A Phase 2 interventional study of nedosiran in Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2, sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company. Completed at 13 sites in 11 countries. Open to participants aged Up to 11 Years. Per ClinicalTrials.gov, last updated 2026-04-16.
Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (part of Novo Nordisk A/S) · Phase 2, Interventional, and Treatment
The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.
This is an open-label, repeat-dose, Phase 2 study of nedosiran in participants 11 years of age or younger who have PH1, PH2 or PH 3 and relatively intact renal function.
Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180.
The total duration of this study is approximately 15 months from first participant, first visit, until last participant, last visit.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's enrollment of 27 is close to 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.
Compared within Primary hyperoxaluria type 1, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Hyperoxaluria, Primary. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Average spot Uox to creatinine ratio at Screening above 2 times the 95th percentile for age (Matos et al, 1999):
Male or Female
Male participants:
A male participant with a female partner of must agree to use , as detailed in Section 10.5.2, during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.
Female participants:
A female participant is eligible to participate if she is not pregnant (see Section 10.5.1), not breastfeeding, and at least 1 of the following conditions applies:
Not a woman of childbearing potential (WOCBP) as defined in Section 10.5.1 OR A WOCBP who agrees to follow the contraceptive guidance in Section 10.5.2 during the treatment period and for at least 12 weeks after the last dose of study intervention.
Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Note: If the childbearing potential changes after start of the study (e.g., a premenarchal female participant experiences menarche) or the risk of pregnancy changes (e.g., a female participant who is not heterosexually active becomes active), the participant must discuss this with the Investigator, who should determine if a female participant must begin a highly effective method of contraception or a male participant must use a condom. If reproductive status is questionable, additional evaluation should be considered.
Participant's parent or legal guardian is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
a. For children younger than 12 years of age, assent will be based on local regulation. If assent is required, participant must be able to provide written assent for participation.
Exclusion Criteria:
Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact participant's safety including, but not restricted to:
History of 1 or more of the following reactions to an oligonucleotide-based therapy:
Participation in any clinical study in which they received an IMP within 4 months or 5 times the half-life of the drug (whichever is longer) before Screening
a. For IMPs with the potential to reduce urine and/or plasma oxalate concentrations, these concentrations must have returned to historical baseline levels prior to Screening
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.
Drug: nedosiran
Monthly subcutaneous dosing throughout study period
Also known as: DCR-PHXC
Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups
This outcome measure reported percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed primary hyperoxaluria type 1 (PH1), primary hyperoxaluria type 2 (PH2), or primary hyperoxaluria type 3 (PH3) subgroups.
Time frame: Baseline (Week 0), Month 6
Absolute Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups
This outcome measure reported absolute change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.
Time frame: Baseline (Week 0), Month 6
Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
This outcome measure reported number of incidents of TEAEs and SAEs. 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 is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE will be defined as treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention.
Time frame: From baseline (Week 0) up to Month 6
Number of Treatment Emergent Adverse Events and Serious Adverse Events-Nature
This outcome measure reported nature of TEAEs and SAEs. An AE is any untoward medical occurrence in clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE is treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention. TEAEs are considered as leading to discontinuation if the action taken is marked as "drug withdrawn" on the case report form. TEAEs of special interest include injection site reactions, muscle pain and weakness, and kidney stone events.
