A Phase 3 interventional study of DCR-PHXC in Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2) and Kidney Diseases, sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company. Active, not recruiting at 23 sites in 13 countries. Per ClinicalTrials.gov, last updated 2026-10-08.
Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (part of Novo Nordisk A/S) · Phase 3, Interventional, and Treatment
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (\<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's planned enrollment of 75 is above the median of 28 across 16 interventional studies indexed under Primary hyperoxaluria type 1.
Browse Primary hyperoxaluria type 1 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:
•Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.
OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.
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.
Open label, monthly subcutaneous injection
Drug: DCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Also known as: Nedosiran
The annual rate of decline in eGFR in participants with PH1
To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
Time frame: Annual change from baseline
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings
To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via change from baseline and abnormal ECG findings. Standard 12-lead ECGs will be performed in the supine position after the subject has rested comfortably for 10 minutes. The parameters assessed will be rhythm, ventricular rate, PR interval, QRS duration, QT interval, and corrected QT interval (QTcF, Fridericia correction). The Investigator or designee is responsible for reviewing the ECG(s) to assess whether the results are within normal limits and to determine the clinical significance of the results. Standardized ECG acquisition equipment will be provided to all clinical trial sites at the start of the trial, to ensure parity across all sites.
Time frame: TEAEs and SAEs are evaluated monthly for 6 years
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal physical examination findings
To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via change from baseline and incidence of abnormal physical exam findings. A full physical examination will include a complete review of body systems: eyes, ears, nose, and throat, chest/respiratory, heart/cardiovascular, gastrointestinal/liver, musculoskeletal/extremities, dermatological/skin, thyroid/neck, lymph nodes, and neurological. A full physical exam is done at Screening, Day 180 and if a participant ends the study early. A brief physical examination will minimally include chest/respiratory, heart/cardiovascular, dermatological/skin, and gastrointestinal/liver. A brief physical examination will be performed at the Investigator's discretion at all other visits.
Time frame: TEAEs and SAEs are evaluated monthly for 6 years
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal vital signs
To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via the change from baseline and incidence of abnormal vital signs. Vital signs include blood pressure, pulse/heart rate, oral body temperature, and respiratory rate. Parameters will be measured in the supine position, using an automated instrument or manually, after the participant has rested comfortably for 10 minutes. In the pediatric population, an age-appropriate cuff size should be used for blood pressure measurements. Temperature will be obtained in degrees Celsius (°C), pulse rate will be counted for a full minute and recorded in beats per minute, and respirations will be counted for a full minute and recorded in breaths per minute.
Time frame: TEAEs and SAEs are evaluated monthly for 6 years
The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs related to abnormal clinical laboratory tests (hematology, chemistry, coagulation parameters, and urinalysis)
To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via the change from baseline and incidence of abnormal clinical laboratory tests.
Time frame: TEAEs and SAEs are evaluated monthly for 6 years
To identify the proportion of participants with normalized or near-normalized 24 hour urinary oxalate (Uox)
The proportion of participants with a 24 hour Uox level (\< 0.46 mmol/24 hours or ≥ 0.46 - \< 0.60 mmol/24 hours \[adjusted per 1.73 m2 body surface area (BSA) in participants aged \< 18 years\]) at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups
Time frame: 24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
To identify the percentage of participants with spot urinary oxalate-to-creatinine ratio ≤ the ULN or ≤ 1.5 x ULN
The percentage of participants with spot urinary oxalate-to-creatinine ratio ≤ the ULN or ≤ 1.5 x ULN at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups
Time frame: Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
To assess the effect of DCR-PHXC on stone events in patients with PH
Change from Baseline in the number of stone events over a 12-month period, annually in Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups
Time frame: Evaluated yearly for 6 years
To assess the effect of DCR-PHXC on stone burden grade in patients with PH
Change from Baseline in the stone burden grade at Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups
Time frame: Evaluated yearly for 6 years
To assess the effect of DCR-PHXC in nephrocalcinosis grade in patients with PH
Change from Baseline in nephrocalcinosis grade at Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups
Time frame: Evaluated yearly for 6 years
To evaluate the incidence of chronic kidney disease (CKD) and end-stage renal disease (ESRD) in participants with PH
The number of participants with severe CKD (GFR = 15-29 mL/min) or ESRD (GFR \<15 mL/min); adjusted per 1.73 m2 BSA in participants aged \< 18 years in PH1, PH2, and PH3 participant subgroups
Time frame: eGFR is evaluated monthly for 6 months (or quarterly for multidose rollovers), quarterly for 2 1/2 years, and every 6 months for 3 years after that.
