Primary Hyperoxaluria Type 1 (PH1)Phase 2PHYOX2NCT03847909
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
A Phase 2 study testing DCR-PHXC and Sterile Normal Saline (0.9% NaCl) in people with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2), run by Novo Nordisk A/S at 28 sites in 15 countries.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2) Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases
- What is being tested?
- DCR-PHXC and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 28 sitesin 15 countries
- Study dates
- 28 Oct 2019to 29 Jun 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Capable and willing to provide written informed consent or assent
- Documented diagnosis of PH1 or PH2, confirmed by genotyping
- Must meet the 24 hour urine oxalate excretion requirements
- Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period
- Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA
Who cannot join
- Renal or hepatic transplantation (prior or planned within the study period)
- Currently on dialysis or anticipated requirement for dialysis during the study period
- Plasma oxalate >30 µmol/L
- Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
- Use of an RNA interference (RNAi) drug within the last 6 months
- Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening
- Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN) for age and gender
- Inability or unwillingness to comply with study procedures
Trial locations and contacts
28 sites in 15 countries.
28 sites in 15 countries
United States5 sites
Clinical Trial Site
Boston, Massachusetts, 02115
Clinical Trial Site
New York, New York, 10016
Clinical Trial Site
Pittsburgh, Pennsylvania, 15224
Clinical Trial Site
Rochester, Minnesota, 55905
Clinical Trial Site
San Francisco, California, 94143
Japan3 sites
Clinical Trial Site
Nagoya, 467-8601
Clinical Trial Site
Tochigi, 329-0431
Clinical Trial Site
Tokyo, 183-8561
United Kingdom3 sites
Clinical Trial Site
Birmingham
Clinical Trial Site
London, WCIN 3JH
Clinical Trial Site
London
Australia2 sites
Clinical Trial Site
Herston, 4029
Clinical Trial Site
Parkville, 3052
Germany2 sites
Clinical Trial Site
Bonn, 53127
Clinical Trial Site
Heidelberg, 69120
Spain2 sites
Clinical Trial Site
Barcelona, 08035
Clinical Trial Site
Santa Cruz, 38320
About this study
Official title: A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
- Being tested
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Keywords
- PH1, PH2, Primary Hyperoxaluria, RNAi, RNAi therapeutic, GalNAc, LDHA gene, LDH, siRNA
- Medical subject headings
- Primary hyperoxaluria type 1, Primary hyperoxaluria type 2, Kidney Diseases, Urologic Diseases, Genetic Diseases, Inborn, Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DCR-PHXCDrug
Also called nedosiran
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)Drug
Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo
| Study group | Role | What they receive |
|---|---|---|
| DCR-PHXC | Experimental | Intervention, drug, DCR-PHXC Receives: Drug: DCR-PHXC |
| Placebo - Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
Measured: From Day 90 to 180
Secondary outcomes (36)
Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
Percentage of participants whose 24-hour Uox values normalized or near-normalized on at least 2 consecutive visits are presented. Normalization of Uox was defined as less than (<) 0.46 millimole per 24 hours (mmol/24 hours) and near normalization was defined as greater than or equal to (>=) 0.46 to < 0.60 mmol/24 hours (values adjusted per 1.73 square meter [1.73 m^2] body surface area [BSA] in participants aged <18 years).
Measured: From Day 90 to 180
Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm^2) of kidney stones is presented.
Measured: Baseline, Day 180
Percent Change From Baseline to Day 180 in the Number of Kidney Stones
Percent change from baseline to Day 180 in the number of kidney stones is presented.
Measured: Baseline, Day 180
Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented.
Measured: Baseline, Day 180
Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation.
Measured: Baseline, Day 180
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
Number of TEAEs and TESAEs are presented. An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE was defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalisation, results in persistent disability/incapacity or is a congenital anomaly/birth defect. TEAE was defined as any AE with an onset date/time on or after administration (including any partial administration) of the first dose of study intervention and through the study completion date from the end of study case report form (CRF).
Measured: From Baseline up to Day 180
Change From Baseline in Electrocardiogram (ECG): Heart Rate
Change from baseline in heart rate is presented.
Measured: Baseline, Day 180
Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented.
Measured: Baseline, Day 180
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Number of participants who had most abnormal post-baseline shift in physical examination are presented. Physical examination shifts were categories into 4 categories: 1) missing; 2) normal; 3) abnormal-not clinically significant (NCS) and 4) abnormal-clinically significant (CS). Each category was presented according body systems including: 1) eyes, ears, nose and throat; 2) chest/respiratory; 3) heart/cardiovascular; 4) gastrointestinal/liver; 5) musculoskeletal/extremities; 6) dermatological/skin; 7) thyroid/neck; 8) lymph nodes; 9) neurological.
