A Phase 3 interventional study of Lumasiran in Primary Hyperoxaluria and Primary Hyperoxaluria Type 1 (PH1), sponsored by Alnylam Pharmaceuticals. Completed at 9 sites in 5 countries. Open to participants aged 0 Years to 5 Years. Per ClinicalTrials.gov, last updated 2025-02-14.
Sponsored by Alnylam Pharmaceuticals · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed 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 18 is below 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.
Compared within Primary hyperoxaluria type 1, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Hyperoxaluria, Primary. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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.
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.
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.
Lumasiran will be administered by subcutaneous (SC) injection.
Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Also known as: ALN-GO1
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
Time frame: Baseline to Month 6
Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60])
A negative change from baseline indicates a favorable outcome.
Time frame: From Month 6 to Month 60
Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age)
Percentage of time that spot urinary oxalate: creatinine (UOx:Cr) ratio level is at or below ≤ 1.5xULN is calculated as (cumulative months at or below near normalization threshold divided by cumulative months of assessments)\*100. Cumulative months in near-normalization is defined as the summation across all intervals that met the near-normal threshold and cumulative months of valid assessments is defined as the summation across all valid post-baseline collections. ULN levels of spot urinary oxalate to creatinine ratio where urine oxalate levels were analyzed using enzymatic assay. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Time frame: Up to 60 months
Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline
The absolute change is represented as ratio of millimoles of urinary oxalate to millimoles of urinary creatinine.
Time frame: From Baseline to Month 6 and Month 60
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ the ULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Time frame: Up to 60 months
Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ 1.5xULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
Time frame: Up to 60 months
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
The lower limit of quantification (LLOQ) was 5.55 micromoles per liter (μmol/L). A negative change from baseline indicates a favorable outcome.
Time frame: From Baseline to Month 6 and Month 60
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
The LLOQ was 5.55 μmol/L. A negative change from Baseline indicates a favorable outcome.
Time frame: From Baseline to Month 6 and Month 60
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Cmax was the maximum plasma concentration post-dose within the pharmacokinetic (PK) sampling time frame. Higher Cmax generally indicates higher drug exposure.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Tmax was estimated by calculating the time required to reach the maximum plasma concentration (Cmax) after the drug administration. Lower Tmax generally indicates faster drug absorption from the administration site.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Elimination Half-life (t1/2beta) of Lumasiran
Elimination half-life was estimated from the terminal phase of the plasma concentration-time profile post-dose. Shorter half-life generally indicates rapid drug elimination from the body.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to 24 Hours (AUC0-24) of Lumasiran
AUC0-24 was the total drug exposure calculated as the area under the plasma concentration-time curve from the time of dosing (t = 0) to 24 hours.
Time frame: 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Last Quantifiable Concentration (AUC0-last) of Lumasiran
AUC0-last was the total drug exposure calculated as the area under the plasma concentration-time curve from time 0 to the time of the last measurable (quantifiable) concentration (C_last).
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Area Under the Concentration-time Curve From 0 to Infinity (AUC0-infinity) of Lumasiran
AUC0-infinity was the total drug exposure estimated as the area under the plasma concentration-time curve from time 0 extrapolated to infinity.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Clearance (CL/F) of Lumasiran
Apparent clearance was calculated by dividing the area under the plasma concentration-time curve from zero infinity by the dose administered. A higher clearance generally indicates faster elimination from the body.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Apparent Volume of Distribution (V/F) of Lumasiran
Apparent Volume of Distribution generally indicates the extent of drug distribution in the body.
Time frame: 2 hours, 4 hours, 8 hours, 12 hours, and 24 hours post-dose on Day 1, Month 6, Month 12, Month 18 and Month 24
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
eGFR \[in milliliters per minute per 1.73 meters square (mL/min/1.73m\^2)\] was calculated from serum creatinine (SCr) based on the Schwartz Bedside Formula for participants ≥12 months of age at the time of assessment.
