Trial · NCT02706886
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
NCT02706886, Phase 1, Phase 2, Completed, Alnylam Pharmaceuticals; results first posted 2020-01-30
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1. Trials, record NCT02706886. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02706886
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
- NCT number
- NCT02706886
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 52 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- Lumasiran, Placebo
- First posted
- 2016-03-11
- Last updated
- 2020-01-30
- Other registries' IDs
- eudract:2015-004407-23
- Results first posted
- 2020-01-30
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs) | Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days | An AE is any untoward medical occurrence in a clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. |
| Secondary | Maximum Concentration (Cmax) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Time to Cmax (Tmax) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Area Under the Concentration-Time Curve From Time 0 to Time of Last Measurable Concentration (AUC0-last) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Terminal Half-life (t1/2) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Fraction Excreted in Urine in 24 Hours (Fe0-24) of Lumasiran | Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Renal Clearance (CLR) of Lumasiran | Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h | Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. |
| Secondary | Baseline Plasma Glycolate Concentration | Part A (SAD): Baseline, Part B (MAD): Baseline | The pharmacodynamic (PD) outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. |
| Secondary | Percentage Change From Baseline in Plasma Glycolate Concentration | Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85 | The PD outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. |
| Secondary | Baseline Spot Urine Glycolate:Creatinine Ratio in Part A | Part A (SAD): Baseline | The endpoint was only measured in Part A. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs) | Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days | Safety Analysis Set consisted of all healthy participants and patients, who received at least 1 dose of study drug (lumasiran, placebo), grouped according to actual treatment received. Part B participants who crossed over from placebo to the lumasiran arms are included in the placebo arm as well as lumasiran arms. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Concentration (Cmax) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | Pharmacokinetic (PK) Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | ng/mL | MEAN |
| SECONDARY | Time to Cmax (Tmax) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | PK Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | hours | MEDIAN |
| SECONDARY | Area Under the Concentration-Time Curve From Time 0 to Time of Last Measurable Concentration (AUC0-last) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | PK Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | h*ng/mL | MEAN |
| SECONDARY | Terminal Half-life (t1/2) of Lumasiran in Plasma | Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h | PK Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | hours | MEAN |
| SECONDARY | Fraction Excreted in Urine in 24 Hours (Fe0-24) of Lumasiran | Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h | PK Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | percentage fractional excretion | MEAN |
| SECONDARY | Renal Clearance (CLR) of Lumasiran | Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h | PK Analysis Set consisted of all healthy participants and patients who received at least 1 dose of lumasiran and had at least 1 postdose sample for PK parameters and who had evaluable PK data. | L/h | MEAN |
| SECONDARY | Baseline Plasma Glycolate Concentration | Part A (SAD): Baseline, Part B (MAD): Baseline | PD Analysis Set consisted of all participants who received at least 1 dose of study drug (lumasiran, placebo) and had at least 1 postdose blood sample available that was evaluable for PD assessments. Due to an issue with plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. | umol/L | MEAN |
| SECONDARY | Percentage Change From Baseline in Plasma Glycolate Concentration | Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85 | PD Analysis Set consisted of all participants who received at least 1 dose of study drug (lumasiran, placebo) and had at least 1 postdose blood sample available that was evaluable for PD assessments. Due to an issue with plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. | percentage change from baseline | MEAN |
