Primary Hyperoxaluria Type 1 (PH1)Phase 1/2NCT02706886
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
A Phase 1/2 study testing Lumasiran in people with Primary Hyperoxaluria Type 1 (PH1), run by Alnylam Pharmaceuticals at 9 sites in 5 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). Read more about this study
- Who is it for?
- Ages 6 to 64 yearsHealthy volunteers can take part
- Conditions the study is for
- Primary Hyperoxaluria Type 1 (PH1)
- What is being tested?
- Lumasiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 8 Mar 2016to 23 Jan 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 64 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.
- Willing to provide written informed consent and to comply with study requirements.
- Additional Inclusion Criteria for Part B:
- Confirmation of PH1 disease
- Meet 24 hour urine oxalate excretion requirements
- Estimated glomerular filtration rate (GFR) of >45 mL/min/1.73m^2
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Who cannot join
- Clinically significant health concerns (with the exception of PH1 for patients in Part B)
- Clinically significant electrocardiogram (ECG) abnormalities
- Abnormal for aspartate aminotransferase (AST)/alanine aminotransferase (ALT) and any other clinical safety laboratory result considered clinically significant
- Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study
- Known history of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)
- History of intolerance to subcutaneous injection
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
France3 sites
Clinical Trial Site
Bordeaux
Clinical Trial Site
Lyon
Clinical Trial Site
Paris
United Kingdom2 sites
Clinical Trial Site
Birmingham
Clinical Trial Site
London
About this study
Official title: A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). In Part A, single ascending dose (SAD) part, healthy adults were dosed with lumasiran or placebo once. In Part B, multiple ascending doses (MAD) part, patients with primary hyperoxaluria type 1 (PH1) were dosed with lumasiran or placebo. All patients that initially received placebo received lumasiran after completing placebo dosing.
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Being tested
- Lumasiran, Placebo
- Keywords
- PH1, Primary Hyperoxaluria, RNAi therapeutic, siRNA, AGT
- Medical subject headings
- Primary hyperoxaluria type 1, Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LumasiranDrug
Also called ALN-GO1
Lumasiran will be administered SC once in Part A. In Part B lumasiran will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).
PlaceboDrug
Matching placebo (sterile saline: 0.9% sodium chloride [NaCl]) will be administered SC once in Part A. In Part B matching placebo will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).
| Study group | Role | What they receive |
|---|---|---|
| Part A: SAD: Placebo | Placebo comparator | A single dose of matching placebo will be administered subcutaneously (SC). Receives: Drug: Placebo |
| Part A: SAD: Lumasiran 0.3 mg/kg | Experimental | A single dose of 0.3 mg/kg lumasiran will be administered SC. Receives: Drug: Lumasiran |
| Part A: SAD: Lumasiran 1.0 mg/kg | Experimental | A single dose of 1.0 mg/kg lumasiran will be administered SC. Receives: Drug: Lumasiran |
| Part A: SAD: Lumasiran 3.0 mg/kg | Experimental | A single dose of 3.0 mg/kg lumasiran will be administered SC. Receives: Drug: Lumasiran |
| Part A: SAD: Lumasiran 6.0 mg/kg | Experimental | A single dose of 6.0 mg/kg lumasiran will be administered SC. Receives: Drug: Lumasiran |
| Part B: MAD: Placebo | Placebo comparator | Participants with primary hyperoxaluria type 1 (PH1) will be treated with placebo matching one of the lumasiran dosages in the lumasiran arms (one placebo participant for each lumasiran arm). At Day 85 these placebo treated participants will cross over to their respective Part B lumasiran arms in the Part B: MAD Study Day 85-End of Study Period and will then be treated with lumasiran. The estimated total time on study was up to 546 days. Receives: Drug: Placebo |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | Experimental | Participants with PH1 will be treated with 1.0 mg/kg lumasiran SC once monthly (qM) on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. Receives: Drug: Lumasiran |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | Experimental | Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC qM on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. Receives: Drug: Lumasiran |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | Experimental | Participants with PH1 will be treated with 3.0 mg/kg lumasiran SC once every three months (q3M) on Days 1 and 85. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. Receives: Drug: Lumasiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs)
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.
