoxaluria, Primary.
Browse Hyperoxaluria, Primary studies →Dicerna Pharmaceuticals, Inc., a Novo Nordisk company is the lead sponsor of 18 studies on the registry; 2 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 4 (50%) have results posted.
First submitted before Dicerna Pharmaceuticals, Inc. became part of Novo Nordisk A/S.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
History of at least one stone event within the last 12 months. Stone events are defined as any of the following:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants that are at least 12 years old will receive a single dose of 3 mg/kg DCR-PHXC (or nedosiran) via subcutaneous (SC) injection. Participants that are 6-11 years old will receive a single dose of 3.5 mg/kg DCR-PHXC (nedosiran) via SC injection.
Drug: DCR-PHXC
Participants will receive a single dose of Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo.
Drug: Sterile Normal Saline (0.9% NaCl)
Intervention, drug, DCR-PHXC
Also known as: Nedosiran
Placebo comparator
Safety profile of a single dose of DCR-PHXC in PH3 Patients
Number of patients with abnormalities in clinically significant laboratory results, vital signs, and 12-lead ECG findings
Time frame: Screening through Day 85
Plasma pharmacokinetics (PK) of a single dose of DCR-PHXC in PH3 patients
Measure maximum plasma concentration of DCR-PHXC
Time frame: Day 1 (dosing) through Day 29
The proportion of participants achieving a > 30% decrease from baseline in 24-hour Urine Oxalate (Uox) on 2 consecutive visits
Participants must maintain at least a 30% decrease from the average of 2 screening 24-hour Uox values to be considered "responders" to treatment. The proportion of responders to non-responders will be utilize to assess the efficacy of a single dose of DCR-PHXC in PH3 patients.
Time frame: After screening, 24-hour Uox will be measured at Days 29, 43, 57, and 85.
This study is completed, as verified in Sep 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nedosiran
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company →