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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04152200
Registration number
NCT04152200
Ethics application status
Date submitted
31/10/2019
Date registered
5/11/2019
Date last updated
13/05/2024
Titles & IDs
Public title
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
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Scientific 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)
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Secondary ID [1]
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2019-001346-17
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Secondary ID [2]
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ALN-GO1-005
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Universal Trial Number (UTN)
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Trial acronym
ILLUMINATE-C
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Primary Hyperoxaluria Type 1
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Primary Hyperoxaluria
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Condition category
Condition code
Renal and Urogenital
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Other renal and urogenital disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Treatment: Drugs - Lumasiran
Experimental: Lumasiran - All patients will receive open-label lumasiran.
Treatment: Drugs: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline to Month 6
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Primary outcome [2]
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Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline to Month 6
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Secondary outcome [1]
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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
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline to Month 6
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Secondary outcome [2]
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Absolute Change in Plasma Oxalate From Baseline to Month 6
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline to Month 6
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Secondary outcome [3]
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Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline to Month 6
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Secondary outcome [4]
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Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline to Month 6
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Secondary outcome [5]
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Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline to Month 6
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Secondary outcome [6]
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Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline to Month 6
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Secondary outcome [7]
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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
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Assessment method [7]
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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.
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Timepoint [7]
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Baseline to Month 6
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Secondary outcome [8]
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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
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline to Month 6
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Secondary outcome [9]
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Maximum Plasma Concentration (Cmax) of Lumasiran
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Assessment method [9]
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Cmax is the highest concentration of lumasiran in the plasma after a dose is given.
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Timepoint [9]
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Day 1; Month 6
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Secondary outcome [10]
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Time to Maximum Plasma Concentration (Tmax) of Lumasiran
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Assessment method [10]
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Tmax is the time it takes for lumasiran to reach the maximum concentration (Cmax) after administration.
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Timepoint [10]
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Day 1; Month 6
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Secondary outcome [11]
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Elimination Half-life (t½ß) of Lumasiran
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Assessment method [11]
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t½ß is the time it takes for the concentration of the drug in the plasma to be reduced by 50%.
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Timepoint [11]
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Day 1; Month 6
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Secondary outcome [12]
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Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran
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Assessment method [12]
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AUC(0-24) is the area under the plasma concentration-time curve over the last 24-h dosing interval.
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Timepoint [12]
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Day 1; Month 6
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Secondary outcome [13]
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Apparent Clearance (CL/F) of Lumasiran
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Assessment method [13]
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CL/F is the rate at which lumasiran is eliminated from the body.
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Timepoint [13]
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Day 1; Month 6
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Secondary outcome [14]
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Apparent Volume of Distribution (V/F) of Lumasiran
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Assessment method [14]
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V/F is the propensity of lumasiran to either remain in the plasma or redistribute to other tissues.
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Timepoint [14]
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Day 1; Month 6
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Secondary outcome [15]
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Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 60
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Assessment method [15]
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Timepoint [15]
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Baseline to Month 60
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Secondary outcome [16]
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Absolute Change in Plasma Oxalate From Baseline to Month 60
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Assessment method [16]
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Timepoint [16]
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Baseline to Month 60
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Secondary outcome [17]
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Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
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Assessment method [17]
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Timepoint [17]
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Baseline to Month 60
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Secondary outcome [18]
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Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 60
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Assessment method [18]
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Timepoint [18]
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Baseline to Month 60
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Secondary outcome [19]
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Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
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Assessment method [19]
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Timepoint [19]
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Baseline to Month 60
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Secondary outcome [20]
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Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60
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Assessment method [20]
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Timepoint [20]
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Baseline to Month 60
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Secondary outcome [21]
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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
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Assessment method [21]
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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.
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Timepoint [21]
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Baseline to Month 60
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Secondary outcome [22]
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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
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Assessment method [22]
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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.
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Timepoint [22]
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Baseline to Month 60
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Secondary outcome [23]
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Percent Change in Plasma Oxalate From Baseline to End of Study
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Assessment method [23]
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Timepoint [23]
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Baseline to Month 60
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Secondary outcome [24]
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Change in Nephrocalcinosis From Baseline to End of Study
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Assessment method [24]
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Nephrocalcinosis will be assessed by renal ultrasound.
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Timepoint [24]
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Baseline to Month 60
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Secondary outcome [25]
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Change in Frequency of Dialysis From Baseline to End of Study
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Assessment method [25]
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Timepoint [25]
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Baseline to Month 60
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Secondary outcome [26]
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Change in Mode of Dialysis From Baseline to End of Study
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Assessment method [26]
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Modes of dialysis are defined as hemodialysis and peritoneal dialysis.
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Timepoint [26]
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Baseline to Month 60
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Secondary outcome [27]
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Change in Frequency of Renal Stone Events From Baseline to End of Study
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Assessment method [27]
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Timepoint [27]
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Baseline to Month 60
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Secondary outcome [28]
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Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study
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Assessment method [28]
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Timepoint [28]
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Baseline to Month 60
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Secondary outcome [29]
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Change in Measures of Systemic Oxalosis From Baseline to End of Study
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Assessment method [29]
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Systemic oxalosis will be assessed in cardiac, skeletal, ophthalmologic, and dermatologic systems.
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Timepoint [29]
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Baseline to Month 60
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Eligibility
Key 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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
21/01/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/07/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
21
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Clinical Trial Site - Garran
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Recruitment postcode(s) [1]
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- Garran
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Minnesota
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Country [2]
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United States of America
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State/province [2]
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Texas
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Country [3]
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Belgium
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State/province [3]
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Brussels
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Country [4]
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France
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State/province [4]
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Bron
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Country [5]
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France
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State/province [5]
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Lyon
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Country [6]
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Israel
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State/province [6]
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Haifa
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Country [7]
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Israel
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State/province [7]
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Nahariya
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Country [8]
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Italy
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State/province [8]
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Rome
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Country [9]
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Jordan
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State/province [9]
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Irbid
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Country [10]
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Lebanon
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State/province [10]
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Beirut
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Country [11]
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Netherlands
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State/province [11]
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Amsterdam
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Country [12]
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Turkey
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State/province [12]
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Yenimahalle
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Country [13]
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United Arab Emirates
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State/province [13]
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Dubai
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alnylam Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04152200
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director
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Address
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Alnylam Pharmaceuticals
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04152200
Download to PDF