A Study to Evaluate Lumasiran in Children and Adults With... | NCT03681184

ID	Type	Description	Link
2018-001981-40	EudraCT Number

Label	Type	Description	Intervention Names
Placebo/Lumasiran	Placebo Comparator	Lumasiran-matching placebo (normal saline [0.9% NaCl]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period.	Drug: Placebo Drug: Lumasiran
Lumasiran/Lumasiran	Experimental	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.	Drug: Placebo Drug: Lumasiran

Name	Type	Description	Arm Group Labels	Other Names
Placebo	Drug	Placebo by SC injection	Lumasiran/Lumasiran Placebo/Lumasiran

Measure	Description	Time Frame
Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6	Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.	Baseline to Month 6

Measure	Description	Time Frame
Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6	Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

Measure	Description	Time Frame
Rate of Renal Stone Events	A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone. Lower rates indicate a favorable outcome.	12-Month Period prior to Informed Consent, 6-Month DB Period

Name	Affiliation	Role
Medical Director	Alnylam Pharmaceuticals	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Trial Site		San Diego	California	92120	United States
Clinical Trial Site

PubMed Identifier	Type	Citation	Retractions
41343248	Derived	Frishberg Y, Saland JM, Lieske JC, Du W, Coenen M, Hogan J, Sellier-Leclerc AL, Groothoff JW, Kaspar C, Gansner JM, Hulton SA; ILLUMINATE-A study investigators. Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1. Clin J Am Soc Nephrol. 2026 Mar 1;21(3):377-388. doi: 10.2215/CJN.0000000916. Epub 2025 Dec 4.
33789010

ID	Title	Description
FG000	Placebo/Lumasiran	Lumasiran-matching placebo (normal saline [0.9% NaCl]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	May 6, 2020	Jul 12, 2024

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6

Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

FAS: All randomized participants who received any amount of study drug.

Posted

Least Squares Mean

Standard Error

percent change

Baseline to Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Units	Counts
Participants	OG00013 OG00126

Title

Denominators

Categories

Title	Measurements
	OG000-11.8± 3.8 OG001-65.4± 2.9

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001	The Mixed-Effect Model Repeated Measures (MMRM) includes fixed effects of treatment arms (lumasiran vs. placebo) and scheduled visits (months 3, 4, 5, and 6), baseline 24-hour urinary oxalate corrected for BSA as a continuous fixed covariate, and patient as a random effect. The variance-covariance matrix is assumed to be unstructured. Satterthwaite approximation is used to estimate denominator degrees of freedom.	MMRM		<0.0001	P=1.685E-14	Difference in Least Squares (LS) Mean	-53.546	Standard Error of the Mean	4.3224	2-Sided	95	-62.314	-44.778					Superiority

Secondary

Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6

Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline across Months 3 through 6. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

FAS: All randomized participants who received any amount of study drug.

Posted

Least Squares Mean

Standard Error

mmol/24hr/1.73m^2

Baseline to Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

Percent change in 24-hour urinary oxalate:creatine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.

FAS: All randomized participants who received any amount of study drug.

Posted

Least Squares Mean

Standard Error

percent change

Baseline to Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6

The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available.

Posted

Number

percentage of participants

Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Units	Counts

Secondary

Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6

The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available.

Posted

Number

percentage of participants

Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Units	Counts

Secondary

Percentage Change in Plasma Oxalate From Baseline to Month 6

Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.

Plasma Oxalate Analysis Set: all participants who received any amount of study drug and had baseline plasma oxalate level ≥1.5×lower limit of quantitation (LLOQ). LLOQ is 5.55 μmol/L.

Posted

Least Squares Mean

Standard Error

percent change

Baseline to Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Absolute Change in Plasma Oxalate From Baseline to Month 6

Absolute change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.

Plasma Oxalate Analysis Set: all participants who received any amount of study drug and had baseline plasma oxalate level ≥1.5×LLOQ. LLOQ is 5.55 μmol/L.

Posted

Least Squares Mean

Standard Error

μmol/L

Baseline to Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6

eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients >1 year to <18 years of age at screening. Change from baseline to Month 6 is reported.

Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available.

Posted

Mean

Standard Deviation

mL/min/1.73m^2

Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period

Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during the 6-month double-blinded period and participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period. Overall number analyzed is number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint.