Change from Baseline in the Short Form (36) Health Survey (SF-36®) in PH1, PH2, and PH3 participant subgroups
To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The SF 36 is a set of generic, coherent, and easily administered quality-of-life measures that taps 8 health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. It also includes a single item that provides an indication of perceived change in health. The 36 items are identical to the MOS SF 36 described in Ware and Sherbourne (1992). Participants respond to each item on a categorical scale. Categorical answers are transformed to a 0 to 100 range so that the lowest and highest possible scores are 0 and 100, respectively. All items are scored so that a high score defines a more favorable health state.
Time frame: Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
Change from Baseline in the EQ-5D-5L™ in adults in PH1, PH2, and PH3 participant subgroups
To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The EQ-5D-5L consists of the EQ 5D descriptive system and the EQ visual analogue scale (EQ VAS). The descriptive system has 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The digits for the 5 dimensions can be combined into a 5-digit number that describes the participant's health state. The EQ VAS records the participant's self-rated health on a 20-cm vertical VAS, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine.' Participants are asked to place an "X" on the line that represents their health on that day.
Time frame: Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children in PH1, PH2, and PH3 participant subgroups
To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The 23-item PedsQL is comprised of 5 items in the Emotional, Social, and School Functioning dimensions (Psychosocial Health) and 8 items in the Physical Functioning (Physical Health) dimension. Items are reverse-scored on a 0 to 4 Likert scale and linearly transformed to a 0 to 100 scale, so that higher scores indicate better functioning and HRQOL. Scale Scores are the sum of the items in each dimension, divided by the number of items answered.
Time frame: Surveys are administered at screening, Day 180, yearly for 3.5 years, then at Month 72 (EOS).
To assess the efficacy of DCR PHXC in reducing Uox burden in patients with PH: TWS AUC
Time-weighted standardized area under the curve (TWS AUC) of 24-hour Uox from Day 90 to Day 180, based on percent change from Baseline in PH1, PH2, and PH3 participant subgroups. This endpoint will only be assessed in participants previously randomized to placebo in a previous study of DCR- PHXC and pediatric siblings.
Time frame: Monthly for 4 months (D90 through D180)
To assess the long-term efficacy of DCR PHXC in reducing Uox burden in patients with PH
Percent change from Baseline in 24-hour Uox at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups. In those participants randomized to placebo in a previous study of DCR-PHXC and pediatric siblings, this endpoint will be assessed only after Month 6
Time frame: 24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
To assess the long-term efficacy of DCR-PHXC in reducing Uox burden in patients with PH
Percent and absolute change from Baseline in spot urinary oxalate-to-creatinine ratio at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups. In pediatric siblings, this endpoint will be assessed only after Month 6
Time frame: Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that.
To evaluate the effect of DCR PHXC on eGFR in participants with PH2 and PH3
The annual rate of decline in eGFR in participants with PH2 and PH3
Time frame: Annual change from baseline
To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin).
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including minimum observed concentration (Cmin)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including time to maximum concentration (Tmax)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing terminal elimination half-life (t1/2).
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including terminal elimination half-life (t1/2)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing clearance.
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including clearance (CL)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing volume of distribution of estimates.
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including volume of distribution (V) estimates
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing the area under the curve (AUC)
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including secondary parameters of area under the curve (AUC)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax).
Population and/or individual pharmacokinetic (PK) parameters for DCR PHXC, including maximum observed concentration (Cmax)
Time frame: Participants rolling from a single dose study will be analyzed at Day 1, Day 2, Day 30, Day 31, Day 150, and Day 180; multidose rollovers will just collect Day 1 and 180. Then there will be analyses every 6 months for 2.5 years, and annually for 3 years.
Plan to share: No
Started Jul 2019 (actual). Primary completion is estimated for Apr 2030, 128 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nedosiran
Primary hyperoxaluria type 1 →
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company →