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CompletedNCT04152200ILLUMINATE-CUpdated Jul 18, 2025Results posted

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

A Phase 3 interventional study of Lumasiran in Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria, sponsored by Alnylam Pharmaceuticals. Completed at 14 sites in 11 countries. Per ClinicalTrials.gov, last updated 2025-07-18.

Sponsored by Alnylam Pharmaceuticals · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
21
Allocation
Not applicable
Sex
All
01

Study summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

02

Conditions studied

  • Primary Hyperoxaluria Type 1
  • Primary Hyperoxaluria

Keywords

  • PH1
  • Primary Hyperoxaluria
  • Hyperoxaluria
  • RNAi Therapeutic
  • siRNA
  • AGT
  • Systemic Oxalosis
03

In context

Primary hyperoxaluria type 1

22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.

This study's enrollment of 21 is below the median of 28 across 16 interventional studies indexed under Primary hyperoxaluria type 1.

Browse Primary hyperoxaluria type 1 studies →

Lead sponsor

Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) have results posted.

Browse Alnylam Pharmaceuticals studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

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.

Inclusion criteria

  • Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
  • Estimated (eGFR) ≤45 mL/min/1.73 m\^2 for patients ≥12 months of age (\<12 months of age, must have serum creatinine considered elevated for age)
  • Meets plasma oxalate level requirements
  • If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
  • If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks

Exclusion criteria

Exclusion Criteria:

  • Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone
  • Diagnosis of conditions other than PH1 contributing to renal insufficiency
  • History of liver transplant
  • History of kidney transplant and currently receiving
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

GFR 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.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    Lumasiran

    All patients will receive open-label lumasiran.

    Drug: Lumasiran

Interventions

  • DrugLumasiran

    Lumasiran will be administered by subcutaneous (SC) injection.

    Also known as: ALN-GO1, OXLUMO

06

What researchers measure

Primary outcomes

  1. Cohort A: Percent 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. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran.

    Time frame: Baseline to Month 6

  2. Cohort B: Percent Change in Pre-dialysis 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. For Cohort B, the baseline is defined as the mean of the last four pre-dialysis plasma oxalate samples collected prior to the first dose of lumasiran. In Cohort B, only pre-dialysis samples are utilized.

    Time frame: Baseline to Month 6

Secondary outcomes

  1. Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6

    Percent change in plasma oxalate AUC(0-24) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean value of all valid AUC (μmol/L/24h) profiles being computed prior to the first dose of lumasiran.

    Time frame: Baseline to Month 6

  2. Absolute Change in Plasma Oxalate From Baseline to Month 6

    Absolute change in plasma oxalate was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran; for Cohort B, the baseline was defined as the mean of the last 4 pre-dialysis plasma oxalate level values collected prior to the first dose of lumasiran. In Cohort B patients, the plasma oxalate baseline was similarly defined except that the values obtained from the plasma oxalate profile assessment visits only included the pre-dialysis sample collected per visit.

    Time frame: Baseline to Month 6

  3. Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (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. Baseline is defined as the median of all valid 24-hr urine assessments collected prior to the first dose date/time of lumasiran without any non-protocol-related sample issues.

    Time frame: Baseline to Month 6

  4. Cohort A: 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 is defined as the mean of all assessments prior to the first dose date/time of lumasiran.

    Time frame: Baseline to Month 6

  5. Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

    Absolute change in spot urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran.

    Time frame: Baseline to Month 6

  6. Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

    Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran.

