A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2

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.

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).

Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm^2) of kidney stones is presented.

Percent change from baseline to Day 180 in the number of kidney stones is presented.

Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented.

Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation.

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).

Change from baseline in heart rate is presented.

Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented.

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.

Change from baseline to Day 180 in height is presented.

Change from baseline to Day 180 in weight is presented.

Change from baseline to Day 180 in BMI is presented.

Change from baseline to Day 180 in oral body temperature is presented.

Change from baseline to Day 180 in heart rate is presented.

Change from baseline to Day 180 in respiratory rate is presented.

Change from baseline to Day 180 in systolic and diastolic blood pressure is presented.

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.

Change from baseline to Day 180 in bilirubin, direct bilirubin and creatinine are presented.

Change from baseline to Day 180 in protein and albumin are presented.

Change from baseline to Day 180 in sodium, chloride, potassium and urea are presented.

Change from baseline to Day 180 in vitamin B6 is presented.

Change from baseline to Day 180 in erythrocytes is presented.

Change from baseline to Day 180 in hemoglobin and erythrocytes mean corpuscular hemoglobin concentration are presented.

Change from baseline to Day 180 in hematocrit is presented.

Change from baseline to Day 180 in ery. mean corpuscular volume and mean platelet volume are presented.

Change from baseline to Day 180 in erythrocytes mean corpuscular hemoglobin is presented.

Change from baseline to Day 180 in reticulocytes, platelets, leukocytes, lymphocytes, monocytes, eosinophils, basophils, neutrophils are presented.

Change from baseline to Day 180 in lymphocytes/leukocytes is presented.

Change from baseline to Day 180 in monocytes/leukocytes is presented.

Change from baseline to Day 180 in eosinophils/leukocytes is presented.

Change from baseline to Day 180 in basophils/leukocytes is presented.

Change from baseline to Day 180 in neutrophils/leukocytes is presented.

Change from baseline to Day 180 in urine specific gravity is presented.

Change from baseline to Day 180 in urine pH is presented.

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.

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.