Time frame: From Baseline to Month 6 and Month 60
Number of Participants With Adverse Events (AEs)
An Adverse Event (AE) is any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
Time frame: Up to 60 months
Participants took part in the study at investigative sites in France, Germany, Israel, the United Kingdom, and the United States from 22 April 2019 to 26 July 2024.
| Milestone | Lumasiran |
|---|---|
| Started | 18 |
| Completed | 18 |
| Not completed | 0 |
| Milestone | Lumasiran |
|---|---|
| Started | 18 |
| Completed | 18 |
| Not completed | 0 |
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.
| percent change | Lumasiran |
|---|---|
| Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | -71.97 ± 2.706 |
A negative change from baseline indicates a favorable outcome.
| percent change | Lumasiran |
|---|---|
| Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60]) | -74.48 ± 4.246 |
Percentage of time that spot urinary oxalate: creatinine (UOx:Cr) ratio level is at or below ≤ 1.5xULN is calculated as (cumulative months at or below near normalization threshold divided by cumulative months of assessments)\*100. Cumulative months in near-normalization is defined as the summation across all intervals that met the near-normal threshold and cumulative months of valid assessments is defined as the summation across all valid post-baseline collections. ULN levels of spot urinary oxalate to creatinine ratio where urine oxalate levels were analyzed using enzymatic assay. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
| percentage of time | Lumasiran |
|---|---|
| Percentage of Time That Spot Urinary Oxalate: Creatinine Ratio is at or Below the Near-normalization Threshold (≤1.5 × Upper Limit of Normal (ULN) for Age) | 68.95 (18.3 to 98.7) |
The absolute change is represented as ratio of millimoles of urinary oxalate to millimoles of urinary creatinine.
| mmol/mmol | Lumasiran |
|---|---|
| Month 6 | -0.4880 ± 0.09127 |
| Month 60 | -0.5179 ± 0.09929 |
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ the ULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
| percentage of participants | Lumasiran |
|---|---|
| Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age | 100 |
The percentage of participants meeting the criteria (UOx:Cr ratio ≤ 1.5xULN for age) at least at one post-baseline visit were reported for this outcome measure. The age-dependent reference ULN levels of spot urinary oxalate to creatinine ratio are as follows: 1-1.5 years=0.158; 1.5-2 years=0.138; 2-2.5 years=0.124; 2.5-3 years=0.116; 3-3.5 years=0.102; 3.5-4 years=0.094; 4-4.5 years=0.088; 4.5-5 years=0.082; 5-5.5 years=0.077; 5.5-6 years=0.073.
| percentage of participants | Lumasiran |
|---|---|
| Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age | 100 |
The lower limit of quantification (LLOQ) was 5.55 micromoles per liter (μmol/L). A negative change from baseline indicates a favorable outcome.
| percent change | Lumasiran |
|---|---|
| Month 6 | -32.06 ± 6.711 |
| Month 60 | -24.78 ± 12.814 |
The LLOQ was 5.55 μmol/L. A negative change from Baseline indicates a favorable outcome.
| µmol/L | Lumasiran |
|---|---|
| Month 6 | -5.03 ± 1.294 |
| Month 60 | -5.03 ± 1.859 |
Cmax was the maximum plasma concentration post-dose within the pharmacokinetic (PK) sampling time frame. Higher Cmax generally indicates higher drug exposure.
| nanograms per milliliter (ng/mL) | Lumasiran |
|---|---|
| Day 1 | 886 ± 58.9 |
| Month 6 | 869 ± 84.0 |
| Month 12 | 1180 ± 83.5 |
| Month 18 | 878 ± 94.0 |
| Month 24 | 790 ± 91.9 |
Tmax was estimated by calculating the time required to reach the maximum plasma concentration (Cmax) after the drug administration. Lower Tmax generally indicates faster drug absorption from the administration site.
| hours (h) | Lumasiran |
|---|---|
| Day 1 | 3.74 (1.87 to 8.1) |
| Month 6 | 4 (1.75 to 10.1) |
| Month 12 | 4 (1.75 to 7.25) |
| Month 18 | 3.64 (1.98 to 10) |
| Month 24 | 3.73 (1.67 to 10.3) |
Elimination half-life was estimated from the terminal phase of the plasma concentration-time profile post-dose. Shorter half-life generally indicates rapid drug elimination from the body.
| hours (h) | Lumasiran |
|---|---|
| Day 1 | 5.46 (1.42 to 10.3) |
| Month 6 | 5.96 (3.04 to 9) |
| Month 12 | 3.52 (1.56 to 6.64) |
| Month 18 | 3.74 (1.62 to 13.9) |
| Month 24 | 4.99 (2.69 to 10.8) |
AUC0-24 was the total drug exposure calculated as the area under the plasma concentration-time curve from the time of dosing (t = 0) to 24 hours.