    Time frame: Baseline to Month 6

  7. Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent

    Peds-QL is a modular approach to measuring quality of life (QOL) in healthy children and adolescents and those with acute and chronic health conditions. The PedsQL Generic Core Scales contain 23 items designed to measure core domains of health (physical, emotional, and social functioning) and role (school functioning). Scores are summarized as Total Scale Score, Physical Health Summary Score, and Psychosocial Health Summary Score. The Total Scale Score will be reported for this study. The PedsQL will be completed by patients (or caregivers, as appropriate) who are ≥2 to \<18 years of age at the time of consent. Total score range: 0 - 100, with higher scores indicating better health-related quality of life (HRQoL). Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

    Time frame: Baseline to Month 6

  8. Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent

    The KDQOL questionnaire is used to assess 3 core domains of kidney disease including: burden, symptoms/problems, and effects of kidney disease on daily life. The KDQOL subscales (Burden of Kidney Disease, Effect of Kidney Disease on Daily Life, and Symptoms and Problems of Kidney Disease), and the accompanying Short Form-12 (SF-12) Physical Component Summary and Mental Component Summary will be assessed for this study. These will be completed by patients who are ≥18 years of age at the time of consent. Score range (per domain): 0 - 100; higher scores indicate better HRQoL. Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

    Time frame: Baseline to Month 6

  9. Maximum Plasma Concentration (Cmax) of Lumasiran

    Cmax is the highest concentration of lumasiran in the plasma after a dose is given.

    Time frame: Day 1; Month 6

  10. Time to Maximum Plasma Concentration (Tmax) of Lumasiran

    Tmax is the time it takes for lumasiran to reach the maximum concentration (Cmax) after administration.

    Time frame: Day 1; Month 6

  11. Elimination Half-life (t½β) of Lumasiran

    t½β is the time it takes for the concentration of the drug in the plasma to be reduced by 50%.

    Time frame: Day 1; Month 6

  12. Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran

    AUC(0-24) is the area under the plasma concentration-time curve over the last 24-h dosing interval.

    Time frame: Day 1; Month 6

  13. Apparent Clearance (CL/F) of Lumasiran

    CL/F is the rate at which lumasiran is eliminated from the body.

    Time frame: Day 1; Month 6

  14. Apparent Volume of Distribution (V/F) of Lumasiran

    V/F is the propensity of lumasiran to either remain in the plasma or redistribute to other tissues.

    Time frame: Day 1; Month 6

  15. Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 60

    Time frame: Baseline to Month 60

  16. Absolute Change in Plasma Oxalate From Baseline to Month 60

    Time frame: Baseline to Month 60

  17. Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60

    Time frame: Baseline to Month 60

  18. Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60

    Time frame: Baseline to Month 60

  19. Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60

    Time frame: Baseline to Month 60

  20. Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60

    Time frame: Baseline to Month 60

  21. Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 60 in Patients >=2 to 18 Years of Age at Time of Informed Consent

    Peds-QL is a modular approach to measuring quality of life (QOL) in healthy children and adolescents and those with acute and chronic health conditions. The PedsQL Generic Core Scales contain 23 items designed to measure core domains of health (physical, emotional, and social functioning) and role (school functioning). Scores are summarized as Total Scale Score, Physical Health Summary Score, and Psychosocial Health Summary Score. The Total Scale Score will be reported for this study. The PedsQL will be completed by patients (or caregivers, as appropriate) who are ≥2 to \<18 years of age at the time of consent. Total score range: 0 - 100, with higher scores indicating better health-related quality of life (HRQoL). Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

    Time frame: Baseline to Month 60

  22. Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 60 in Patients ≥18 Years of Age at Time of Informed Consent

    The KDQOL questionnaire is used to assess 3 core domains of kidney disease including: burden, symptoms/problems, and effects of kidney disease on daily life. The KDQOL subscales (Burden of Kidney Disease, Effect of Kidney Disease on Daily Life, and Symptoms and Problems of Kidney Disease), and the accompanying Short Form-12 (SF-12) Physical Component Summary and Mental Component Summary will be assessed for this study. These will be completed by patients who are ≥18 years of age at the time of consent. Score range (per domain): 0 - 100; higher scores indicate better HRQoL. Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

    Time frame: Baseline to Month 60

  23. Percent Change in Plasma Oxalate From Baseline to End of Study

    Time frame: Baseline to Month 60

  24. Change in Nephrocalcinosis From Baseline to End of Study

    Nephrocalcinosis will be assessed by renal ultrasound.

    Time frame: Baseline to Month 60

  25. Change in Frequency of Dialysis From Baseline to End of Study

    Time frame: Baseline to Month 60

  26. Change in Mode of Dialysis From Baseline to End of Study

    Modes of dialysis are defined as hemodialysis and peritoneal dialysis.

