Trial · NCT03681184
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
NCT03681184, Phase 3, Completed, Alnylam Pharmaceuticals; results first posted 2021-01-19
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1. Trials, record NCT03681184. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03681184
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
- NCT number
- NCT03681184
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 39 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- Placebo, Lumasiran
- First posted
- 2018-09-21
- Last updated
- 2024-08-12
- Other registries' IDs
- eudract:2018-001981-40
- Results first posted
- 2021-01-19
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | 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. |
| Secondary | Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 | 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. |
| Secondary | Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | 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. |
| Secondary | Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 | Month 6 | The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA. |
| Secondary | Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 | Month 6 | The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m\^2 for 24-hour urinary oxalate excretion corrected for BSA. |
| Secondary | Percentage Change in Plasma Oxalate From Baseline to Month 6 | 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. |
| Secondary | Absolute Change in Plasma Oxalate From Baseline to Month 6 | 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. |
| Secondary | Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6 | Baseline, Week 2, 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. |
| Secondary | Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period | From Baseline to Month 54 and Month 60 | 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. |
| Secondary | Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period | From Baseline to Month 54 and Month 60 | 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. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Baseline to Month 6 | FAS: All randomized participants who received any amount of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 | Baseline to Month 6 | FAS: All randomized participants who received any amount of study drug. | mmol/24hr/1.73m^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Baseline to Month 6 | FAS: All randomized participants who received any amount of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 | Month 6 | Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 | Month 6 | Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available. | percentage of participants | NUMBER |
| SECONDARY | Percentage Change in Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | Plasma Oxalate Analysis Set: all participants who received any amount of study drug and had baseline plasma oxalate level ≥1.5×lower limit of quantitation (LLOQ). LLOQ is 5.55 μmol/L. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | Plasma Oxalate Analysis Set: all participants who received any amount of study drug and had baseline plasma oxalate level ≥1.5×LLOQ. LLOQ is 5.55 μmol/L. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6 | Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6 | Participants from the FAS (all randomized participants who received any amount of study drug) for whom data are available. | mL/min/1.73m^2 | MEAN |
| SECONDARY | Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period | From Baseline to Month 54 and Month 60 | All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during the 6-month double-blinded period and participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period. Overall number analyzed is number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | mmol/24hr/1.73m^2 | MEAN |
| SECONDARY | Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period | From Baseline to Month 54 and Month 60 | All Lumasiran Treated Set: All participants who received any amount of lumasiran including participants who took lumasiran during the 6-month double-blinded period and participants who initially took placebo during the 6-month double-blinded period and then switched to lumasiran during the extension period. Overall number analyzed is number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | percent change | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group60
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Placebo/Lumasiran | -11.8 | Standard Error 3.8 | percent change | |
| Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Lumasiran/Lumasiran | -65.4 | Standard Error 2.9 | percent change | |
| Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 | Placebo/Lumasiran | -0.27 | Standard Error 0.08 | mmol/24hr/1.73m^2 | |
| Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 | Lumasiran/Lumasiran | -1.24 | Standard Error 0.06 | mmol/24hr/1.73m^2 | |
| Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Placebo/Lumasiran | -10.8 | Standard Error 5.4 | percent change | |
| Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Lumasiran/Lumasiran | -62.5 | Standard Error 4.0 | percent change | |
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 | Placebo/Lumasiran | 0 | percentage of participants | ||
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 | Lumasiran/Lumasiran | 84.0 | percentage of participants | ||
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 | Placebo/Lumasiran | 0 | percentage of participants | ||
| Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 | Lumasiran/Lumasiran | 52.0 | percentage of participants |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| 6-Month Double-Blind Period | STARTED | Placebo/Lumasiran | 13 | |
| 6-Month Double-Blind Period | STARTED | Lumasiran/Lumasiran | 26 | |
| 6-Month Double-Blind Period | COMPLETED | Placebo/Lumasiran | 13 | |
| 6-Month Double-Blind Period | COMPLETED | Lumasiran/Lumasiran | 25 | |
| 6-Month Double-Blind Period | NOT COMPLETED | Placebo/Lumasiran | 0 | |
| 6-Month Double-Blind Period | NOT COMPLETED | Lumasiran/Lumasiran | 1 | |
| 3-Month Blinded Extension Period | STARTED | Placebo/Lumasiran | 13 | |
| 3-Month Blinded Extension Period | STARTED | Lumasiran/Lumasiran | 24 | |
| 3-Month Blinded Extension Period | COMPLETED | Placebo/Lumasiran | 13 | |
| 3-Month Blinded Extension Period | COMPLETED | Lumasiran/Lumasiran | 24 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events489
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 | Abdominal discomfort | Gastrointestinal disorders | Placebo | 1 | 13 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Placebo/Lumasiran | 0 | 13 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Lumasiran/Lumasiran | 1 | 26 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 0 | 13 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo/Lumasiran | 1 | 13 | |
| other | Abdominal pain | Gastrointestinal disorders | Lumasiran/Lumasiran | 8 | 26 | |
| other | Injection site erythema | General disorders | Placebo | 0 | 13 | |
| other | Injection site erythema | General disorders | Placebo/Lumasiran | 1 | 13 | |
| other | Injection site erythema | General disorders | Lumasiran/Lumasiran | 3 | 26 | |
| other | Injection site reaction | General disorders | Placebo | 0 | 13 |
1 to 10 of 489Source ClinicalTrials.gov
See all 489 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 13 |
| Total, all-cause mortality | Placebo/Lumasiran | 0 | 13 |
| Total, all-cause mortality | Lumasiran/Lumasiran | 0 | 26 |
| Total, other adverse events | Placebo | 9 | 13 |
| Total, other adverse events | Placebo/Lumasiran | 12 | 13 |
| Total, other adverse events | Lumasiran/Lumasiran | 25 | 26 |
| Total, serious adverse events | Placebo | 0 | 13 |
| Total, serious adverse events | Placebo/Lumasiran | 1 | 13 |
| Total, serious adverse events | Lumasiran/Lumasiran | 5 | 26 |
Source ClinicalTrials.gov
Papers2
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03681184
GET https://conductscience.org/api/trials/v1/NCT03681184/results?page=2