| hours*nanograms per milliliter (h*ng/mL) | Lumasiran |
|---|---|
| Day 1 | 8050 ± 37.7 |
| Month 6 | 8450 ± 41.1 |
| Month 12 | 10800 ± 39.3 |
| Month 18 | 8420 ± 57.2 |
| Month 24 | 7420 ± 61.6 |
AUC0-last was the total drug exposure calculated as the area under the plasma concentration-time curve from time 0 to the time of the last measurable (quantifiable) concentration (C_last).
| h*ng/mL | Lumasiran |
|---|---|
| Day 1 | 7870 ± 33.4 |
| Month 6 | 6620 ± 95.5 |
| Month 12 | 8480 ± 76.1 |
| Month 18 | 5770 ± 155.0 |
| Month 24 | 7100 ± 80.0 |
AUC0-infinity was the total drug exposure estimated as the area under the plasma concentration-time curve from time 0 extrapolated to infinity.
| h*ng/mL | Lumasiran |
|---|---|
| Day 1 | 9180 ± 31.7 |
| Month 6 | 8840 ± 40.9 |
| Month 12 | 11900 ± 29.2 |
| Month 18 | 11600 ± 48.6 |
| Month 24 | 9610 ± 48.8 |
Apparent clearance was calculated by dividing the area under the plasma concentration-time curve from zero infinity by the dose administered. A higher clearance generally indicates faster elimination from the body.
| liters per hour (L/h) | Lumasiran |
|---|---|
| Day 1 | 8.62 ± 24.0 |
| Month 6 | 10.2 ± 32.3 |
| Month 12 | 7.06 ± 28.3 |
| Month 18 | 6.87 ± 30.6 |
| Month 24 | 8.22 ± 37.7 |
Apparent Volume of Distribution generally indicates the extent of drug distribution in the body.
| Liters (L) | Lumasiran |
|---|---|
| Day 1 | 57.2 ± 69.8 |
| Month 6 | 81 ± 71.1 |
| Month 12 | 36 ± 75.6 |
| Month 18 | 43.1 ± 107.1 |
| Month 24 | 57.6 ± 70.6 |
eGFR \[in milliliters per minute per 1.73 meters square (mL/min/1.73m\^2)\] was calculated from serum creatinine (SCr) based on the Schwartz Bedside Formula for participants ≥12 months of age at the time of assessment.
| mL/min/1.73m^2 | Lumasiran |
|---|---|
| Month 6 | -0.263 ± 3.8461 |
| Month 60 | -4.525 ± 4.8873 |
An Adverse Event (AE) is any untoward medical occurrence in a participant or clinical investigational participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
| Participants | Lumasiran |
|---|---|
| Number of Participants With Adverse Events (AEs) | 18 |
Collected over Up to 60 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Lumasiran | 0/18 (0%) | 2/18 (11.1%) | 18/18 (100%) |
| Event | Lumasiran |
|---|---|
| Viral infectionInfections and infestations | 1/18 |
| Ear haemorrhageEar and labyrinth disorders | 1/18 |
| Event | Lumasiran |
|---|---|
| PyrexiaGeneral disorders | 8/18 |
| VomitingGastrointestinal disorders | 6/18 |
| GastroenteritisInfections and infestations | 5/18 |
| RhinitisInfections and infestations | 5/18 |
| Upper respiratory tract infectionInfections and infestations | 5/18 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 5/18 |
| Ear painEar and labyrinth disorders | 4/18 |
| Abdominal painGastrointestinal disorders | 4/18 |
| InfluenzaInfections and infestations | 4/18 |
| NasopharyngitisInfections and infestations | 4/18 |
Safety analysis set included all participants who had received at least one dose of Lumasiran during study.
| Age, Continuous(months) | Lumasiran |
|---|---|
| Mean | 43.7 ± 21.30 |
| Sex: Female, Male(Participants) | Lumasiran |
|---|---|
| Female | 10 |
| Male | 8 |
| Race/Ethnicity, Customized(Participants) | Lumasiran |
|---|---|
| White | 16 |
| Other | 2 |
| Race/Ethnicity, Customized(Participants) | Lumasiran |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 16 |
| Spot Urinary Oxalate:Creatinine Ratio(mmol/mmol) | Lumasiran |
|---|---|
| Mean | 0.6306 ± 0.42636 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the United States (US) and/or the European Union (EU). Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
Ran from Apr 2019 to primary completion in Jun 2020, 14 months.
Results were first posted in Jul 2021, 1 year after primary completion.
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This study is completed, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Lumasiran
Primary hyperoxaluria type 1 →