Time frame: From baseline (Week 0) up to Month 6
Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate
This outcome measure reported change from baseline to Month 6 in ECG mean heart rate.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in 12-lead ECG- RR Interval
This outcome measure reported change from baseline to Month 6 in RR Interval.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in 12-lead ECG-PR Interval, Aggregate
This outcome measure reported change from baseline to Month 6 in aggregate PR Interval.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in 12-lead ECG-QRS Duration, Aggregate
This outcome measure reported change from baseline to Month 6 in aggregate QRS Interval.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in 12-lead ECG-QT Interval, Aggregate
This outcome measure reported change from baseline to Month 6 in aggregate QT Interval.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in 12-lead ECG-QTcF Interval, Aggregate
This outcome measure reported change from baseline to Month 6 in aggregate QTcF Interval.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Participants With Significant Findings- Physical Examination
This outcome measure reported change from baseline to Month 6 in number of participants with significant findings (physical examination). Change from baseline was calculated using formula: value at current time point - baseline value.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment- Height
This outcome measure reported change from baseline to Month 6 in participants heights.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Weight
This outcome measure reported change from baseline to Month 6 in participants weights.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Body Mass Index (BMI)
This outcome measure reported change from baseline to Month 6 in participants BMI.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Oral Body Temperature
This outcome measure reported change from baseline to Month 6 in participants oral body temperature.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Respiratory Rate
This outcome measure reported change from baseline to Month 6 in participants respiratory rate.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Heart Rate
This outcome measure reported change from baseline to Month 6 in participants heart rate.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Vital Sign Assessment-Systolic Blood Pressure and Diastolic Blood Pressure
This outcome measure reported change from baseline to Month 6 in participants systolic and diastolic blood pressure.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Erythrocytes
This outcome measure reported change from baseline to Month 6 in participants erythrocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Hemoglobin
This outcome measure reported change from baseline to Month 6 in participants hemoglobin.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Hematocrit
This outcome measure reported change from baseline to Month 6 in participants hematocrit.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Volume
This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular volume.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin
This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular hemoglobin.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin Concentration
This outcome measure reported change from baseline to Month 6 in participant's erythrocytes mean corpuscular hemoglobin concentration.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Reticulocytes
This outcome measure reported change from baseline to Month 6 in participants reticulocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Haematology Assessment: Platelets
This outcome measure reported change from baseline to Month 6 in participants platelets.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Mean Platelet Volume
This outcome measure reported change from baseline to Month 6 in participants mean platelet volume.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Leukocytes
This outcome measure reported change from baseline to Month 6 in participants leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Lymphocytes
This outcome measure reported change from baseline to Month 6 in participants lymphocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Monocytes
This outcome measure reported change from baseline to Month 6 in participants monocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Eosinophils
This outcome measure reported change from baseline to Month 6 in participants eosinophils.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Basophils
This outcome measure reported change from baseline to Month 6 in participants basophils.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Neutrophils
This outcome measure reported change from baseline to Month 6 in participants neutrophils.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Lymphocytes/Leukocytes
This outcome measure reported change from baseline to Month 6 in the ratio of lymphocytes/leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Monocytes/Leukocytes
This outcome measure reported change from baseline to Month 6 in the ratio of monocytes/leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Eosinophils/Leukocytes
This outcome measure reported change from baseline to Month 6 in the ratio of eosinophils/leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Basophils/Leukocytes
This outcome measure reported change from baseline to Month 6 in the ratio of basophils/leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Hematology Assessment: Neutrophils/Leukocytes
This outcome measure reported change from baseline to Month 6 in the ratio of neutrophils/leukocytes.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Alanine Aminotransferase
This outcome measure reported change from baseline to Month 6 in alanine aminotransferase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Aspartate Aminotransferase
This outcome measure reported change from baseline to Month 6 in aspartate aminotransferase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Lactate Dehydrogenase
This outcome measure reported change from baseline to Month 6 in lactate dehydrogenase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Glutamate Dehydrogenase
This outcome measure reported change from baseline to Month 6 in glutamate dehydrogenase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Gamma Glutamyl Transferase
This outcome measure reported change from baseline to Month 6 in gamma glutamyl transferase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Alkaline Phosphatase
This outcome measure reported change from baseline to Month 6 in alkaline phosphatase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Bilirubin and Direct Bilirubin
This outcome measure reported change from baseline to Month 6 in bilirubin and direct bilirubin.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Protein
This outcome measure reported change from baseline to Month 6 in protein.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Albumin
This outcome measure reported change from baseline to Month 6 in albumin.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Creatine Kinase
This outcome measure reported change from baseline to Month 6 in creatine kinase.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Sodium
This outcome measure reported change from baseline to Month 6 in sodium.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Chloride
This outcome measure reported change from baseline to Month 6 in chloride.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Potassium
This outcome measure reported change from baseline to Month 6 in potassium.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Creatinine
This outcome measure reported change from baseline to Month 6 in creatinine.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Blood Urea Nitrogen
This outcome measure reported change from baseline to Month 6 in blood urea nitrogen.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Cystatin C
This outcome measure reported change from baseline to Month 6 in blood urea Cystatin C.
