Trial · NCT03905694
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
NCT03905694, Phase 3, Completed, Alnylam Pharmaceuticals; results first posted 2021-07-19
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1. Trials, record NCT03905694. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03905694
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
- NCT number
- NCT03905694
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 18 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- Lumasiran
- First posted
- 2019-04-05
- Last updated
- 2025-02-14
- Other registries' IDs
- eudract:2018-004014-17
- Results first posted
- 2021-07-19
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | 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. |
| Secondary | Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60]) | From Month 6 to Month 60 | A negative change from baseline indicates a favorable outcome. |
| Secondary | 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) | Up to 60 months | 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. |
| Secondary | Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline | From Baseline to Month 6 and Month 60 | The absolute change is represented as ratio of millimoles of urinary oxalate to millimoles of urinary creatinine. |
| Secondary | Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age | Up to 60 months | 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. |
| Secondary | Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age | Up to 60 months | 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. |
| Secondary | Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) | From Baseline to Month 6 and 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. |
| Secondary | Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) | From Baseline to Month 6 and Month 60 | The LLOQ was 5.55 μmol/L. A negative change from Baseline indicates a favorable outcome. |
| Secondary | Maximum Observed Plasma Concentration (Cmax) of Lumasiran | 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 | Cmax was the maximum plasma concentration post-dose within the pharmacokinetic (PK) sampling time frame. Higher Cmax generally indicates higher drug exposure. |
| Secondary | Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran | 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 | 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. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Baseline to Month 6 | Efficacy Analysis Set: All participants who received any amount of lumasiran and had at least 1 valid spot urinary oxalate:creatinine ratio value at baseline and at least 1 valid spot urinary oxalate:creatinine ratio value from assessment(s) at Month 3 through Month 6. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60]) | From Month 6 to Month 60 | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) at Month 6 through Month 60. | percent change | MEAN |
| SECONDARY | 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) | Up to 60 months | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | percentage of time | MEDIAN |
| SECONDARY | Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline | From Baseline to Month 6 and Month 60 | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | mmol/mmol | MEAN |
| SECONDARY | Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age | Up to 60 months | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age | Up to 60 months | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | percentage of participants | NUMBER |
| SECONDARY | Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) | From Baseline to Month 6 and Month 60 | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | percent change | MEAN |
| SECONDARY | Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) | From Baseline to Month 6 and Month 60 | Efficacy analysis set included all participants who were treated with Lumasiran and had at least one spot urinary oxalate:creatinine ratio value at baseline and at least one spot urinary oxalate:creatinine ratio value from assessment(s) throughout the study duration. | µmol/L | MEAN |
| SECONDARY | Maximum Observed Plasma Concentration (Cmax) of Lumasiran | 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 | PK analysis set included all participants who had received at least one full dose of Lumasiran and had at least one post-dose blood sample for PK parameters and had evaluable PK data. Number analyzed is the number of participants with data available for analysis at specified timepoints. | nanograms per milliliter (ng/mL) | GEOMETRIC_MEAN |
| SECONDARY | Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran | 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 | PK analysis set included all participants who had received at least one full dose of Lumasiran and had at least one post-dose blood sample for PK parameters and had evaluable PK data. Number analyzed is the number of participants with data available for analysis at specified timepoints. | hours (h) | MEDIAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Lumasiran | -71.97 | Standard Error 2.706 | percent change | |
| Percentage Change in Spot Urinary Oxalate: Creatinine Ratio in the Extension Period (Month 6 to End of Study [Month 60]) | Lumasiran | -74.48 | Standard Error 4.246 | percent change | |
| 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) | Lumasiran | 68.95 | Full Range 18.3 to 98.7 | percentage of time | |
| Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline | Lumasiran | Month 6 | -0.4880 | Standard Error 0.09127 | mmol/mmol |
| Absolute Change in Spot Urinary Oxalate: Creatinine Ratio From Baseline | Lumasiran | Month 60 | -0.5179 | Standard Error 0.09929 | mmol/mmol |
| Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ the ULN for Age | Lumasiran | 100 | percentage of participants | ||
| Percentage of Participants With Spot Urinary Oxalate: Creatinine Ratio Levels ≤ 1.5xULN for Age | Lumasiran | 100 | percentage of participants | ||
| Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) | Lumasiran | Month 6 | -32.06 | Standard Error 6.711 | percent change |
| Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) | Lumasiran | Month 60 | -24.78 | Standard Error 12.814 | percent change |
| Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) | Lumasiran | Month 6 | -5.03 | Standard Error 1.294 | µmol/L |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Analysis Period | STARTED | Lumasiran | 18 | |
| Primary Analysis Period | COMPLETED | Lumasiran | 18 | |
| Primary Analysis Period | NOT COMPLETED | Lumasiran | 0 | |
| Long-term Extension Period | STARTED | Lumasiran | 18 | |
| Long-term Extension Period | COMPLETED | Lumasiran | 18 | |
| Long-term Extension Period | NOT COMPLETED | Lumasiran | 0 |
Source ClinicalTrials.gov
Adverse events100
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Eosinophilia | Blood and lymphatic system disorders | Lumasiran | 1 | 18 | 2 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Lumasiran | 1 | 18 | 1 |
| other | Leukopenia | Blood and lymphatic system disorders | Lumasiran | 1 | 18 | 1 |
| other | Microcytosis | Blood and lymphatic system disorders | Lumasiran | 1 | 18 | 1 |
| other | Factor XII deficiency | Congenital, familial and genetic disorders | Lumasiran | 1 | 18 | 1 |
| other | Ear pain | Ear and labyrinth disorders | Lumasiran | 4 | 18 | 5 |
| other | Conjunctivitis allergic | Eye disorders | Lumasiran | 1 | 18 | 1 |
| other | Myopia | Eye disorders | Lumasiran | 1 | 18 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Lumasiran | 4 | 18 | 4 |
| other | Anal pruritus | Gastrointestinal disorders | Lumasiran | 1 | 18 | 1 |
1 to 10 of 100Source ClinicalTrials.gov
See all 100 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lumasiran | 0 | 18 |
| Total, other adverse events | Lumasiran | 18 | 18 |
| Total, serious adverse events | Lumasiran | 2 | 18 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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