Posted

Mean

Standard Deviation

mmol/24hr/1.73m^2

From Baseline to Month 54 and Month 60

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period

Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.

Posted

Mean

Standard Deviation

percent change

From Baseline to Month 54 and Month 60

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During Lumasiran Treatment

The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2 for 24-hour urinary oxalate excretion corrected for BSA.

Posted

Median

Full Range

percentage of time

Up to Month 60

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Absolute Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline in the Extension Period

Absolute change in 24-hour urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline to the end of the OLE period at Month 54 and Month 60. A negative change from Baseline indicates a favorable outcome.

Posted

Mean

Standard Deviation

mmol/mmol

From Baseline to Month 54 and Month 60

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline in the Extension Period

All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during the 6-month double-blinded period and participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period. Overall number analyzed is number of participants with data available for analysis. Number analysed is the number of participants with data available for analysis at the specified timepoint.

Posted

Mean

Standard Deviation

mL/min/1.73m^2

From Baseline to Month 54 and Month 60

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Secondary

Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE is any untoward medical occurrence in a clinical investigational participant administered a medicinal product & which does not necessarily have a causal relationship with this treatment. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability or incapacity, is a congenital anomaly or birth defect, is an important medical event that may not be immediately life-threatening/result in death/hospitalization but may jeopardize the participant & may require intervention to prevent one of the other outcomes listed above. All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during 6-month double-blinded period & participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period.

Per the SAP, long-term safety of lumasiran (in the extension period [EP]) was to be summarized by treatment sequence (placebo/lumasiran & lumasiran/lumasiran) using the All Lumasiran Treated Set. In this set, data is presented during lumasiran treatment only. The lumasiran/lumasiran arm received lumasiran in both DB & EP period hence, safety data is reported together for DB+EP period for this arm. Placebo/Lumasiran: first day of lumasiran dosing (Month 6) was considered baseline (Day 1) of EP.

Posted

Count of Participants

Participants

DB Period (Placebo): From first dose of study drug (Day 1) up to Month 6; Placebo/Lumasiran: From first dose of lumasiran (Month 6) up to end of study (Month 60); Lumasiran/Lumasiran: From first dose of lumasiran (Day 1) up to end of study (Month 60).

ID	Title	Description

Other Pre-specified

Rate of Renal Stone Events

A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone. Lower rates indicate a favorable outcome.

FAS: All randomized participants who received any amount of study drug.

Posted

Number

95% Confidence Interval

events per person-year

12-Month Period prior to Informed Consent, 6-Month DB Period

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Other Pre-specified

Change From Baseline in Nephrocalcinosis as Assessed by Renal Ultrasound

Renal ultrasound data were used to grade medullary nephrocalcinosis findings (range: 0 to 3), where a higher grade indicates greater severity. Improving=if both sides improve, or one side improves and the other side has no change; No change=if both sides have no change; Worsening=if both sides worsen, or one side worsens and the other side has no change; Indeterminate=if one side improves and the other side worsens.

Participants from the FAS (all randomized participants who received any amount of study drug) with both Baseline and Month 6 renal ultrasounds.