Time frame: Baseline (Week 0), Month 6
Change From Baseline in Clinical Chemistry Parameter: Plasma Oxalate
This outcome measure reported change from baseline to Month 6 in blood urea plasma oxalate.
Time frame: Baseline (Week 0), Month 6
Plasma Pharmacokinetic (PK) Parameter: Maximum Observed Concentration (Cmax), if Estimable
This outcome measure, if estimable, was expected to report Cmax which is defined as maximum observed concentration. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate Cmax, at least one data point prior to Tmax and one data point post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although Cmax was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future.
Time frame: Baseline (Week 0), Month 6
Plasma PK Parameters: Area Under the Concentration-time Curve Calculated to the Last Observable Concentration at Time t (AUCt), if Estimable
This outcome measure, if estimable, was expected to report AUCt which is defined as area under the concentration-time curve calculated from time zero to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUCt, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUCt was not estimated in this study, the PK data collected in this study shall be used in a population PK analysis in the future.
Time frame: Day 1: Postdose 0- to 4-hour and 4- to 24-hour, Days 2, 30, and 90: post dose, Day 150: predose and Day 150: postdose: 0- to 4-hour and 4- to 24-hour
Plasma PK Parameters: Area Under the Concentration-time Curve From Time Zero to Infinity (AUC∞), if Estimable
This outcome measure, if estimable, was expected to report AUC∞ which is defined as area under the concentration-time curve calculated to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUC∞, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUC∞ was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future.
Time frame: Day 1: Postdose 0- to 4-hour and 4- to 24-hour, Days 2, 30, and 90: post dose, Day 150: predose and Day 150: post-dose: 0- to 4-hour and 4- to 24-hour
Percentage of Participants With Spot Urinary Oxalate-to-Creatinine Ratio <=Upper Limit of Normal (ULN) or <=1.5*ULN at Any Time Point Through Month 6 in PH1, PH2, or PH3 Participant Subgroups
This outcome measure reported percentage of participants from baseline to Month 6 Oxalate-to-creatinine Ratio less than and equal to (\<=) ULN or \<=1.5\*ULN at any time point through Month 6 in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.
Time frame: From baseline (week 0) through Month 6
Change From Baseline in eGFR at Month 6 (Only in Participants >=12 Months of Age at Screening) in PH1, PH2, or PH3 Participant Subgroups
This outcome measure reported Change from Baseline in GFR estimated Cystatin C at Month 6 (only in participants \>=12 Months of age at Screening) in PH1, PH2, or PH3 participant subgroups. The eGFR was calculated using multivariate Schwartz equation using formula: eGFR=39.8\*\[ht/Scr\]\^0.456 \[1.8/cysC\]\^0.418 \[30/BUN\]\^0.0791.076\^male\[ht/1.4\]\^0.179 where ht (height) = meters, Scr (serum creatinine) = milligrams per deciliter (mg/dL), cysC (cystatin C) = mg/L, and BUN (blood urea nitrogen) = mg/dL.
Time frame: Baseline (Week 0), Month 6
The study was conducted at 13 sites in 9 countries.
| Milestone | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Started | 5 | 13 | 9 |
| Modified intent-to-treat (mitt) | 5 | 13 | 9 |
| Safety | 5 | 13 | 9 |
| Completed | 5 | 13 | 9 |
| Not completed | 0 | 0 | 0 |
This outcome measure reported percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed primary hyperoxaluria type 1 (PH1), primary hyperoxaluria type 2 (PH2), or primary hyperoxaluria type 3 (PH3) subgroups.
| Percent change | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| PH1 | -74.06 ± 13.193 | -68.34 ± 8.574 | -61.44 ± 24.565 |
| PH2 | — | -17.98 ± 37.847 | -16.10 ± 0 |
| PH3 | -41.43 ± 13.633 | — | — |
This outcome measure reported absolute change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.
