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

• Register: ClinicalTrials.gov
• Last refreshed on: 29 April 2024
• Main ID: NCT04152200
• Date of registration: 31/10/2019
• Prospective Registration: Yes
• Primary sponsor: Alnylam Pharmaceuticals
• Public title: A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 ILLUMINATE-C
• 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)
• Date of first enrolment: January 21, 2020
• Target sample size: 21
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04152200
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, France, Israel, Italy, Jordan, Lebanon, Netherlands, Switzerland, Turkey, United Arab Emirates, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Alnylam Pharmaceuticals

Key inclusion & exclusion criteria

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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Hyperoxaluria Type 1

Primary Hyperoxaluria

Intervention(s)

Drug: Lumasiran

Primary Outcome(s)

Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6 [Time Frame: Baseline to Month 6]

Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6 [Time Frame: Baseline to Month 6]

Secondary Outcome(s)

Area Under the Concentration-time Curve From 0 to 24 Hours [AUC(0-24)] for Lumasiran [Time Frame: Day 1; Month 6]

Change in Measures of Systemic Oxalosis From Baseline to End of Study [Time Frame: Baseline to Month 60]

Absolute Change in Plasma Oxalate From Baseline to Month 6 [Time Frame: Baseline to Month 6]

Apparent Clearance (CL/F) of Lumasiran [Time Frame: Day 1; Month 6]

Change in Mode of Dialysis From Baseline to End of Study [Time Frame: Baseline to Month 60]

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 Body Surface Area (BSA) From Baseline to Month 60 [Time Frame: Baseline to Month 60]

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]

Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 [Time Frame: Baseline to Month 6]

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]

Absolute Change in Plasma Oxalate From Baseline to Month 60 [Time Frame: Baseline to Month 60]

Change in Nephrocalcinosis From Baseline to End of Study [Time Frame: Baseline to Month 60]

Apparent Volume of Distribution (V/F) of Lumasiran [Time Frame: Day 1; Month 6]

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]

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]

Change in Frequency of Dialysis From Baseline to End of Study [Time Frame: Baseline to Month 60]

Elimination Half-life (t½ß) of Lumasiran [Time Frame: Day 1; Month 6]

Maximum Plasma Concentration (Cmax) of Lumasiran [Time Frame: Day 1; Month 6]

Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 60 [Time Frame: Baseline to Month 60]

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]

Percent Change in Plasma Oxalate From Baseline to End of Study [Time Frame: Baseline to Month 60]

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]

Change in Renal Function Assessed by Estimated Glomerular Filtration Rate (eGFR) From Baseline to End of Study [Time Frame: Baseline to Month 60]

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]

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]

Change in Frequency of Renal Stone Events From Baseline to End of Study [Time Frame: Baseline to Month 60]

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 From Baseline to Month 60 [Time Frame: Baseline to Month 60]

Time to Maximum Plasma Concentration (Tmax) of Lumasiran [Time Frame: Day 1; Month 6]

Secondary ID(s)

2019-001346-17

2023-503382-29-00

ALN-GO1-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/07/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04152200