Measured: Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days
Secondary outcomes (16)
Maximum Concentration (Cmax) of Lumasiran in Plasma
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.
Measured: 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
Time to Cmax (Tmax) of Lumasiran in Plasma
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.
Measured: 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
Area Under the Concentration-Time Curve From Time 0 to Time of Last Measurable Concentration (AUC0-last) of Lumasiran in Plasma
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.
Measured: 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
Terminal Half-life (t1/2) of Lumasiran in Plasma
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.
Measured: 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
Fraction Excreted in Urine in 24 Hours (Fe0-24) of Lumasiran
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.
Measured: 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
Renal Clearance (CLR) of Lumasiran
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.
Measured: 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
Baseline Plasma Glycolate Concentration
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.
Measured: Part A (SAD): Baseline, Part B (MAD): Baseline
Percentage Change From Baseline in Plasma Glycolate Concentration
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.
Measured: Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85
Baseline Spot Urine Glycolate:Creatinine Ratio in Part A
The endpoint was only measured in Part A.
Measured: Part A (SAD): Baseline
Percentage Change From Baseline in Spot Urine Glycolate:Creatinine Ratio in Part A
The endpoint was only measured in Part A.
Measured: Part A (SAD): Days 29 and 57
Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
The endpoint was only measured in Part B.
Measured: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
The endpoint was only measured in Part B.
Measured: Part B (MAD): 24 hour urine collections on Days 29, 57, 85, 113, 141, 169, 197
Baseline 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
The endpoint was only measured during the initial 85 days in Part B.
Measured: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
The endpoint was only measured during the initial 85 days in Part B.
Measured: Part B (MAD): 24 hour urine collections on Days 29, 57 and 85
Baseline Creatinine Clearance Corrected for BSA in Part B
Measured: Part B (MAD): Baseline
Percentage Change From Baseline of Creatinine Clearance Corrected for BSA in Part B
Measured: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2016 (estimated)
- Study start8 Mar 2016
- Primary completion23 Jan 2019
- Study completion23 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Hyperoxaluria Type 1 (PH1)Studies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tracy McGregor, MD, MSCI, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02706886
- Sponsor's protocol ID
- ALN-GO1-001
- Other IDs
- 2015-004407-23, 2015
- Status last verified
- Jan 2020
- First submitted
- 3 Mar 2016
- Last updated
- 30 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
PubMed 41388232Frishberg Y, Deschenes G, Groothoff JW, Hulton SA, Magen D, Harambat J, Van't Hoff WG, Lorch U, Milliner DS, Lieske JC, Haslett P, Garg PP, Vaishnaw AK, Talamudupula S, Lu J, Habtemariam BA, Erbe DV, McGregor TL, Cochat P; study collaborators. Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial. Clin J Am Soc Nephrol. 2021 Jul;16(7):1025-1036. doi: 10.2215/CJN.14730920. Epub 2021 May 13.
