A Phase 1/2 interventional study of Lumasiran and Placebo in Primary Hyperoxaluria Type 1 (PH1), sponsored by Alnylam Pharmaceuticals. Completed at 9 sites in 5 countries. Open to participants aged 6 Years to 64 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2020-01-30.
Sponsored by Alnylam Pharmaceuticals · Phase 1/2, Interventional, and Treatment
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). In Part A, single ascending dose (SAD) part, healthy adults were dosed with lumasiran or placebo once. In Part B, multiple ascending doses (MAD) part, patients with primary hyperoxaluria type 1 (PH1) were dosed with lumasiran or placebo. All patients that initially received placebo received lumasiran after completing placebo dosing.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's enrollment of 52 is above the median of 28 across 16 interventional studies indexed under Primary hyperoxaluria type 1.
Browse Primary hyperoxaluria type 1 studies →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.
Counted across the registry records on this site, refreshed daily.
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.
Additional Inclusion Criteria for Part B:
Exclusion Criteria for Parts A and B:
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.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A single dose of matching placebo will be administered subcutaneously (SC).
Drug: Placebo
A single dose of 0.3 mg/kg lumasiran will be administered SC.
Drug: Lumasiran
A single dose of 1.0 mg/kg lumasiran will be administered SC.
Drug: Lumasiran
A single dose of 3.0 mg/kg lumasiran will be administered SC.
Drug: Lumasiran
A single dose of 6.0 mg/kg lumasiran will be administered SC.
Drug: Lumasiran
Participants with primary hyperoxaluria type 1 (PH1) will be treated with placebo matching one of the lumasiran dosages in the lumasiran arms (one placebo participant for each lumasiran arm). At Day 85 these placebo treated participants will cross over to their respective Part B lumasiran arms in the Part B: MAD Study Day 85-End of Study Period and will then be treated with lumasiran. The estimated total time on study was up to 546 days.
Drug: Placebo
Participants with PH1 will be treated with 1.0 mg/kg lumasiran SC once monthly (qM) on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85.
Drug: Lumasiran
Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC qM on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85.
Drug: Lumasiran
Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC once every three months (q3M) on Days 1 and 85. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85.
Drug: Lumasiran
Lumasiran will be administered SC once in Part A. In Part B lumasiran will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).
Also known as: ALN-GO1
Matching placebo (sterile saline: 0.9% sodium chloride \[NaCl\]) will be administered SC once in Part A. In Part B matching placebo will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).
Number of Participants With Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Time frame: Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days
Maximum Concentration (Cmax) of Lumasiran in Plasma
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
Time to Cmax (Tmax) of Lumasiran in Plasma
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
Area Under the Concentration-Time Curve From Time 0 to Time of Last Measurable Concentration (AUC0-last) of Lumasiran in Plasma
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
Terminal Half-life (t1/2) of Lumasiran in Plasma
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
Fraction Excreted in Urine in 24 Hours (Fe0-24) of Lumasiran
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
Renal Clearance (CLR) of Lumasiran
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
Time frame: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
Baseline Plasma Glycolate Concentration
The pharmacodynamic (PD) outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated.
Time frame: Part A (SAD): Baseline, Part B (MAD): Baseline
Percentage Change From Baseline in Plasma Glycolate Concentration
The PD outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated.
Time frame: Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85
Baseline Spot Urine Glycolate:Creatinine Ratio in Part A
The endpoint was only measured in Part A.
Time frame: Part A (SAD): Baseline
Percentage Change From Baseline in Spot Urine Glycolate:Creatinine Ratio in Part A
The endpoint was only measured in Part A.
Time frame: Part A (SAD): Days 29 and 57
Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
The endpoint was only measured in Part B.
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
The endpoint was only measured in Part B.
Time frame: Part B (MAD): 24 hour urine collections on Days 29, 57, 85, 113, 141, 169, 197
Baseline 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
The endpoint was only measured during the initial 85 days in Part B.
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
The endpoint was only measured during the initial 85 days in Part B.
Time frame: Part B (MAD): 24 hour urine collections on Days 29, 57 and 85
Baseline Creatinine Clearance Corrected for BSA in Part B
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of Creatinine Clearance Corrected for BSA in Part B
Time frame: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
Participants were enrolled at ten sites in Germany, France, the United Kingdom, Israel, and the Netherlands.