    Time frame: Baseline to Month 60

  27. Change in Frequency of Renal Stone Events From Baseline to End of Study

    Time frame: Baseline to Month 60

  28. Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study

    Time frame: Baseline to Month 60

  29. Change in Measures of Systemic Oxalosis From Baseline to End of Study

    Systemic oxalosis will be assessed in cardiac, skeletal, ophthalmologic, and dermatologic systems.

    Time frame: Baseline to Month 60

07

Results

Posted Jul 12, 2022

Participant flow

Patients with advanced primary hyperoxaluria type 1 (PH1) were enrolled at thirteen sites in Australia, Belgium, France, Israel, Jordan, Lebanon, the Netherlands, Turkey, the United Arab Emirates and the United States.

Primary Analysis Period
Participant flow — Primary Analysis Period
MilestoneCohort A: LumasiranCohort B: Lumasiran
Started615
Completed615
Not completed00
Long-term Extension Period
Participant flow — Long-term Extension Period
MilestoneCohort A: LumasiranCohort B: Lumasiran
Started615
Completed00
Not completed615
Withdrew: Entered long-term extension period after completion of the primary analysis period615

Outcome measures

PrimaryCohort A: Percent 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. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · percent change
Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6
percent changeCohort A: Lumasiran
Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6-33.33 ± 17.63
Statistical analysis
  • Cohort A: Lumasiran · Mixed model repeated measures (MMRM) · Least squares (ls) mean: -33.33 · 95% CI -81.82 to 15.16
PrimaryCohort B: Percent Change in Pre-dialysis 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. For Cohort B, the baseline is defined as the mean of the last four pre-dialysis plasma oxalate samples collected prior to the first dose of lumasiran. In Cohort B, only pre-dialysis samples are utilized.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · percent change
Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6
percent changeCohort B: Lumasiran
Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6-42.43 ± 3.95
Statistical analysis
  • Cohort B: Lumasiran · MMRM · Least squares (ls) mean: -42.43 · 95% CI -50.71 to -34.15
SecondaryCohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6

Percent change in plasma oxalate AUC(0-24) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean value of all valid AUC (μmol/L/24h) profiles being computed prior to the first dose of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · percent change
Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6
percent changeCohort B: Lumasiran
Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6-41.4 ± 4.4
SecondaryAbsolute Change in Plasma Oxalate From Baseline to Month 6

Absolute change in plasma oxalate was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran; for Cohort B, the baseline was defined as the mean of the last 4 pre-dialysis plasma oxalate level values collected prior to the first dose of lumasiran. In Cohort B patients, the plasma oxalate baseline was similarly defined except that the values obtained from the plasma oxalate profile assessment visits only included the pre-dialysis sample collected per visit.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · μmol/L
Absolute Change in Plasma Oxalate From Baseline to Month 6
μmol/LCohort A: LumasiranCohort B: Lumasiran
Absolute Change in Plasma Oxalate From Baseline to Month 6-35.28 ± 7.40-48.33 ± 3.63
SecondaryCohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (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. Baseline is defined as the median of all valid 24-hr urine assessments collected prior to the first dose date/time of lumasiran without any non-protocol-related sample issues.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · mmol/24hr/1.73m^2
Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
mmol/24hr/1.73m^2Cohort A: Lumasiran
Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6-0.533 ± 0.111
SecondaryCohort A: 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 is defined as the mean of all assessments prior to the first dose date/time of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · percent change
Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
percent changeCohort A: Lumasiran
Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6-10.557 ± 6.811
SecondaryCohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

Absolute change in spot urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · ratio
Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
ratioCohort A: Lumasiran
Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6-0.1879 ± 0.0160
SecondaryCohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6

Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Least squares mean · percent change
Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
percent changeCohort A: Lumasiran
Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6-39.5136 ± 9.4294
SecondaryChange in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent

Peds-QL is a modular approach to measuring quality of life (QOL) in healthy children and adolescents and those with acute and chronic health conditions. The PedsQL Generic Core Scales contain 23 items designed to measure core domains of health (physical, emotional, and social functioning) and role (school functioning). Scores are summarized as Total Scale Score, Physical Health Summary Score, and Psychosocial Health Summary Score. The Total Scale Score will be reported for this study. The PedsQL will be completed by patients (or caregivers, as appropriate) who are ≥2 to \<18 years of age at the time of consent. Total score range: 0 - 100, with higher scores indicating better health-related quality of life (HRQoL). Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Mean · score on a scale
Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent
score on a scaleCohort A: LumasiranCohort B: Lumasiran
Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent-23.91 ± 9.220.10 ± 8.52
SecondaryChange in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent

The KDQOL questionnaire is used to assess 3 core domains of kidney disease including: burden, symptoms/problems, and effects of kidney disease on daily life. The KDQOL subscales (Burden of Kidney Disease, Effect of Kidney Disease on Daily Life, and Symptoms and Problems of Kidney Disease), and the accompanying Short Form-12 (SF-12) Physical Component Summary and Mental Component Summary will be assessed for this study. These will be completed by patients who are ≥18 years of age at the time of consent. Score range (per domain): 0 - 100; higher scores indicate better HRQoL. Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

Time frame:
Baseline to Month 6
Reported as:
Mean · score on a scale
Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent
score on a scaleCohort A: LumasiranCohort B: Lumasiran
SF-12 Physical Component Score-1.56 ± 1.19-2.06 ± 6.59
SF-12 Mental Component Score-3.88 ± 4.451.58 ± 9.61
Symptoms/Problems of Kidney Disease-13.64 ± 6.43-5.73 ± 7.49
Effects of Kidney Disease-15.63 ± 22.10-10.16 ± 14.96
Burden of Kidney Disease-3.13 ± 4.42-4.69 ± 16.44
SecondaryMaximum Plasma Concentration (Cmax) of Lumasiran

Cmax is the highest concentration of lumasiran in the plasma after a dose is given.

Time frame:
Day 1; Month 6
Reported as:
Geometric mean · ng/mL
Maximum Plasma Concentration (Cmax) of Lumasiran
ng/mLCohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 11019.9 ± 61.61820.5 ± 113.21542.8 ± 103.7
Month 61038.2 ± 36.91289.3 ± 106.01211.9 ± 87.6
SecondaryTime to Maximum Plasma Concentration (Tmax) of Lumasiran

Tmax is the time it takes for lumasiran to reach the maximum concentration (Cmax) after administration.

Time frame:
Day 1; Month 6
Reported as:
Median · hours
Time to Maximum Plasma Concentration (Tmax) of Lumasiran
hoursCohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 13.8083 (1.767 to 11.533)4.0000 (1.717 to 12.000)4.0000 (1.717 to 12.000)
Month 63.9083 (1.983 to 7.617)4.0167 (1.967 to 12.000)4.0000 (1.967 to 12.000)
SecondaryElimination Half-life (t½β) of Lumasiran

t½β is the time it takes for the concentration of the drug in the plasma to be reduced by 50%.

Time frame:
Day 1; Month 6
Reported as:
Median · hours
Elimination Half-life (t½β) of Lumasiran
hoursCohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 19.0246 (3.364 to 10.593)3.6670 (1.127 to 7.793)3.8302 (1.127 to 10.593)
Month 68.1069 (2.420 to 10.796)4.5131 (1.388 to 7.354)5.7484 (1.388 to 10.796)
SecondaryArea Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran

AUC(0-24) is the area under the plasma concentration-time curve over the last 24-h dosing interval.

Time frame:
Day 1; Month 6
Reported as:
Geometric mean · h*ng/mL
Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran
h*ng/mLCohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 113156.8031 ± 64.613170.3716 ± 35.613164.1075 ± 47.7
Month 611417.0267 ± 33.520647.6317 ± 61.614856.4905 ± 55.9
SecondaryApparent Clearance (CL/F) of Lumasiran

CL/F is the rate at which lumasiran is eliminated from the body.