| SECONDARY | Baseline Spot Urine Glycolate:Creatinine Ratio in Part A | Part A (SAD): Baseline | PD Analysis Set for Part A consisted of all healthy participants who received at least 1 dose of study drug (lumasiran, placebo) and had at least 1 postdose blood and/or urine sample available that was evaluable for PD assessments. | mg/g | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group198
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs) | Part A: SAD: Placebo | 5 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part A: SAD: Lumasiran 0.3 mg/kg | 6 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part A: SAD: Lumasiran 1.0 mg/kg | 2 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part A: SAD: Lumasiran 3.0 mg/kg | 6 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part A: SAD: Lumasiran 6.0 mg/kg | 6 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part B: MAD: Placebo | 2 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part B: MAD: Lumasiran 1.0 mg/kg qM | 8 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part B: MAD: Lumasiran 3.0 mg/kg qM | 7 | Participants | ||
| Number of Participants With Adverse Events (AEs) | Part B: MAD: Lumasiran 3.0 mg/kg q3M | 4 | Participants | ||
| Maximum Concentration (Cmax) of Lumasiran in Plasma | Part A: SAD: Lumasiran 0.3 mg/kg | Day 1 | 39.7940 | Standard Deviation 8.58882 | ng/mL |
1 to 10 of 198Source ClinicalTrials.gov
See all 198 results by groupParticipant flow81
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A: SAD Period | STARTED | Part A: SAD: Placebo | 8 | |
| Part A: SAD Period | STARTED | Part A: SAD: Lumasiran 0.3 mg/kg | 6 | |
| Part A: SAD Period | STARTED | Part A: SAD: Lumasiran 1.0 mg/kg | 6 | |
| Part A: SAD Period | STARTED | Part A: SAD: Lumasiran 3.0 mg/kg | 6 | |
| Part A: SAD Period | STARTED | Part A: SAD: Lumasiran 6.0 mg/kg | 6 | |
| Part A: SAD Period | STARTED | Part B: MAD: Placebo | 0 | |
| Part A: SAD Period | STARTED | Part B: MAD: Lumasiran 1.0 mg/kg qM | 0 | |
| Part A: SAD Period | STARTED | Part B: MAD: Lumasiran 3.0 mg/kg qM | 0 | |
| Part A: SAD Period | STARTED | Part B: MAD: Lumasiran 3.0 mg/kg q3M | 0 | |
| Part A: SAD Period | COMPLETED | Part A: SAD: Placebo | 8 |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 participant flowAdverse events756
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 | Part A: SAD: Placebo | 0 | 8 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part A: SAD: Lumasiran 0.3 mg/kg | 1 | 6 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part A: SAD: Lumasiran 1.0 mg/kg | 0 | 6 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part A: SAD: Lumasiran 3.0 mg/kg | 0 | 6 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part A: SAD: Lumasiran 6.0 mg/kg | 0 | 6 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part B: MAD: Placebo | 0 | 3 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part B: MAD: Lumasiran 1.0 mg/kg qM | 0 | 8 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part B: MAD: Lumasiran 3.0 mg/kg qM | 0 | 8 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Part B: MAD: Lumasiran 3.0 mg/kg q3M | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Part A: SAD: Placebo | 0 | 8 |
1 to 10 of 756Source ClinicalTrials.gov
See all 756 adverse eventsAdverse event totals27
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: SAD: Placebo | 0 | 8 |
| Total, all-cause mortality | Part A: SAD: Lumasiran 0.3 mg/kg | 0 | 6 |
| Total, all-cause mortality | Part A: SAD: Lumasiran 1.0 mg/kg | 0 | 6 |
| Total, all-cause mortality | Part A: SAD: Lumasiran 3.0 mg/kg | 0 | 6 |
| Total, all-cause mortality | Part A: SAD: Lumasiran 6.0 mg/kg | 0 | 6 |
| Total, all-cause mortality | Part B: MAD: Placebo | 0 | 3 |
| Total, all-cause mortality | Part B: MAD: Lumasiran 1.0 mg/kg qM | 0 | 8 |
| Total, all-cause mortality | Part B: MAD: Lumasiran 3.0 mg/kg qM | 0 | 8 |
| Total, all-cause mortality | Part B: MAD: Lumasiran 3.0 mg/kg q3M | 0 | 4 |
| Total, other adverse events | Part A: SAD: Placebo | 5 | 8 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 adverse event totalsPapers2
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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Read this trial from software.
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GET https://conductscience.org/api/trials/v1/NCT02706886/results?page=2