Trial · NCT04152200
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
NCT04152200, Phase 3, Completed, Alnylam Pharmaceuticals; results first posted 2022-07-12
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1. Trials, record NCT04152200. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04152200
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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 Patients With Advanced Primary Hyperoxaluria Type 1
- NCT number
- NCT04152200
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 21 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Primary Hyperoxaluria Type 1, Primary Hyperoxaluria
- Interventions
- Lumasiran
- First posted
- 2019-11-05
- Last updated
- 2025-07-18
- Other registries' IDs
- eudract:2019-001346-17
- Results first posted
- 2022-07-12
Source ClinicalTrials.gov
Registered outcomes31
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cohort A: Percent 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. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran. |
| Primary | Cohort B: Percent Change in Pre-dialysis 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. For Cohort B, the baseline is defined as the mean of the last four pre-dialysis plasma oxalate samples collected prior to the first dose of lumasiran. In Cohort B, only pre-dialysis samples are utilized. |
| Secondary | Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6 | Baseline to Month 6 | Percent change in plasma oxalate AUC(0-24) was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean value of all valid AUC (μmol/L/24h) profiles being computed prior to the first dose of lumasiran. |
| Secondary | Absolute Change in Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | Absolute change in plasma oxalate was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. For Cohort A, the baseline was defined as the mean of all plasma oxalate level values collected prior to the first dose of lumasiran; for Cohort B, the baseline was defined as the mean of the last 4 pre-dialysis plasma oxalate level values collected prior to the first dose of lumasiran. In Cohort B patients, the plasma oxalate baseline was similarly defined except that the values obtained from the plasma oxalate profile assessment visits only included the pre-dialysis sample collected per visit. |
| Secondary | Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (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. Baseline is defined as the median of all valid 24-hr urine assessments collected prior to the first dose date/time of lumasiran without any non-protocol-related sample issues. |
| Secondary | Cohort A: 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. Baseline is defined as the mean of all assessments prior to the first dose date/time of lumasiran. |
| Secondary | Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Baseline to Month 6 | Absolute change in spot urinary oxalate:creatinine ratio was estimated by an average absolute change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran. |
| Secondary | Cohort A: Percent 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. Baseline was defined as the mean of all assessments prior to the first dose date/time of lumasiran. |
| Secondary | Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent | Baseline to Month 6 | Peds-QL is a modular approach to measuring quality of life (QOL) in healthy children and adolescents and those with acute and chronic health conditions. The PedsQL Generic Core Scales contain 23 items designed to measure core domains of health (physical, emotional, and social functioning) and role (school functioning). Scores are summarized as Total Scale Score, Physical Health Summary Score, and Psychosocial Health Summary Score. The Total Scale Score will be reported for this study. The PedsQL will be completed by patients (or caregivers, as appropriate) who are ≥2 to \<18 years of age at the time of consent. Total score range: 0 - 100, with higher scores indicating better health-related quality of life (HRQoL). Baseline is defined as last non-missing value collected prior to the first dose of lumasiran. |
| Secondary | Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent | Baseline to Month 6 | The KDQOL questionnaire is used to assess 3 core domains of kidney disease including: burden, symptoms/problems, and effects of kidney disease on daily life. The KDQOL subscales (Burden of Kidney Disease, Effect of Kidney Disease on Daily Life, and Symptoms and Problems of Kidney Disease), and the accompanying Short Form-12 (SF-12) Physical Component Summary and Mental Component Summary will be assessed for this study. These will be completed by patients who are ≥18 years of age at the time of consent. Score range (per domain): 0 - 100; higher scores indicate better HRQoL. Baseline is defined as last non-missing value collected prior to the first dose of lumasiran. |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 registered outcomesOutcomes31
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | Full Analysis Set (FAS): All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | percent change | LEAST_SQUARES_MEAN |
