Trial · NCT03350451
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
NCT03350451, Phase 2, Completed, Alnylam Pharmaceuticals; results first posted 2024-04-25
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1. Trials, record NCT03350451. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03350451
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
- NCT number
- NCT03350451
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA, AGT
- Interventions
- Lumasiran
- First posted
- 2017-11-22
- Last updated
- 2024-04-25
- Other registries' IDs
- eudract:2016-003134-24
- Results first posted
- 2024-04-25
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With at Least One Adverse Event (AE) | Baseline (Day -1) up to 54 months | AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug. |
| Secondary | Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months | Baseline (Day -1) up to 54 months | Oxalate produced by the liver is the key toxic metabolite that drives disease pathology in participants with primary hyperoxaluria type 1 (PH1). The risk of disease complications increase continuously as oxalate levels increase. 24-hour urinary oxalate (millimole \[mmol\]/ 24 hour \[h\]/1.73 meters squared \[m\^2\]) corrected for BSA at each visit per participant was calculated as follows: \[Urine oxalate concentration (micromole per liter \[umol/L\])/1000 (umol/mmol)\]\*\[24hour urine volume (mL)/1000 (mL/L)\]\* \[24 hours/actual collection hours\]\*1.73/(BSA). Baseline was the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicated a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Overall number of participants analyzed are the number of participants with data available for analysis. |
| Secondary | Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months | Baseline (Day -1) up to 54 months | Baseline is the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicates a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. |
| Secondary | Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months | Baseline (Day -1) up to 54 months | Baseline was defined as the last measurement prior to the first dose of lumasiran in the ALN-GO1-001 study. eGFR was calculated based on the Modification of Diet in Renal Disease (MDRD) formula for participants \>=18 years of age at enrollment and the Schwartz Bedside formula for participants \<18 years of age at enrollment. eGFR based on MDRD formula was calculated as follows: eGFR (mL/min/1.73 m\^2) = 175 × (serum creatinine {SCr} \[μmol/deciliter(dL)\]/88.4)-1.154 × (age)-0.203 × (0.742, if female), or × (1.212, if African American) and based on Schwartz formula: eGFR (mL/min/1.73m2) = (36.2 × height \[cm\])/ SCr (μmol /dL). Safety analysis set included all participants who received any amount of study drug. |
Source ClinicalTrials.gov
Outcomes4
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 at Least One Adverse Event (AE) | Baseline (Day -1) up to 54 months | The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months | Baseline (Day -1) up to 54 months | The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. | mmol/24hr/1.73m^2 | MEAN |
| SECONDARY | Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months | Baseline (Day -1) up to 54 months | The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. | mmol/mmol | MEAN |
| SECONDARY | Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months | Baseline (Day -1) up to 54 months | The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. | mL/min/1.73m^2 | MEAN |
Source ClinicalTrials.gov
Results by group8
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 at Least One Adverse Event (AE) | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | Participants | ||
| Number of Participants With at Least One Adverse Event (AE) | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | Participants | ||
| Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | -1.425 | Standard Error 0.2188 | mmol/24hr/1.73m^2 | |
| Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months | Lumasiran (ALN-GO1): 3.0 mg/kg QM | -2.126 | Standard Error 0.5528 | mmol/24hr/1.73m^2 | |
| Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | -0.2175 | Standard Deviation 0.03880 | mmol/mmol | |
| Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months | Lumasiran (ALN-GO1): 3.0 mg/kg QM | -0.2480 | Standard Deviation 0.04047 | mmol/mmol | |
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 0.674 | Standard Error 3.7970 | mL/min/1.73m^2 | |
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months | Lumasiran (ALN-GO1): 3.0 mg/kg QM | -12.026 | Standard Error 3.6369 | mL/min/1.73m^2 |
Source ClinicalTrials.gov
Participant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | |
| Overall Study | STARTED | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | |
| Overall Study | Safety Analysis Set | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | |
| Overall Study | Safety Analysis Set | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | |
| Overall Study | Pharmacodynamic Analysis Set | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | |
| Overall Study | Pharmacodynamic Analysis Set | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | |
| Overall Study | COMPLETED | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | |
| Overall Study | COMPLETED | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | |
| Overall Study | NOT COMPLETED | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 0 | |
| Overall Study | NOT COMPLETED | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 0 |
Source ClinicalTrials.gov
Adverse events216
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 | Lymphadenopathy | Blood and lymphatic system disorders | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 1 | 13 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 1 | 7 | |
| other | Atrioventricular Block | Cardiac disorders | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 1 | 13 | |
| other | Atrioventricular Block | Cardiac disorders | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 0 | 7 | |
| other | Ear Pain | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 0 | 13 | |
| other | Ear Pain | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 1 | 7 | |
| other | Hyperacusis | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 1 | 13 | |
| other | Hyperacusis | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 0 | 7 | |
| other | Hypoacusis | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 0 | 13 | |
| other | Hypoacusis | Ear and labyrinth disorders | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 1 | 7 |
1 to 10 of 216Source ClinicalTrials.gov
See all 216 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 0 | 13 |
| Total, all-cause mortality | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 0 | 7 |
| Total, other adverse events | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 13 | 13 |
| Total, other adverse events | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 7 | 7 |
| Total, serious adverse events | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | 4 | 13 |
| Total, serious adverse events | Lumasiran (ALN-GO1): 3.0 mg/kg QM | 3 | 7 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M vs Lumasiran (ALN-GO1): 3.0 mg/kg QM: Number of Participants With at Least One Adverse Event (AE) | Trial comparisons |
| Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M vs Lumasiran (ALN-GO1): 3.0 mg/kg QM: deaths from any cause | Trial comparisons |
| Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M vs Lumasiran (ALN-GO1): 3.0 mg/kg QM: 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/NCT03350451
GET https://conductscience.org/api/trials/v1/NCT03350451/events?page=2