| Milestone | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Started | 8 | 6 | 6 | 6 | 6 | 0 | 0 | 0 | 0 |
| Completed | 8 | 6 | 6 | 4 | 6 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 2 | 0 | 0 | 0 | 0 | 0 |
| Milestone | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 0 | 3 | 7 | 7 | 3 |
| Completed | 0 | 0 | 0 | 0 | 0 | 3 | 7 | 7 | 3 |
| Not completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Milestone | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 0 | 0 | 8 | 8 | 4 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 | 8 | 8 | 4 |
| Not completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
An AE is any untoward medical occurrence in a clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
| Participants | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs) | 5 | 6 | 2 | 6 | 6 | 2 | 8 | 7 | 4 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| ng/mL | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|
| Day 1 | 39.7940 ± 8.58882 | 204.3748 ± 111.68091 | 533.4527 ± 160.11060 | 1176.1302 ± 199.89797 | 324.1386 ± 489.71104 | 582.4515 ± 266.90105 | 432.2798 ± 245.02660 |
| Day 57 | — | — | — | — | 147.6780 ± 67.97968 | 701.1708 ± 511.63001 | — |
| Day 85 | — | — | — | — | — | — | 411.5613 ± 174.92146 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| hours | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|
| Day 1 | 5.0167 (4.000 to 8.017) | 1.5000 (0.517 to 8.000) | 3.0000 (0.500 to 8.000) | 7.0000 (0.500 to 8.067) | 3.9917 (0.567 to 5.967) | 4.9917 (0.533 to 12.000) | 9.0000 (5.783 to 12.017) |
| Day 57 | — | — | — | — | 3.0417 (0.500 to 6.000) | 2.9833 (0.500 to 8.000) | — |
| Day 85 | — | — | — | — | — | — | 5.9833 (4.050 to 7.950) |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| h*ng/mL | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|
| Day 1 | 293.5232 ± 96.86989 | 1899.8119 ± 558.25326 | 7211.5890 ± 1125.64173 | 16778.0579 ± 4380.15325 | 1428.0412 ± 697.85233 | 7400.2181 ± 2331.89843 | 6337.9082 ± 3840.03340 |
| Day 57 | — | — | — | — | 1608.1457 ± 708.95156 | 7959.7873 ± 1726.57675 | — |
| Day 85 | — | — | — | — | — | — | 5136.3462 ± 2757.90139 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| hours | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|
| Day 1 | 7.0655 ± 0.37379 | 5.9798 ± 1.52471 | 3.4683 ± NA | 3.2670 ± 1.52759 | 5.4574 ± 3.49432 | 7.8028 ± NA |
| Day 57 | — | — | — | 7.8090 ± 4.52009 | 5.8356 ± 3.12156 | — |
| Day 85 | — | — | — | — | — | 4.6694 ± NA |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| percentage fractional excretion | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|
| Day 1 | 17.4219 ± 2.44129 | 19.0713 ± 3.88914 | 21.0472 ± 5.36667 | 25.7931 ± 3.25937 | 11.0895 ± 3.74207 | 11.1877 ± 6.07719 | 7.1691 ± 2.37465 |
| Day 57 | — | — | — | — | 9.4698 ± 4.21949 | 12.4604 ± 4.02897 | — |
| Day 85 | — | — | — | — | — | — | 13.6938 ± 3.60004 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups.
| L/h | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|
| Day 1 | 8.7817 ± NA | 5.4906 ± 2.07402 | 5.8211 ± 1.31377 | 6.3417 ± 1.15497 | 2.2612 ± 1.17616 | 2.3818 ± 1.13067 | 2.0564 ± 1.20600 |
| Day 57 | — | — | — | — | 1.9610 ± 1.11228 | 2.5150 ± 0.80386 | — |
| Day 85 | — | — | — | — | — | — | 3.3663 ± 1.18371 |
The pharmacodynamic (PD) outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated.
| umol/L | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Baseline Plasma Glycolate Concentration | 5.1 ± 1.73 | 5.3 ± 1.51 | 5.7 ± 1.97 | 6.2 ± 2.56 | 4.8 ± 1.72 | — | — | — | — |
The PD outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated.
| percentage change from baseline | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Day 15 | 18.3 ± 67.19 | 58.3 ± 55.29 | 48.5 ± 82.99 | 56.4 ± 28.50 | 59.5 ± 49.00 | — | — | — | — |
| Day 29 | 22.4 ± 46.83 | 32.9 ± 57.67 | 70.6 ± 82.74 | 146.4 ± 81.99 | 390.1 ± 270.40 | — | — | — | — |
| Day 57 | 126.7 ± 242.68 | 66.3 ± 38.07 | 109.8 ± 124.29 | 230.1 ± 180.36 | 730.4 ± 439.54 | — | — | — | — |
| Day 85 | 31.2 ± 131.04 | 15.6 ± 100.54 | 40.7 ± 110.75 | 196.2 ± 152.41 | 731.3 ± 375.02 | — | — | — | — |
The endpoint was only measured in Part A.
| mg/g | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg |
|---|---|---|---|---|---|
| Baseline Spot Urine Glycolate:Creatinine Ratio in Part A | 12.4 ± 4.63 | 15.7 ± 4.27 | 15.7 ± 3.14 | 13.0 ± 3.52 | 14.8 ± 4.31 |
The endpoint was only measured in Part A.
