A Phase 3 interventional study of Placebo and Lumasiran in Primary Hyperoxaluria Type 1 (PH1), sponsored by Alnylam Pharmaceuticals. Completed at 17 sites in 8 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-08-12.
Sponsored by Alnylam Pharmaceuticals · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
This study's enrollment of 39 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.
Exclusion Criteria:
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.
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.
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.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
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.
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.
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.
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.
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.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
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.
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.
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.
Lumasiran-matching placebo (normal saline \[0.9% NaCl\]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period.
Drug: Placebo · Drug: Lumasiran
Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
Drug: Placebo · Drug: Lumasiran
Placebo by SC injection
Lumasiran by SC injection
Also known as: ALN-GO1
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.
Time frame: Baseline to Month 6
Absolute Change in 24-hour Urinary Oxalate Corrected for 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.
Time frame: Baseline to Month 6
Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Percent change in 24-hour urinary oxalate:creatine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
Time frame: Baseline to Month 6
Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
Time frame: Month 6
Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
Time frame: Month 6
Percentage 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.
Time frame: Baseline to Month 6
Absolute Change in Plasma Oxalate From Baseline to Month 6
Absolute 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.
Time frame: Baseline to Month 6
Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6
eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients \>1 year to \<18 years of age at screening. Change from baseline to Month 6 is reported.
Time frame: Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6
Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period
Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.
Time frame: From Baseline to Month 54 and Month 60
Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period
Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.
Time frame: From Baseline to Month 54 and Month 60
Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During Lumasiran Treatment
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
Time frame: Up to Month 60
Absolute Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline in the Extension Period
Absolute change in 24-hour urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline to the end of the OLE period at Month 54 and Month 60. A negative change from Baseline indicates a favorable outcome.
Time frame: From Baseline to Month 54 and Month 60
Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline in the Extension Period
eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients \>1 year to \<18 years of age at screening.
Time frame: From Baseline to Month 54 and Month 60
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a clinical investigational participant administered a medicinal product \& which does not necessarily have a causal relationship with this treatment. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability or incapacity, is a congenital anomaly or birth defect, is an important medical event that may not be immediately life-threatening/result in death/hospitalization but may jeopardize the participant \& may require intervention to prevent one of the other outcomes listed above. All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during 6-month double-blinded period \& participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period.
Time frame: DB Period (Placebo): From first dose of study drug (Day 1) up to Month 6; Placebo/Lumasiran: From first dose of lumasiran (Month 6) up to end of study (Month 60); Lumasiran/Lumasiran: From first dose of lumasiran (Day 1) up to end of study (Month 60).
Rate of Renal Stone Events
A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone. Lower rates indicate a favorable outcome.
Time frame: 12-Month Period prior to Informed Consent, 6-Month DB Period
Change From Baseline in Nephrocalcinosis as Assessed by Renal Ultrasound
Renal ultrasound data were used to grade medullary nephrocalcinosis findings (range: 0 to 3), where a higher grade indicates greater severity. Improving=if both sides improve, or one side improves and the other side has no change; No change=if both sides have no change; Worsening=if both sides worsen, or one side worsens and the other side has no change; Indeterminate=if one side improves and the other side worsens.
Time frame: Baseline, Month 6
Participants with PH1 were enrolled at sixteen sites in France, Germany, Israel, the Netherlands, Switzerland, the United Arab Emirates, the United Kingdom and the United States.
| Milestone | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Started | 13 | 26 |
| Completed | 13 | 25 |
| Not completed | 0 | 1 |
| Withdrew: Parent/caregiver withdrew consent | 0 | 1 |
| Milestone | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Started | 13 | 24 |
| Completed | 13 | 24 |
| Not completed | 0 | 0 |
| Milestone | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Started | 13 | 24 |
| Completed | 13 | 24 |
| Not completed | 0 | 0 |
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.
| percent change | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | -11.8 ± 3.8 | -65.4 ± 2.9 |
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.