Posted

Count of Participants

Participants

Baseline, Month 6

ID	Title	Description
OG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
OG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal discomfort	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0028 affected26 at risk
Injection site erythema	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Injection site reaction	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0015 affected13 at risk EG0029 affected26 at risk
Otitis media acute	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Pneumonia	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Rhinitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0002 affected13 at risk EG0013 affected13 at risk EG0022 affected26 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0002 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0020 affected26 at risk
Gastrostomy tube site complication	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0020 affected26 at risk
Iron deficiency anaemia	Blood and lymphatic system disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Thrombocytopenia	Blood and lymphatic system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Arrhythmia supraventricular	Cardiac disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Thalassaemia beta	Congenital, familial and genetic disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Ear pain	Ear and labyrinth disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Blepharitis	Eye disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Dacryostenosis acquired	Eye disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Eye pain	Eye disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Vision blurred	Eye disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Abdominal pain lower	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0021 affected26 at risk
Abdominal tenderness	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Constipation	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0021 affected26 at risk
Gastrooesophageal reflux disease	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Gingival hypertrophy	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Haemorrhoidal haemorrhage	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Nausea	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0022 affected26 at risk
Vomiting	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0022 affected26 at risk
Asthenia	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Catheter site extravasation	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Chest pain	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Fatigue	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Influenza like illness	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Oedema peripheral	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Peripheral swelling	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Pyrexia	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0023 affected26 at risk
Vaccination site pain	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0023 affected26 at risk
Vaccination site swelling	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hepatomegaly	Hepatobiliary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hypersensitivity	Immune system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Immunisation reaction	Immune system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0022 affected26 at risk
Milk allergy	Immune system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Asymptomatic COVID-19	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Bronchitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
COVID-19	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0014 affected13 at risk EG0024 affected26 at risk
Cellulitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Ear infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Fungal foot infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Fungal skin infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Furuncle	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Gastroenteritis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0021 affected26 at risk
Gastroenteritis viral	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hand-foot-and-mouth disease	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Herpes simplex reactivation	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Herpes zoster	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Infected bite	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Influenza	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Nasopharyngitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0024 affected26 at risk
Onychomycosis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Pharyngitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Pyelitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Pyelonephritis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Pyelonephritis acute	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Tonsillitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Tooth infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Urinary tract infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0023 affected26 at risk
Viral infection	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Viral sinusitis	Infections and infestations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Corneal abrasion	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Foot fracture	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hand fracture	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Ligament sprain	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Nail injury	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Post-traumatic pain	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0022 affected26 at risk
Skin abrasion	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Skin laceration	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Sunburn	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Tibia fracture	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Traumatic haematoma	Injury, poisoning and procedural complications	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Aspartate aminotransferase increased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Blood creatine phosphokinase increased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Blood creatinine increased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Cardiac murmur	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Electrocardiogram ST segment depression	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Electrocardiogram T wave inversion	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Hepatic enzyme increased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Protein urine present	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Weight decreased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Weight increased	Investigations	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Acidosis	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Gout	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Hypercholesterolaemia	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Hyperkalaemia	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Iron deficiency	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Vitamin D deficiency	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0021 affected26 at risk
Weight gain poor	Metabolism and nutrition disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0011 affected13 at risk EG0022 affected26 at risk
Flank pain	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Groin pain	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Disturbance in attention	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Dizziness	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Headache	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0003 affected13 at risk EG0012 affected13 at risk EG0025 affected26 at risk
Hypoaesthesia	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Migraine	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Presyncope	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Restless legs syndrome	Nervous system disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Post abortion haemorrhage	Pregnancy, puerperium and perinatal conditions	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Anxiety	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Attention deficit hyperactivity disorder	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Enuresis	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Fear of injection	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Irritability	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Separation anxiety disorder	Psychiatric disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Dysuria	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Haematuria	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Hypertonic bladder	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hypocitraturia	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Microalbuminuria	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Polyuria	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Proteinuria	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Renal cyst	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Renal impairment	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Renal pain	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Urinary incontinence	Renal and urinary disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Ovarian cyst	Reproductive system and breast disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Priapism	Reproductive system and breast disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Testicular pain	Reproductive system and breast disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0022 affected26 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (25.0)	Systematic Assessment	EG0001 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Actinic cheilitis	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Alopecia	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0023 affected26 at risk
Dermatitis atopic	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0020 affected26 at risk
Dry skin	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Eczema	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Livedo reticularis	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Skin hyperpigmentation	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Skin lesion	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Hypertension	Vascular disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0021 affected26 at risk
White coat hypertension	Vascular disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0021 affected26 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0012 affected13 at risk EG0022 affected26 at risk
Injection site discomfort	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0010 affected13 at risk EG0022 affected26 at risk
Injection site pain	General disorders	MedDRA (25.0)	Systematic Assessment	EG0000 affected13 at risk EG0011 affected13 at risk EG0024 affected26 at risk

ID	Term
C536414	Primary hyperoxaluria type 1
D006960	Hyperoxaluria, Primary

ID	Term
D006959	Hyperoxaluria
D007674	Kidney Diseases
D014570	Urologic Diseases
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Title	Description
BG000	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
BG001	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period.	13	0	13	9	13
EG001	Placebo/Lumasiran	Lumasiran-matching placebo was administered SC at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.	13	1	13	12	13
EG002	Lumasiran/Lumasiran	Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.	26	5	26	25	26