| percentage change from baseline | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg |
|---|---|---|---|---|---|
| Day 29 | 8.1 ± 43.42 | 32.5 ± 22.6 | 82.9 ± 65.00 | 109.1 ± 66.51 | 210.5 ± 199.30 |
| Day 57 | 73.8 ± 108.9 | 38.0 ± 50.62 | 47.8 ± 41.03 | 215.0 ± 178.72 | 310.7 ± 94.51 |
The endpoint was only measured in Part B.
| mmol/24h/1.73m^2 | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B | 1.96 ± 0.321 | 1.73 ± 0.696 | 1.84 ± 0.621 | 1.30 ± 0.350 |
The endpoint was only measured in Part B.
| percentage change from baseline | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Day 29 | -2.4 ± NA | -41.1 ± 24.76 | -57.5 ± 10.84 | -49.2 ± 5.40 |
| Day 57 | -27.8 ± 47.11 | -49.7 ± 20.08 | -72.5 ± 10.70 | -49.1 ± 5.82 |
| Day 85 | 9.1 ± NA | -65.6 ± 16.64 | -68.4 ± 10.60 | -53.3 ± 3.66 |
| Day 113 | — | -61.4 ± 12.24 | -78.1 ± 7.80 | -59.1 ± 20.75 |
| Day 141 | — | -64.6 ± 13.55 | -73.5 ± 8.11 | -68.4 ± 3.21 |
| Day 169 | — | -61.6 ± 14.19 | -69.3 ± 9.61 | -48.7 ± 14.19 |
| Day 197 | — | -63.8 ± 13.85 | -71.2 ± 11.70 | -52.7 ± 6.38 |
The endpoint was only measured during the initial 85 days in Part B.
| mg/g | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Baseline 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days | 193 ± 117.2 | 241 ± 85.6 | 289 ± 146.0 | 281 ± 139.0 |
The endpoint was only measured during the initial 85 days in Part B.
| percentage change from baseline | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Day 29 | -15.1 ± 6.47 | 53.1 ± 42.18 | 31.4 ± 35.99 | 33.0 ± 36.37 |
| Day 57 | -13.8 ± 29.02 | 82.3 ± 40.12 | 42.3 ± 57.56 | 81.8 ± 35.66 |
| Day 85 | -23.0 ± 10.45 | 71.0 ± 55.62 | 43.7 ± 62.15 | 42.0 ± 20.86 |
| mL/min/1.73 m^2 | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Baseline Creatinine Clearance Corrected for BSA in Part B | 64.389 ± 19.8024 | 108.149 ± 44.1783 | 86.268 ± 22.1226 | 88.251 ± 30.0118 |
| percentage change from baseline | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|
| Day 29 | 9.019 ± NA | -6.672 ± 11.9614 | -0.624 ± 15.1771 | 2.748 ± 5.6858 |
| Day 57 | 20.184 ± 23.5572 | -8.426 ± 19.7271 | 4.505 ± 35.2457 | 37.030 ± 56.1210 |
| Day 85 | -5.945 ± NA | -13.536 ± 22.8679 | -3.720 ± 22.6231 | -6.113 ± 43.5441 |
| Day 113 | — | -14.424 ± 24.1107 | 3.444 ± 29.6749 | 7.570 ± 40.5928 |
| Day 141 | — | -8.804 ± 27.4802 | 9.315 ± 27.5044 | -30.691 ± 12.8912 |
| Day 169 | — | -19.796 ± 31.7492 | -8.544 ± 14.8481 | 20.210 ± NA |
| Day 197 | — | -8.546 ± 13.5672 | -13.513 ± 25.0560 | 28.857 ± 22.1891 |
| Day 225 | — | -18.221 ± 14.2322 | 29.013 ± 53.5563 | -14.964 ± 9.9130 |
| Day 253 | — | -5.140 ± 19.2839 | 10.232 ± 24.4929 | — |
| Day 281 | — | -14.283 ± 22.6126 | 5.841 ± 13.0381 | — |
| Day 309 | — | -0.403 ± 34.6839 | 8.441 ± 11.5329 | — |
| Day 337 | — | -5.168 ± 31.0756 | 8.688 ± 24.2094 | — |
| Day 365 | — | 2.268 ± 33.7565 | 9.201 ± 14.4856 | — |
| Day 393 | — | -11.942 ± 45.3027 | -4.484 ± 12.6320 | — |
| Day 421 | — | 7.610 ± 11.2195 | -6.498 ± 12.9022 | — |
| Day 449 | — | — | -15.093 ± 10.3705 | — |
Collected over Part A (SAD phase): Up to 405 days; Part B (MAD phase): Up to 546 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: SAD: Placebo | 0/8 (0%) | 0/8 (0%) | 5/8 (62.5%) |
| Part A: SAD: Lumasiran 0.3 mg/kg | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Part A: SAD: Lumasiran 1.0 mg/kg | 0/6 (0%) | 0/6 (0%) | 3/6 (50%) |