| mmol/24hr/1.73m^2 | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 | -0.27 ± 0.08 | -1.24 ± 0.06 |
Percent change in 24-hour urinary oxalate:creatine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
| percent change | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | -10.8 ± 5.4 | -62.5 ± 4.0 |
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
| percentage of participants | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 | 0 | 84.0 |
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
| percentage of participants | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 | 0 | 52.0 |
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.
| percent change | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percentage Change in Plasma Oxalate From Baseline to Month 6 | -0.3 ± 4.3 | -39.8 ± 2.9 |
Absolute 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.
| μmol/L | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Absolute Change in Plasma Oxalate From Baseline to Month 6 | 1.3 ± 1.1 | -7.5 ± 0.8 |
eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients \>1 year to \<18 years of age at screening. Change from baseline to Month 6 is reported.
| mL/min/1.73m^2 | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Week 2 | -5 ± 9 | -4 ± 9 |
| Month 1 | -6 ± 7 | -2 ± 12 |
| Month 2 | -5 ± 8 | -2 ± 15 |
| Month 3 | -3 ± 6 | 0 ± 11 |
| Month 4 | -4 ± 8 | -4 ± 10 |
| Month 5 | -4 ± 7 | -6 ± 13 |
| Month 6 | 0 ± 6 | -3 ± 11 |
Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.
| mmol/24hr/1.73m^2 | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| At Month 54 | -0.951 ± 0.6148 | -1.086 ± 0.7678 |
| At Month 60 | — | -1.129 ± 0.7581 |
Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline in the extension periods. Only valid urine samples without any non-protocol-related issues were included in the analysis. A negative change from Baseline indicates a favorable outcome.
| percent change | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| At Month 54 | -55.57 ± 11.903 | -53.87 ± 41.919 |
| At Month 60 | — | -53.98 ± 28.476 |
The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA.
| percentage of time | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During Lumasiran Treatment | 89.44 (21.1 to 99.3) | 89.23 (1.7 to 98.6) |
Absolute change in 24-hour urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline to the end of the OLE period at Month 54 and Month 60. A negative change from Baseline indicates a favorable outcome.
| mmol/mmol | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| At Month 54 | -0.145 ± 0.1242 | -0.127 ± 0.1063 |
| At Month 60 | — | -0.138 ± 0.1162 |
eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients \>1 year to \<18 years of age at screening.
| mL/min/1.73m^2 | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| At Month 54 | -12.860 ± 9.5386 | -6.899 ± 12.9302 |
| At Month 60 | — | -2.892 ± 11.6544 |
An AE is any untoward medical occurrence in a clinical investigational participant administered a medicinal product \& which does not necessarily have a causal relationship with this treatment. SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability or incapacity, is a congenital anomaly or birth defect, is an important medical event that may not be immediately life-threatening/result in death/hospitalization but may jeopardize the participant \& may require intervention to prevent one of the other outcomes listed above. All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during 6-month double-blinded period \& participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period.
| Participants | DB Period: Placebo | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|---|
| Adverse Event (AE) | 9 | 12 | 25 |
| Serious Adverse Event (SAE) | 0 | 1 | 5 |
A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone. Lower rates indicate a favorable outcome.
| events per person-year | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| 12-Month Period prior to Informed Consent | 0.54 (0.26 to 1.13) | 3.19 (2.57 to 3.96) |
| 6-Month DB Period | 0.66 (0.25 to 1.76) | 1.09 (0.63 to 1.87) |
Renal ultrasound data were used to grade medullary nephrocalcinosis findings (range: 0 to 3), where a higher grade indicates greater severity. Improving=if both sides improve, or one side improves and the other side has no change; No change=if both sides have no change; Worsening=if both sides worsen, or one side worsens and the other side has no change; Indeterminate=if one side improves and the other side worsens.