Measured: Baseline up to Day 180
Change From Baseline in Vital Signs: Height
Change from baseline to Day 180 in height is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Weight
Change from baseline to Day 180 in weight is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Body Mass Index (BMI)
Change from baseline to Day 180 in BMI is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Oral Body Temperature
Change from baseline to Day 180 in oral body temperature is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Heart Rate
Change from baseline to Day 180 in heart rate is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Respiratory Rate
Change from baseline to Day 180 in respiratory rate is presented.
Measured: Baseline, Day 180
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Change from baseline to Day 180 in systolic and diastolic blood pressure is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase
Change from baseline to Day 180 in alanine aminotransferase, aspartate aminotransferase, glutamate dehydrogenase, gamma glutamyl transferase, alkaline phosphatase, lactate dehydrogenase and creatine kinase are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine
Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Change from baseline to Day 180 in protein and albumin are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
Change from baseline to Day 180 in vitamin B6 is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
Change from baseline to Day 180 in erythrocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration
Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
Change from baseline to Day 180 in hematocrit is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume
Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils
Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
Change from baseline to Day 180 in lymphocytes/leukocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
Change from baseline to Day 180 in monocytes/leukocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
Change from baseline to Day 180 in eosinophils/leukocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
Change from baseline to Day 180 in basophils/leukocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
Change from baseline to Day 180 in neutrophils/leukocytes is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
Change from baseline to Day 180 in urine specific gravity is presented.
Measured: Baseline, Day 180
Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
Change from baseline to Day 180 in urine pH is presented.
Measured: Baseline, Day 180
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
The Cmax was defined as the maximum observed plasma concentration during a dosing interval. Data for this endpoint is reported only for adults and adolescent participants from PK population.
Measured: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC
AUC0-last was defined as the area under the curve from time of administration to the last measurable concentration. Data for this endpoint is reported only for adults and adolescent participants from PK population.
Measured: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2019
- Study start28 Oct 2019
- Primary completion21 Jun 2021
- Study completion29 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Primary Hyperoxaluria Type 1 (PH1)Studies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency (dept. 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03847909
- Sponsor's protocol ID
- DCR-PHXC-201
- Acronym
- PHYOX2
- Status last verified
- May 2024
- First submitted
- 15 Feb 2019
- Last updated
- 22 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zhang S, Gamallo P, Rawson V. Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. Clin Pharmacokinet. 2025 Sep;64(9):1395-1411. doi: 10.1007/s40262-025-01540-1. Epub 2025 Jul 2.
PubMed 40601241Cox JH, Boily MO, Caron A, Sheng T, Wu J, Ding J, Gaudreault S, Chong O, Surendradoss J, Gomez R, Lester J, Dumais V, Li X, Gumpena R, Hall MD, Waterson AG, Stott G, Flint AJ, Moore WJ, Lowther WT, Knight J, Percival MD, Tong V, Oballa R, Powell DA, King AJ. Characterization of CHK-336, A First-in-Class, Liver-Targeted, Small-Molecule Lactate Dehydrogenase Inhibitor for Hyperoxaluria Treatment. J Am Soc Nephrol. 2025 Apr 7;36(8):1535-1547. doi: 10.1681/ASN.0000000690.
PubMed 40193200Baum MA, Langman C, Cochat P, Lieske JC, Moochhala SH, Hamamoto S, Satoh H, Mourani C, Ariceta G, Torres A, Wolley M, Belostotsky V, Forbes TA, Groothoff J, Hayes W, Tonshoff B, Takayama T, Rosskamp R, Russell K, Zhou J, Amrite A, Hoppe B; PHYOX2 study investigators. PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2. Kidney Int. 2023 Jan;103(1):207-217. doi: 10.1016/j.kint.2022.07.025. Epub 2022 Aug 22.
PubMed 36007597
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
- ClinicalTrials.gov ID
- NCT03847909
- Acronym / sponsor ID
- PHYOX2 / DCR-PHXC-201
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
- Keywords
- PH1, PH2, Primary Hyperoxaluria, RNAi, RNAi therapeutic, GalNAc, LDHA gene, LDH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2019 (actual)
- Primary completion
- 21 Jun 2021 (actual)
- Study completion
- 29 Jun 2021 (actual)
- First posted
- 20 Feb 2019 (actual)
- Results
- Posted 22 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| DCR-PHXC | Experimental | Drug: DCR-PHXCIntervention, drug, DCR-PHXC |
| Placebo - Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl)Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Capable and willing to provide written informed consent or assent
- Documented diagnosis of PH1 or PH2, confirmed by genotyping
- Must meet the 24 hour urine oxalate excretion requirements
- Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period
- Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA
Key exclusion criteria (8 listed)
- Renal or hepatic transplantation (prior or planned within the study period)
- Currently on dialysis or anticipated requirement for dialysis during the study period
- Plasma oxalate >30 µmol/L
- Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
- Use of an RNA interference (RNAi) drug within the last 6 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DCR-PHXCAlso: nedosiranMultiple fixed doses of DCR-PHXC by subcutaneous (SC) injection | Drug | DCR-PHXC |
| Sterile Normal Saline (0.9% NaCl)Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo | Drug | Placebo - Sterile Normal Saline (0.9% NaCl) |
Primary outcome
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox
Time frame: From Day 90 to 180
The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC.