| micromole/millimole | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| PH1 | -563.972 ± 378.3308 | -316.344 ± 150.6332 | -189.106 ± 120.5939 |
| PH2 | — | -64.458 ± 92.5196 | -27.583 ± 0 |
| PH3 | -135.283 ± 17.1591 | — | — |
This outcome measure reported number of incidents of TEAEs and SAEs. 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 is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE will be defined as treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention.
| Events | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| TEAEs | 37 | 66 | 38 |
| SAEs | 1 | 4 | 2 |
This outcome measure reported nature of TEAEs and SAEs. An AE is any untoward medical occurrence in clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, results in persistent disability/incapacity, is a congenital anomaly/birth defect, and medical events. An AE is treatment emergent if they have an onset or worsen in severity after a participant receives the study intervention. TEAEs are considered as leading to discontinuation if the action taken is marked as "drug withdrawn" on the case report form. TEAEs of special interest include injection site reactions, muscle pain and weakness, and kidney stone events.
| Events | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Treatment Related TEAEs | 2 | 7 | 4 |
| TEAEs Leading to Treatment Interruption | 1 | 0 | 0 |
| TEAEs Leading to Treatment Discontinuation | 0 | 0 | 0 |
| Serious Treatment Related TEAEs | 0 | 0 | 0 |
| Fatal TEAEs | 0 | 0 | 0 |
| TEAEs of Special Interest | 7 | 8 | 8 |
This outcome measure reported change from baseline to Month 6 in ECG mean heart rate.
| beats/minute | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate | -11.6 ± 32.34 | -4.2 ± 15.73 | 6.2 ± 16.98 |
This outcome measure reported change from baseline to Month 6 in RR Interval.
| seconds | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead ECG- RR Interval | 0.1 ± 0.16 | 0.0 ± 0.10 | -0.1 ± 0.12 |
This outcome measure reported change from baseline to Month 6 in aggregate PR Interval.
| milliseconds | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead ECG-PR Interval, Aggregate | 3.6 ± 8.56 | 1.8 ± 6.39 | -1.0 ± 8.75 |
This outcome measure reported change from baseline to Month 6 in aggregate QRS Interval.
| milliseconds | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead ECG-QRS Duration, Aggregate | 3.2 ± 5.45 | 0.5 ± 6.44 | 1.7 ± 3.24 |
This outcome measure reported change from baseline to Month 6 in aggregate QT Interval.
| milliseconds | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead ECG-QT Interval, Aggregate | 28.0 ± 30.60 | 8.2 ± 20.46 | -6.7 ± 23.69 |
This outcome measure reported change from baseline to Month 6 in aggregate QTcF Interval.
| milliseconds | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in 12-lead ECG-QTcF Interval, Aggregate | 21.0 ± 17.68 | 4.5 ± 14.88 | 1.8 ± 13.29 |
This outcome measure reported change from baseline to Month 6 in number of participants with significant findings (physical examination). Change from baseline was calculated using formula: value at current time point - baseline value.
| Participants | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Participants With Significant Findings- Physical Examination | 0 | 0 | 1 |
This outcome measure reported change from baseline to Month 6 in participants heights.
| centimeters | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment- Height | 6.24 ± 2.220 | 4.01 ± 1.237 | 2.76 ± 1.548 |
This outcome measure reported change from baseline to Month 6 in participants weights.
| kilograms (kg) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment-Weight | 1.130 ± 0.3033 | 1.488 ± 1.2636 | 2.411 ± 1.4575 |
This outcome measure reported change from baseline to Month 6 in participants BMI.
| Kilograms per meter square (kg/m^2) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment-Body Mass Index (BMI) | -0.668 ± 0.8318 | 0.152 ± 0.9265 | 0.608 ± 0.9316 |
This outcome measure reported change from baseline to Month 6 in participants oral body temperature.
| degree celcius (˚C) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment-Oral Body Temperature | 0.36 ± 0.555 | -0.01 ± 0.441 | -0.00 ± 0.339 |
This outcome measure reported change from baseline to Month 6 in participants respiratory rate.