Time frame:
Day 1; Month 6
Reported as:
Geometric mean · Liters/hour (L/h)
Apparent Clearance (CL/F) of Lumasiran
Liters/hour (L/h)Cohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 17.8440 ± 105.55.1003 ± 77.75.7356 ± 82.6
Month 64.8496 ± 112.86.8556 ± 44.85.9104 ± 70.1
SecondaryApparent Volume of Distribution (V/F) of Lumasiran

V/F is the propensity of lumasiran to either remain in the plasma or redistribute to other tissues.

Time frame:
Day 1; Month 6
Reported as:
Geometric mean · Liters (L)
Apparent Volume of Distribution (V/F) of Lumasiran
Liters (L)Cohort A: LumasiranCohort B: LumasiranAll Lumasiran
Day 177.5329 ± 91.125.7429 ± 196.434.7734 ± 185.7
Month 641.7081 ± 229.336.8305 ± 166.938.8468 ± 161.3
SecondaryPercent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryAbsolute Change in Plasma Oxalate From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryAbsolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryPercent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryAbsolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryPercent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 60 in Patients >=2 to 18 Years of Age at Time of Informed Consent

Peds-QL is a modular approach to measuring quality of life (QOL) in healthy children and adolescents and those with acute and chronic health conditions. The PedsQL Generic Core Scales contain 23 items designed to measure core domains of health (physical, emotional, and social functioning) and role (school functioning). Scores are summarized as Total Scale Score, Physical Health Summary Score, and Psychosocial Health Summary Score. The Total Scale Score will be reported for this study. The PedsQL will be completed by patients (or caregivers, as appropriate) who are ≥2 to \<18 years of age at the time of consent. Total score range: 0 - 100, with higher scores indicating better health-related quality of life (HRQoL). Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 60 in Patients ≥18 Years of Age at Time of Informed Consent

The KDQOL questionnaire is used to assess 3 core domains of kidney disease including: burden, symptoms/problems, and effects of kidney disease on daily life. The KDQOL subscales (Burden of Kidney Disease, Effect of Kidney Disease on Daily Life, and Symptoms and Problems of Kidney Disease), and the accompanying Short Form-12 (SF-12) Physical Component Summary and Mental Component Summary will be assessed for this study. These will be completed by patients who are ≥18 years of age at the time of consent. Score range (per domain): 0 - 100; higher scores indicate better HRQoL. Baseline is defined as last non-missing value collected prior to the first dose of lumasiran.

Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryPercent Change in Plasma Oxalate From Baseline to End of Study
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Nephrocalcinosis From Baseline to End of Study

Nephrocalcinosis will be assessed by renal ultrasound.

Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Frequency of Dialysis From Baseline to End of Study
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Mode of Dialysis From Baseline to End of Study

Modes of dialysis are defined as hemodialysis and peritoneal dialysis.

Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Frequency of Renal Stone Events From Baseline to End of Study
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study
Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

SecondaryChange in Measures of Systemic Oxalosis From Baseline to End of Study

Systemic oxalosis will be assessed in cardiac, skeletal, ophthalmologic, and dermatologic systems.

Time frame:
Baseline to Month 60

Results for this outcome have not been posted.