36 secondary outcomes
Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits
Time frame: From Day 90 to 180
Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones
Time frame: Baseline, Day 180
Percent Change From Baseline to Day 180 in the Number of Kidney Stones
Time frame: Baseline, Day 180
Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only)
Time frame: Baseline, Day 180
Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180
Time frame: Baseline, Day 180
Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs)
Time frame: From Baseline up to Day 180
Change From Baseline in Electrocardiogram (ECG): Heart Rate
Time frame: Baseline, Day 180
Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval
Time frame: Baseline, Day 180
Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination
Time frame: Baseline up to Day 180
Change From Baseline in Vital Signs: Height
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Weight
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Body Mass Index (BMI)
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Oral Body Temperature
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Heart Rate
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Respiratory Rate
Time frame: Baseline, Day 180
Change From Baseline in Vital Signs: Systolic and Diastolic Blood Pressure
Time frame: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Alanine Aminotransferase, Aspartate Aminotransferase, Glutamate Dehydrogenase, Gamma Glutamyl Transferase, Alkaline Phosphatase, Lactate Dehydrogenase and Creatine Kinase
Time frame: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Bilirubin, Direct Bilirubin and Creatinine
Time frame: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Protein, Albumin
Time frame: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Sodium, Chloride, Potassium and Urea
Time frame: Baseline, Day 180
Change From Baseline in Clinical Chemistry Laboratory Tests: Vitamin B6
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Hemoglobin and Erythrocytes Mean Corpuscular Hemoglobin Concentration
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Hematocrit
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes (Ery.) Mean Corpuscular Volume and Mean Platelet Volume
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Erythrocytes Mean Corpuscular Hemoglobin
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Reticulocytes, Platelets, Leukocytes, Lymphocytes, Monocytes, Eosinophils, Basophils, Neutrophils
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Lymphocytes/Leukocytes
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Monocytes/Leukocytes
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Eosinophils/Leukocyte
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Basophils/Leukocytes
Time frame: Baseline, Day 180
Change From Baseline in Clinical Hematology Laboratory Tests: Neutrophils/Leukocytes
Time frame: Baseline, Day 180
Change From Baseline in Clinical Urinalysis Laboratory Tests: Specific Gravity
Time frame: Baseline, Day 180
Change From Baseline in Clinical Urinalysis Laboratory Tests: pH
Time frame: Baseline, Day 180
Maximum Observed Plasma Concentration (Cmax) of DCR-PHXC
Time frame: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours (hrs) postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Area Under the Curve From Time of Administration to the Last Measurable Concentration (AUC0-last) of of DCR-PHXC
Time frame: For adults: Day 1 and 30: predose, 5, 15, and 30 minutes and 1, 2, 4, 6, 10, and 12 hours postdose; Day 150: predose, 2, 6, and 12 hours postdose For adolescents: Days 1 and 30: predose, 30 minutes and 2 and 10 hours postdose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40601241Zhang S, Gamallo P, Rawson V. Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. Clin Pharmacokinet. 2025 Sep;64(9):1395-1411. doi: 10.1007/s40262-025-01540-1. Epub 2025 Jul 2.
- Derived from this study: PubMed 40193200Cox JH, Boily MO, Caron A, Sheng T, Wu J, Ding J, Gaudreault S, Chong O, Surendradoss J, Gomez R, Lester J, Dumais V, Li X, Gumpena R, Hall MD, Waterson AG, Stott G, Flint AJ, Moore WJ, Lowther WT, Knight J, Percival MD, Tong V, Oballa R, Powell DA, King AJ. Characterization of CHK-336, A First-in-Class, Liver-Targeted, Small-Molecule Lactate Dehydrogenase Inhibitor for Hyperoxaluria Treatment. J Am Soc Nephrol. 2025 Apr 7;36(8):1535-1547. doi: 10.1681/ASN.0000000690.
- Derived from this study: PubMed 36007597Baum MA, Langman C, Cochat P, Lieske JC, Moochhala SH, Hamamoto S, Satoh H, Mourani C, Ariceta G, Torres A, Wolley M, Belostotsky V, Forbes TA, Groothoff J, Hayes W, Tonshoff B, Takayama T, Rosskamp R, Russell K, Zhou J, Amrite A, Hoppe B; PHYOX2 study investigators. PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2. Kidney Int. 2023 Jan;103(1):207-217. doi: 10.1016/j.kint.2022.07.025. Epub 2022 Aug 22.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency (dept. 1452) study director
Novo Nordisk A/S
28 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing DCR-PHXC and Sterile Normal Saline (0.9% NaCl) in people with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2), run by Novo Nordisk A/S at 28 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Japan, United Kingdom, Australia, France and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox (time frame: From Day 90 to 180). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03847909, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2024.