| Part A: SAD: Lumasiran 3.0 mg/kg | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Part A: SAD: Lumasiran 6.0 mg/kg | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Part B: MAD: Placebo | 0/3 (0%) | 1/3 (33.3%) | 2/3 (66.7%) |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 0/8 (0%) | 1/8 (12.5%) | 7/8 (87.5%) |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 0/8 (0%) | 3/8 (37.5%) | 7/8 (87.5%) |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Event | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| NephrolithiasisRenal and urinary disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 1/3 | 0/8 | 1/8 | 0/4 |
| Pyelonephritis acuteInfections and infestations | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 1/3 | 0/8 | 0/8 | 0/4 |
| VomitingGastrointestinal disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 0/8 | 2/8 | 0/4 |
| Abdominal painGastrointestinal disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 0/8 | 1/8 | 0/4 |
| PyrexiaGeneral disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 0/8 | 1/8 | 0/4 |
| GastroenteritisInfections and infestations | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 1/8 | 0/8 | 0/4 |
| Event | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M |
|---|---|---|---|---|---|---|---|---|---|
| Injection site painGeneral disorders | 0/8 | 0/6 | 0/6 | 0/6 | 4/6 | 0/3 | 0/8 | 0/8 | 0/4 |
| NasopharyngitisInfections and infestations | 3/8 | 1/6 | 1/6 | 4/6 | 4/6 | 0/3 | 0/8 | 0/8 | 2/4 |
| HeadacheNervous system disorders | 1/8 | 1/6 | 2/6 | 1/6 | 3/6 | 0/3 | 2/8 | 2/8 | 0/4 |
| Abdominal painGastrointestinal disorders | 0/8 | 0/6 | 0/6 | 1/6 | 0/6 | 0/3 | 1/8 | 3/8 | 1/4 |
| RhinitisInfections and infestations | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 1/8 | 3/8 | 0/4 |
| NephrolithiasisRenal and urinary disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 0/3 | 0/8 | 3/8 | 0/4 |
| GastroenteritisInfections and infestations | 1/8 | 0/6 | 0/6 | 0/6 | 2/6 | 0/3 | 0/8 | 0/8 | 0/4 |
| Viral pharyngitisInfections and infestations | 1/8 | 0/6 | 0/6 | 0/6 | 0/6 | 1/3 | 0/8 | 0/8 | 0/4 |
| ContusionInjury, poisoning and procedural complications | 0/8 | 0/6 | 0/6 | 0/6 | 1/6 | 1/3 | 0/8 | 0/8 | 0/4 |
| ConstipationGastrointestinal disorders | 0/8 | 0/6 | 0/6 | 0/6 | 0/6 | 1/3 | 1/8 | 0/8 | 0/4 |
Safety Analysis Set consisted of all healthy participants and participants who received at least 1 dose of study drug (lumasiran, placebo), grouped according to actual treatment received during the first period that they entered the study.
| Age, Continuous(years) | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 29.8 ± 6.25 | 28.8 ± 7.36 | 30.2 ± 7.81 | 27.3 ± 3.44 | 28.8 ± 5.46 | 20.7 ± 19.40 | 14.0 ± 9.93 | 15.4 ± 7.89 | 9.7 ± 5.51 | 23.6 ± 10.41 |
| Sex: Female, Male(Participants) | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Female | 5 | 0 | 3 | 3 | 5 | 1 | 6 | 4 | 2 | 29 |
| Male | 3 | 6 | 3 | 3 | 1 | 2 | 1 | 3 | 1 | 23 |
| Ethnicity (NIH/OMB)(Participants) | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 3 |
| Not Hispanic or Latino | 7 | 5 | 6 | 6 | 5 | 3 | 7 | 7 | 3 | 49 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Part A: SAD: Placebo | Part A: SAD: Lumasiran 0.3 mg/kg | Part A: SAD: Lumasiran 1.0 mg/kg | Part A: SAD: Lumasiran 3.0 mg/kg | Part A: SAD: Lumasiran 6.0 mg/kg | Part B: MAD: Placebo | Part B: MAD: Lumasiran 1.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg qM | Part B: MAD: Lumasiran 3.0 mg/kg q3M | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 1 | 0 | 0 | 1 | 0 | 1 | 2 | 1 | 0 | 6 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 2 |
| White | 7 | 6 | 5 | 3 | 4 | 2 | 5 | 5 | 3 | 40 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 | 1 | 0 | 0 | 1 | 0 | 4 |
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Studies of the drug tested here: Lumasiran
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