Adverse events

Collected over Non-serious adverse events (AEs) are reported from the administration of the first dose of study drug up to approximately 6 months. Serious adverse events (SAEs) are reported from the time when the informed consent is signed up to approximately 6 months.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Cohort A: Lumasiran0/6 (0%)1/6 (16.7%)5/6 (83.3%)
Cohort B: Lumasiran0/15 (0%)8/15 (53.3%)13/15 (86.7%)
Most frequent serious events
Showing 10 of 20
Most frequent serious events
EventCohort A: LumasiranCohort B: Lumasiran
VomitingGastrointestinal disorders1/60/15
HypokalaemiaMetabolism and nutrition disorders1/60/15
Device related infectionInfections and infestations0/62/15
Renal transplantSurgical and medical procedures0/62/15
AnaemiaBlood and lymphatic system disorders0/61/15
Spontaneous haematomaBlood and lymphatic system disorders0/61/15
Abdominal painGastrointestinal disorders0/61/15
PancreatitisGastrointestinal disorders0/61/15
Catheter site swellingGeneral disorders0/61/15
Device related thrombosisGeneral disorders0/61/15
Most frequent other events
Showing 10 of 55
Most frequent other events
EventCohort A: LumasiranCohort B: Lumasiran
PyrexiaGeneral disorders1/66/15
Injection site reactionGeneral disorders1/64/15
DiarrhoeaGastrointestinal disorders1/63/15
ConstipationGastrointestinal disorders1/61/15
Candida nappy rashInfections and infestations1/60/15
Upper respiratory tract infectionInfections and infestations1/61/15
Urinary tract infectionInfections and infestations1/60/15
Burns second degreeInjury, poisoning and procedural complications1/60/15
Upper limb fractureInjury, poisoning and procedural complications1/60/15
Blood phosphorus increasedInvestigations1/60/15

Baseline characteristics

Safety Analysis Set (SAS): All patients who received any amount of lumasiran during the study.

Age, Continuous
Age, Continuous(years)Cohort A: LumasiranCohort B: LumasiranTotal
Median9.0 (0 to 40)6.0 (1 to 59)8.0 (0 to 59)
Sex: Female, Male
Sex: Female, Male(Participants)Cohort A: LumasiranCohort B: LumasiranTotal
Female369
Male3912
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Cohort A: LumasiranCohort B: LumasiranTotal
Asian134
White41216
Other101
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Cohort A: LumasiranCohort B: LumasiranTotal
Hispanic or Latino011
Not Hispanic or Latino61420
Plasma Oxalate
Plasma Oxalate(μmol/L)Cohort A: LumasiranCohort B: LumasiranTotal
Mean64.73 ± 41.30108.35 ± 29.5395.89 ± 38.01
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Study locations

14 sites
  • Clinical Trial Site
    Rochester, Minnesota 55902, United States
  • Clinical Trial Site
    Houston, Texas 77030, United States
  • Clinical Trial Site
    Garran, Australia
  • Clinical Trial Site
    Brussels, Belgium
  • Clinical Trial Site
    Bron, France
  • Clinical Trial Site
    Lyon, France
  • Clinical Trial Site
    Haifa, Israel
  • Clinical Trial Site
    Nahariya, Israel
  • Clinical Trial Site
    Rome, Italy
  • Clinical Trial Site
    Irbid, Jordan
  • Clinical Trial Site
    Beirut, Lebanon
  • Clinical Trial Site
    Amsterdam, Netherlands
  • Clinical Trial Site
    Yenimahalle, Turkey (Türkiye)
  • Clinical Trial Site
    Dubai, United Arab Emirates
09

References and documents

Publications

  • Sankar A, Y S RK, Singh A, Roy R, Shukla R, Verma B. Next-generation therapeutics for rare genetic disorders. Mutagenesis. 2024 Apr 24;39(3):157-171. doi: 10.1093/mutage/geae002. PubMed 38332115 ↗
  • Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2023 Feb;81(2):145-155.e1. doi: 10.1053/j.ajkd.2022.05.012. Epub 2022 Jul 14. PubMed 35843439 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: Yes — Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Jan 2020 to primary completion in May 2021, 15 months.

Results were first posted in Jul 2022, 1 year 1 month after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 31, 2019
First posted
Nov 5, 2019
StartActual
Jan 21, 2020
Primary completionActual
May 18, 2021
CompletionActual
Jun 23, 2025
Results first posted
Jul 12, 2022
Last update
Jul 18, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jul 18, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04152200
Lead sponsor
Alnylam Pharmaceuticals
Responsible party
Sponsor
First posted
Nov 5, 2019
Start date
Jan 21, 2020
Primary completion
May 18, 2021
Completion
Jun 23, 2025
Results posted
Jul 12, 2022
Last update
Jul 18, 2025

Study contacts

Medical Director
study director · Alnylam Pharmaceuticals

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Lumasiran

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