| breaths/minute | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment-Respiratory Rate | -1.4 ± 6.77 | -1.8 ± 4.17 | 0.8 ± 2.44 |
This outcome measure reported change from baseline to Month 6 in participants heart rate.
| beats/minute | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Vital Sign Assessment-Heart Rate | 6.2 ± 14.13 | -1.5 ± 12.52 | 4.7 ± 16.96 |
This outcome measure reported change from baseline to Month 6 in participants systolic and diastolic blood pressure.
| Millimeters of Mercury (mmHg) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Systolic Blood Pressure | 6.8 ± 22.20 | -6.8 ± 17.21 | 2.8 ± 7.95 |
| Diastolic Blood Pressure | 7.6 ± 17.84 | 1.1 ± 13.62 | 2.8 ± 4.27 |
This outcome measure reported change from baseline to Month 6 in participants erythrocytes.
| Tera cells per liter (10^12 cells /L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Erythrocytes | 0.195 ± 0.3375 | 0.173 ± 0.2765 | 0.055 ± 0.2076 |
This outcome measure reported change from baseline to Month 6 in participants hemoglobin.
| Grams per deciliter (g/dL) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Hemoglobin | 0.575 ± 0.7632 | 0.155 ± 0.8092 | 0.250 ± 0.7051 |
This outcome measure reported change from baseline to Month 6 in participants hematocrit.
| Ratio of Hematocrit | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Hematocrit | 0.01 ± 0.025 | 0.01 ± 0.019 | 0.00 ± 0.023 |
This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular volume.
| Femtoliter (fL) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Volume | 1.70 ± 3.818 | -1.25 ± 2.393 | -0.05 ± 2.530 |
This outcome measure reported change from baseline to Month 6 in participants erythrocytes mean corpuscular hemoglobin.
| picograms | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin | 0.25 ± 0.624 | -0.48 ± 1.244 | 0.18 ± 0.738 |
This outcome measure reported change from baseline to Month 6 in participant's erythrocytes mean corpuscular hemoglobin concentration.
| g/dL | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Erythrocytes Mean Corpuscular Hemoglobin Concentration | -0.42 ± 1.754 | -0.09 ± 1.691 | 0.19 ± 0.783 |
This outcome measure reported change from baseline to Month 6 in participants reticulocytes.
| Percentage of Reticulocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Reticulocytes | 0.15 ± 0.208 | 0.14 ± 0.781 | -0.08 ± 0.443 |
This outcome measure reported change from baseline to Month 6 in participants platelets.
| Giga cells per liter (10^9 cells /L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Haematology Assessment: Platelets | -1.8 ± 40.87 | -18.6 ± 62.25 | -27.7 ± 102.53 |
This outcome measure reported change from baseline to Month 6 in participants mean platelet volume.
| fL | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Mean Platelet Volume | 0.05 ± 0.507 | -0.10 ± 0.610 | 0.03 ± 0.553 |
This outcome measure reported change from baseline to Month 6 in participants leukocytes.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Leukocytes | -1.460 ± 2.1985 | 0.383 ± 2.3556 | -0.625 ± 2.2732 |
This outcome measure reported change from baseline to Month 6 in participants lymphocytes.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Lymphocytes | -1.808 ± 2.3547 | 0.429 ± 1.1896 | -0.577 ± 0.9109 |
This outcome measure reported change from baseline to Month 6 in participants monocytes.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Monocytes | -0.013 ± 0.2295 | -0.024 ± 0.1306 | -0.038 ± 0.1288 |
This outcome measure reported change from baseline to Month 6 in participants eosinophils.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Eosinophils | 0.078 ± 0.1441 | 0.029 ± 0.1495 | 0.106 ± 0.3064 |
This outcome measure reported change from baseline to Month 6 in participants basophils.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Basophils | 0.035 ± 0.0545 | -0.008 ± 0.0606 | -0.014 ± 0.0320 |
This outcome measure reported change from baseline to Month 6 in participants neutrophils.