| Participants | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|
| Improving | 0 | 3 |
| No Change | 11 | 19 |
| Worsening | 1 | 0 |
| Indeterminate | 0 | 0 |
Collected over DB Period (Placebo): From first dose of study drug (Day 1) up to Month 6; Placebo/Lumasiran: From first dose of lumasiran (Month 6) up to end of study (Month 60); Lumasiran/Lumasiran: From first dose of lumasiran (Day 1) up to end of study (Month 60).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 0/13 (0%) | 0/13 (0%) | 9/13 (69.2%) |
| Placebo/Lumasiran | 0/13 (0%) | 1/13 (7.7%) | 12/13 (92.3%) |
| Lumasiran/Lumasiran | 0/26 (0%) | 5/26 (19.2%) | 25/26 (96.2%) |
| Event | Placebo | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|---|
| Abdominal painGastrointestinal disorders | 0/13 | 1/13 | 1/26 |
| DysuriaRenal and urinary disorders | 0/13 | 1/13 | 0/26 |
| Post procedural infectionInfections and infestations | 0/13 | 0/13 | 1/26 |
| Urinary tract infectionInfections and infestations | 0/13 | 0/13 | 1/26 |
| UrosepsisInfections and infestations | 0/13 | 0/13 | 1/26 |
| Post procedural complicationInjury, poisoning and procedural complications | 0/13 | 0/13 | 1/26 |
| Follicular lymphomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/13 | 0/13 | 1/26 |
| Renal impairmentRenal and urinary disorders | 0/13 | 0/13 | 1/26 |
| Event | Placebo | Placebo/Lumasiran | Lumasiran/Lumasiran |
|---|---|---|---|
| Injection site reactionGeneral disorders | 0/13 | 5/13 | 9/26 |
| Abdominal painGastrointestinal disorders | 0/13 | 1/13 | 8/26 |
| COVID-19Infections and infestations | 0/13 | 4/13 | 4/26 |
| RhinitisInfections and infestations | 2/13 | 3/13 | 2/26 |
| HeadacheNervous system disorders | 3/13 | 2/13 | 5/26 |
| Upper respiratory tract infectionInfections and infestations | 2/13 | 1/13 | 3/26 |
| Abdominal pain lowerGastrointestinal disorders | 0/13 | 2/13 | 1/26 |
| DiarrhoeaGastrointestinal disorders | 0/13 | 2/13 | 1/26 |
| NauseaGastrointestinal disorders | 0/13 | 2/13 | 2/26 |
| VomitingGastrointestinal disorders | 0/13 | 2/13 | 2/26 |
Full Analysis Set (FAS): All randomized participants who received any amount of study drug, was used for all baseline characteristics, except for 24-hour urinary oxalate excretion corrected for body surface area (BSA), which used the Safety Analysis Set (SAS): All participants who received any amount of study drug.
| Age, Customized(Participants) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| 6 to <12 Years | 7 | 9 | 16 |
| 12 to <18 Years | 1 | 5 | 6 |
| 18 to <65 Years | 5 | 12 | 17 |
| Sex: Female, Male(Participants) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Female | 5 | 8 | 13 |
| Male | 8 | 18 | 26 |
| Race/Ethnicity, Customized(Participants) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Asian | 3 | 3 | 6 |
| White | 9 | 21 | 30 |
| Other | 0 | 2 | 2 |
| More than one race | 1 | 0 | 1 |
| Race/Ethnicity, Customized(Participants) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 1 |
| Not Hispanic or Latino | 13 | 25 | 38 |
| Age at Informed Consent(years) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Mean | 17.0 ± 15.19 | 18.7 ± 11.52 | 18.1 ± 12.68 |
| 24-Hour Urinary Oxalate Excretion Corrected for BSA(mmol/24hr/1.73m^2) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Mean | 1.79 ± 0.68 | 1.83 ± 0.60 | 1.82 ± 0.62 |
| Estimated Glomerular Filtration Rate (eGFR)(mL/min/1.73m^2) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Mean | 78.834 ± 29.9841 | 82.967 ± 25.5499 | 81.589 ± 26.7820 |
| 24-hour Urinary Oxalate:Creatinine Ratio(mmol/mmol) | Placebo/Lumasiran | Lumasiran/Lumasiran | Total |
|---|---|---|---|
| Mean | 0.231 ± 0.1306 | 0.209 ± 0.1012 | 0.216 ± 0.1106 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
This study is completed, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Lumasiran
Primary hyperoxaluria type 1 →