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Completed

NCT Number: NCT04152200

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

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).

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Key information

Conditions

Primary Hyperoxaluria Type 1 Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hyperoxaluria Hyperoxaluria, Primary Kidney Diseases Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Primary Hyperoxaluria Urogenital Diseases Urologic Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Garran, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Has documented diagnosis of primary hyperoxaluria type 1 (PH1)
  • Estimated glomerular filtration rate (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

  • 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 immunosuppressants

Treatment and study plan

Lumasiran

Drug

Lumasiran will be administered by subcutaneous (SC) injection.

Other names: ALN-GO1, OXLUMO

Primary outcomes

  1. Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6

    Time frame: 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.

  2. Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6

    Time frame: 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.

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

    Time frame: 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.

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

    Time frame: 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.

  3. Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6

    Time frame: 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.

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

    Time frame: 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.

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

    Time frame: 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.

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

    Time frame: 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.

  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

    Time frame: Baseline to Month 6

    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.

  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

    Time frame: Baseline to Month 6

    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.

  9. Maximum Plasma Concentration (Cmax) of Lumasiran

    Time frame: Day 1; Month 6

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

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

    Time frame: Day 1; Month 6

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

  11. Elimination Half-life (t½β) of Lumasiran

    Time frame: Day 1; Month 6

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

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

    Time frame: Day 1; Month 6

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

  13. Apparent Clearance (CL/F) of Lumasiran

    Time frame: Day 1; Month 6

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

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

    Time frame: Day 1; Month 6

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

  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

    Time frame: Baseline to Month 60

    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.

  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

    Time frame: Baseline to Month 60

    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.

  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

    Time frame: Baseline to Month 60

    Nephrocalcinosis will be assessed by renal ultrasound.

  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

    Time frame: Baseline to Month 60

    Modes of dialysis are defined as hemodialysis and peritoneal dialysis.

  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

    Time frame: Baseline to Month 60

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

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)

Acronym: ILLUMINATE-C

Important dates

Study start
2020
Primary completion
2021
Study completion
2025
First posted
Nov 5, 2019
Registry last updated
Jul 18, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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