| 10^9 cells /L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Neutrophils | -0.165 ± 2.1021 | -0.073 ± 1.4642 | -0.106 ± 1.3720 |
This outcome measure reported change from baseline to Month 6 in the ratio of lymphocytes/leukocytes.
| Ratio of Lymphocytes/Leukocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Lymphocytes/Leukocytes | -10.25 ± 16.801 | 2.20 ± 9.901 | -2.51 ± 13.862 |
This outcome measure reported change from baseline to Month 6 in the ratio of monocytes/leukocytes.
| Ratio of Monocytes/Leukocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Monocytes/Leukocytes | 0.70 ± 2.534 | -0.45 ± 1.109 | -0.14 ± 1.255 |
This outcome measure reported change from baseline to Month 6 in the ratio of eosinophils/leukocytes.
| Ratio of Eosinophils/Leukocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Eosinophils/Leukocytes | 1.33 ± 1.531 | 0.22 ± 1.829 | 1.29 ± 3.061 |
This outcome measure reported change from baseline to Month 6 in the ratio of basophils/leukocytes.
| Ratio of Basophils/Leukocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Basophils/Leukocytes | 0.55 ± 0.777 | -0.01 ± 0.517 | -0.09 ± 0.606 |
This outcome measure reported change from baseline to Month 6 in the ratio of neutrophils/leukocytes.
| Ratio of Neutrophils/Leukocytes | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Hematology Assessment: Neutrophils/Leukocytes | 7.63 ± 14.717 | -2.31 ± 9.655 | 1.50 ± 12.398 |
This outcome measure reported change from baseline to Month 6 in alanine aminotransferase.
| Units per liter (U/L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Alanine Aminotransferase | -1.8 ± 4.44 | 14.9 ± 24.68 | 5.1 ± 4.61 |
This outcome measure reported change from baseline to Month 6 in aspartate aminotransferase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Aspartate Aminotransferase | -4.0 ± 11.94 | 6.4 ± 13.83 | 7.1 ± 6.94 |
This outcome measure reported change from baseline to Month 6 in lactate dehydrogenase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Lactate Dehydrogenase | -29.3 ± 17.29 | 19.5 ± 67.20 | 1.6 ± 23.44 |
This outcome measure reported change from baseline to Month 6 in glutamate dehydrogenase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Glutamate Dehydrogenase | 0.00 ± 0.000 | 0.28 ± 0.601 | 0.06 ± 0.316 |
This outcome measure reported change from baseline to Month 6 in gamma glutamyl transferase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Gamma Glutamyl Transferase | -0.4 ± 4.45 | 1.4 ± 1.89 | 1.6 ± 0.92 |
This outcome measure reported change from baseline to Month 6 in alkaline phosphatase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Alkaline Phosphatase | -6.6 ± 19.65 | 14.6 ± 37.61 | -8.4 ± 14.88 |
This outcome measure reported change from baseline to Month 6 in bilirubin and direct bilirubin.
| micromole per liter (umol/L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Bilirubin | -0.46 ± 1.389 | 1.62 ± 3.467 | 1.23 ± 2.355 |
| Direct Bilirubin | -0.07 ± 0.096 | 0.20 ± 1.224 | -0.03 ± 0.588 |
This outcome measure reported change from baseline to Month 6 in protein.
| Grams per liter (g/L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Protein | -0.8 ± 6.76 | 0.6 ± 3.71 | -2.0 ± 5.55 |
This outcome measure reported change from baseline to Month 6 in albumin.
| g/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Albumin | -0.2 ± 2.17 | 1.2 ± 2.79 | -0.2 ± 2.64 |
This outcome measure reported change from baseline to Month 6 in creatine kinase.
| U/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Creatine Kinase | -45.6 ± 58.24 | 9.5 ± 37.12 | 5.0 ± 20.34 |
This outcome measure reported change from baseline to Month 6 in sodium.
| millimole per liter (mmol/L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Sodium | -0.4 ± 2.51 | -0.6 ± 2.79 | -0.2 ± 2.91 |
This outcome measure reported change from baseline to Month 6 in chloride.