| PRIMARY | Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Plasma Oxalate From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran (with data available for analysis) and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. No analyses were performed for patients in Cohort B that were anuric (ie, patients able to continue to produce urine ≥100 mL per day). | mmol/24hr/1.73m^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Cohort A: 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 patients who received any amount of lumasiran (with data available for analysis) and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. No analyses were performed for patients in Cohort B that were anuric (ie, patients able to continue to produce urine ≥100 mL per day). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | ratio | LEAST_SQUARES_MEAN |
| SECONDARY | Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Baseline to Month 6 | FAS: All patients who received any amount of lumasiran and had at least 1 evaluable plasma oxalate value (pre-dialysis in Cohort B) at baseline and at least 1 evaluable plasma oxalate value from assessment(s) at Month 3 through Month 6. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent | Baseline to Month 6 | Participants from the FAS who were ≥2 to \<18 years of age at the time of consent with data available for analysis. | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life As Assessed by Kidney Disease and Quality of Life (KDQOL) Scores From Baseline to Month 6 in Patients ≥18 Years of Age at Time of Informed Consent | Baseline to Month 6 | Participants from the FAS who were ≥18 years of age at the time of consent. | score on a scale | MEAN |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 outcomesResults by group57
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6 | Cohort A: Lumasiran | -33.33 | Standard Error 17.63 | percent change | |
| Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6 | Cohort B: Lumasiran | -42.43 | Standard Error 3.95 | percent change | |
| Cohort B: Percent Change in Plasma Oxalate Area Under the Curve From 0-24 Hours [AUC(0-24)] Between Dialysis Sessions From Baseline to Month 6 | Cohort B: Lumasiran | -41.4 | Standard Error 4.4 | percent change | |
| Absolute Change in Plasma Oxalate From Baseline to Month 6 | Cohort A: Lumasiran | -35.28 | Standard Error 7.40 | μmol/L | |
| Absolute Change in Plasma Oxalate From Baseline to Month 6 | Cohort B: Lumasiran | -48.33 | Standard Error 3.63 | μmol/L | |
| Cohort A: Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Cohort A: Lumasiran | -0.533 | Standard Error 0.111 | mmol/24hr/1.73m^2 | |
| Cohort A: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 | Cohort A: Lumasiran | -10.557 | Standard Error 6.811 | percent change | |
| Cohort A: Absolute Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Cohort A: Lumasiran | -0.1879 | Standard Error 0.0160 | ratio | |
| Cohort A: Percent Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | Cohort A: Lumasiran | -39.5136 | Standard Error 9.4294 | percent change | |
| Change in Quality of Life As Assessed by Pediatric Quality of Life Inventory (PedsQL) Total Score From Baseline to Month 6 in Patients >=2 to 18 Years of Age at Time of Informed Consent | Cohort A: Lumasiran | -23.91 | Standard Deviation 9.22 | score on a scale |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Analysis Period | STARTED | Cohort A: Lumasiran | 6 | |
| Primary Analysis Period | STARTED | Cohort B: Lumasiran | 15 | |
| Primary Analysis Period | COMPLETED | Cohort A: Lumasiran | 6 | |
| Primary Analysis Period | COMPLETED | Cohort B: Lumasiran | 15 | |
| Primary Analysis Period | NOT COMPLETED | Cohort A: Lumasiran | 0 | |
| Primary Analysis Period | NOT COMPLETED | Cohort B: Lumasiran | 0 | |
| Long-term Extension Period | STARTED | Cohort A: Lumasiran | 6 | |
| Long-term Extension Period | STARTED | Cohort B: Lumasiran | 15 | |
| Long-term Extension Period | COMPLETED | Cohort A: Lumasiran | 0 | |
| Long-term Extension Period | COMPLETED | Cohort B: Lumasiran | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events150
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 | Blood loss anaemia | Blood and lymphatic system disorders | Cohort A: Lumasiran | 0 | 6 | |
| other | Blood loss anaemia | Blood and lymphatic system disorders | Cohort B: Lumasiran | 1 | 15 | |
| other | Hyperparathyroidism tertiary | Endocrine disorders | Cohort A: Lumasiran | 0 | 6 | |
| other | Hyperparathyroidism tertiary | Endocrine disorders | Cohort B: Lumasiran | 1 | 15 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Cohort A: Lumasiran | 0 | 6 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Cohort B: Lumasiran | 1 | 15 | |
| other | Constipation | Gastrointestinal disorders | Cohort A: Lumasiran | 1 | 6 | |
| other | Constipation | Gastrointestinal disorders | Cohort B: Lumasiran | 1 | 15 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort A: Lumasiran | 1 | 6 | |
| other | Diarrhoea | Gastrointestinal disorders | Cohort B: Lumasiran | 3 | 15 |
1 to 10 of 150Source ClinicalTrials.gov
See all 150 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort A: Lumasiran | 0 | 6 |
| Total, all-cause mortality | Cohort B: Lumasiran | 0 | 15 |
| Total, other adverse events | Cohort A: Lumasiran | 5 | 6 |
| Total, other adverse events | Cohort B: Lumasiran | 13 | 15 |
| Total, serious adverse events | Cohort A: Lumasiran | 1 | 6 |
| Total, serious adverse events | Cohort B: Lumasiran | 8 | 15 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort A: Lumasiran vs Cohort B: Lumasiran: deaths from any cause | Trial comparisons |
| Cohort A: Lumasiran vs Cohort B: Lumasiran: serious adverse events | Trial comparisons |
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/NCT04152200
GET https://conductscience.org/api/trials/v1/NCT04152200/registered?page=2