| mmol/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Chloride | -1.0 ± 1.22 | 0.4 ± 2.93 | -0.9 ± 2.64 |
This outcome measure reported change from baseline to Month 6 in potassium.
| mmol/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Potassium | -0.42 ± 0.356 | -0.11 ± 0.571 | -0.15 ± 0.411 |
This outcome measure reported change from baseline to Month 6 in creatinine.
| umol/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Creatinine | 0.66 ± 5.062 | 1.10 ± 6.327 | 1.26 ± 1.965 |
This outcome measure reported change from baseline to Month 6 in blood urea nitrogen.
| mmol/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Blood Urea Nitrogen | 0.56 ± 2.016 | 0.06 ± 1.999 | -0.11 ± 1.082 |
This outcome measure reported change from baseline to Month 6 in blood urea Cystatin C.
| milligrams per liter (mg/L) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Change From Baseline in Clinical Chemistry Parameter: Cystatin C | -0.120 ± 0.1595 | -0.026 ± 0.1962 | 0.024 ± 0.1283 |
This outcome measure reported change from baseline to Month 6 in blood urea plasma oxalate.
| umol/L | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| PH1 | -9.000 ± 10.5830 | -5.667 ± 2.6583 | -4.400 ± 4.3932 |
| PH2 | — | -2.500 ± 0.7071 | — |
| PH3 | -2.000 ± 1.4142 | — | — |
This outcome measure, if estimable, was expected to report Cmax which is defined as maximum observed concentration. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate Cmax, at least one data point prior to Tmax and one data point post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although Cmax was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future.
| nanograms per milliliter (ng/mL) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Plasma Pharmacokinetic (PK) Parameter: Maximum Observed Concentration (Cmax), if Estimable | NA ± NA | NA ± NA | NA ± NA |
This outcome measure, if estimable, was expected to report AUCt which is defined as area under the concentration-time curve calculated from time zero to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUCt, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUCt was not estimated in this study, the PK data collected in this study shall be used in a population PK analysis in the future.
| hour*nanograms per milliliter | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Plasma PK Parameters: Area Under the Concentration-time Curve Calculated to the Last Observable Concentration at Time t (AUCt), if Estimable | NA ± NA | NA ± NA | NA ± NA |
This outcome measure, if estimable, was expected to report AUC∞ which is defined as area under the concentration-time curve calculated to the last observable concentration at time t. However, in this study, a non-compartmental PK analysis was not planned or executed due to the limited PK samples (2 PK concentration - time data points between 0 to 4 hours and 4 to 24 hours following dose administration on Day 1) collected in pediatric participants per protocol, hence this PK parameter was not estimated. To estimate AUC∞, at least one data point prior to Tmax and three data points post-Tmax at the designated time points are needed; however, only two flexible post-dose PK concentration data points were available in this study. Although AUC∞ was not estimated in this study, the PK data collected in this study will be used in a population PK analysis in the future.
| hour*nanograms per milliliter | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| Plasma PK Parameters: Area Under the Concentration-time Curve From Time Zero to Infinity (AUC∞), if Estimable | NA ± NA | NA ± NA | NA ± NA |
This outcome measure reported percentage of participants from baseline to Month 6 Oxalate-to-creatinine Ratio less than and equal to (\<=) ULN or \<=1.5\*ULN at any time point through Month 6 in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.
| Percentage of participants | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| PH1: <= 1.0 * ULN | 66.7 | 44.4 | 62.5 |
| PH1: <= 1.5 * ULN | 100 | 88.9 | 100 |
| PH2: <= 1.0 * ULN | — | 50.0 | 0 |
| PH2: <= 1.5 * ULN | — | 75.0 | 100 |
| PH3: <= 1.0 * ULN | 50.0 | — | — |
| PH3: <= 1.5 * ULN | 100 | — | — |
This outcome measure reported Change from Baseline in GFR estimated Cystatin C at Month 6 (only in participants \>=12 Months of age at Screening) in PH1, PH2, or PH3 participant subgroups. The eGFR was calculated using multivariate Schwartz equation using formula: eGFR=39.8\*\[ht/Scr\]\^0.456 \[1.8/cysC\]\^0.418 \[30/BUN\]\^0.0791.076\^male\[ht/1.4\]\^0.179 where ht (height) = meters, Scr (serum creatinine) = milligrams per deciliter (mg/dL), cysC (cystatin C) = mg/L, and BUN (blood urea nitrogen) = mg/dL.
| milliliters/minute/1.73m^2 | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years |
|---|---|---|---|
| PH1 | 0.0 ± 17.06 | 0.3 ± 10.2 | -0.8 ± 7.21 |
| PH2 | — | 3.5 ± 13.48 | -7.0 ± 0 |
| PH3 | 15.0 ± 9.90 | — | — |
Collected over Baseline (Week 0) to Month 6. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Children 0 to <2 Years | 0/5 (0%) | 1/5 (20%) | 5/5 (100%) |
| Children 2 to <6 Years | 0/13 (0%) | 3/13 (23.1%) | 11/13 (84.6%) |
| Children 6 to 11 Years | 0/9 (0%) | 1/9 (11.1%) | 7/9 (77.8%) |
| Overall | 0/27 (0%) | 5/27 (18.5%) | 23/27 (85.2%) |
| Event | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Overall |
|---|---|---|---|---|
| Upper respiratory tract infectionInfections and infestations | 1/5 | 0/13 | 0/9 | 1/27 |
| GastroenteritisInfections and infestations | 0/5 | 0/13 | 1/9 | 1/27 |
| NephrolithiasisRenal and urinary disorders | 0/5 | 1/13 | 1/9 | 2/27 |
| PneumoniaInfections and infestations | 0/5 | 1/13 | 0/9 | 1/27 |
| PyelocaliectasisRenal and urinary disorders | 0/5 | 1/13 | 0/9 | 1/27 |
| Event | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Overall |
|---|---|---|---|---|
| PyrexiaGeneral disorders | 4/5 | 0/13 | 2/9 | 6/27 |
| InfluenzaInfections and infestations | 2/5 | 1/13 | 0/9 | 3/27 |
| NasopharyngitisInfections and infestations | 2/5 | 2/13 | 0/9 | 4/27 |
| NephrolithiasisRenal and urinary disorders | 2/5 | 0/13 | 1/9 | 3/27 |
| VomitingGastrointestinal disorders | 2/5 | 0/13 | 0/9 | 2/27 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/5 | 4/13 | 0/9 | 4/27 |
| Upper respiratory tract infectionInfections and infestations | 0/5 | 3/13 | 0/9 | 3/27 |
| Calculus urinaryRenal and urinary disorders | 0/5 | 1/13 | 2/9 | 3/27 |
| DysuriaRenal and urinary disorders | 0/5 | 0/13 | 2/9 | 2/27 |
| Injection site painGeneral disorders | 1/5 | 2/13 | 2/9 | 5/27 |
Safety population included all participants who received at least 1 dose of study intervention.
| Age, Continuous(years) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Total |
|---|---|---|---|---|
| Mean | 1.30 ± 0.529 | 3.54 ± 1.198 | 7.78 ± 1.093 | 4.54 ± 2.687 |
| Sex: Female, Male(Participants) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Total |
|---|---|---|---|---|
| Female | 1 | 6 | 4 | 11 |
| Male | 4 | 7 | 5 | 16 |
| Ethnicity (NIH/OMB)(Participants) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Total |
|---|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 1 | 2 |
| Not Hispanic or Latino | 4 | 12 | 8 | 24 |
| Unknown or Not Reported | 0 | 1 | 0 | 1 |
| Race/Ethnicity, Customized(Participants) | Children 0 to <2 Years | Children 2 to <6 Years | Children 6 to 11 Years | Total |
|---|---|---|---|---|
| Asian | 1 | 5 | 0 | 6 |
| White | 3 | 2 | 4 | 9 |
| Multiple | 1 | 5 | 5 | 11 |
| Unknown | 0 | 1 | 0 | 1 |
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Studies of the drug tested here: Nedosiran
Primary hyperoxaluria type 1 →
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company →