PubMed 33985991
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Type 1
- ClinicalTrials.gov ID
- NCT02706886
- Acronym / sponsor ID
- ALN-GO1-001
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Keywords
- PH1, Primary Hyperoxaluria, RNAi therapeutic, siRNA, AGT
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Mar 2016 (actual)
- Primary completion
- 23 Jan 2019 (actual)
- Study completion
- 23 Jan 2019 (actual)
- First posted
- 11 Mar 2016 (estimated)
- Results
- Posted 30 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). In Part A, single ascending dose (SAD) part, healthy adults were dosed with lumasiran or placebo once. In Part B, multiple ascending doses (MAD) part, patients with primary hyperoxaluria type 1 (PH1) were dosed with lumasiran or placebo. All patients that initially received placebo received lumasiran after completing placebo dosing.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Part A: SAD: Placebo | Placebo comparator | Drug: PlaceboA single dose of matching placebo will be administered subcutaneously (SC). |
| Part A: SAD: Lumasiran 0.3 mg/kg | Experimental | Drug: LumasiranA single dose of 0.3 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 1.0 mg/kg | Experimental | Drug: LumasiranA single dose of 1.0 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 3.0 mg/kg | Experimental | Drug: LumasiranA single dose of 3.0 mg/kg lumasiran will be administered SC. |
| Part A: SAD: Lumasiran 6.0 mg/kg | Experimental | Drug: LumasiranA single dose of 6.0 mg/kg lumasiran will be administered SC. |
| Part B: MAD: Placebo | Placebo comparator | Drug: PlaceboParticipants with primary hyperoxaluria type 1 (PH1) will be treated with placebo matching one of the lumasiran dosages in the lumasiran arms (one placebo participant for each lumasiran arm). At Day 85 these placebo treated participants will cross over to their respective Part B lumasiran arms in the Part B: MAD Study Day 85-End of Study Period and will then be treated with lumasiran. The estimated total time on study was up to 546 days. |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | Experimental | Drug: LumasiranParticipants with PH1 will be treated with 1.0 mg/kg lumasiran SC once monthly (qM) on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | Experimental | Drug: LumasiranParticipants with PH1 will be treated with 3.0 mg/kg lumasiran SC qM on Days 1, 29 and 57. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | Experimental | Drug: LumasiranParticipants with PH1 will be treated with 3.0 mg/kg lumasiran SC once every three months (q3M) on Days 1 and 85. The estimated total time on study is up to 546 days. One participant from the Part B: MAD: Placebo arm will cross over to this lumasiran arm at Day 85. For this participant treatment with lumasiran starts at Day 85. |
Who may qualify
- Ages
- 6 to 64 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.
- Willing to provide written informed consent and to comply with study requirements.
- Confirmation of PH1 disease
- Meet 24 hour urine oxalate excretion requirements
- Estimated glomerular filtration rate (GFR) of >45 mL/min/1.73m^2
Key exclusion criteria (6 listed)
- Clinically significant health concerns (with the exception of PH1 for patients in Part B)
- Clinically significant electrocardiogram (ECG) abnormalities
- Abnormal for aspartate aminotransferase (AST)/alanine aminotransferase (ALT) and any other clinical safety laboratory result considered clinically significant
- Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study
- Known history of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LumasiranAlso: ALN-GO1Lumasiran will be administered SC once in Part A. In Part B lumasiran will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85). | Drug | Part A: SAD: Lumasiran 0.3 mg/kg, Part A: SAD: Lumasiran 1.0 mg/kg, Part A: SAD: Lumasiran 3.0 mg/kg, Part A: SAD: Lumasiran 6.0 mg/kg, Part B: MAD: Lumasiran 1.0 mg/kg qM, Part B: MAD: Lumasiran 3.0 mg/kg q3M, Part B: MAD: Lumasiran 3.0 mg/kg qM |
| PlaceboMatching placebo (sterile saline: 0.9% sodium chloride [NaCl]) will be administered SC once in Part A. In Part B matching placebo will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85). | Drug | Part A: SAD: Placebo, Part B: MAD: Placebo |
Primary outcome
Number of Participants With Adverse Events (AEs)
Time frame: 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.
16 secondary outcomes
Maximum Concentration (Cmax) of Lumasiran in Plasma
Time frame: 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
Time to Cmax (Tmax) of Lumasiran in Plasma
Time frame: 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
Area Under the Concentration-Time Curve From Time 0 to Time of Last Measurable Concentration (AUC0-last) of Lumasiran in Plasma
Time frame: 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
Terminal Half-life (t1/2) of Lumasiran in Plasma
Time frame: 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
Fraction Excreted in Urine in 24 Hours (Fe0-24) of Lumasiran
Time frame: 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
Renal Clearance (CLR) of Lumasiran
Time frame: 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
Baseline Plasma Glycolate Concentration
Time frame: Part A (SAD): Baseline, Part B (MAD): Baseline
Percentage Change From Baseline in Plasma Glycolate Concentration
Time frame: Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85
Baseline Spot Urine Glycolate:Creatinine Ratio in Part A
Time frame: Part A (SAD): Baseline
Percentage Change From Baseline in Spot Urine Glycolate:Creatinine Ratio in Part A
Time frame: Part A (SAD): Days 29 and 57
Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Oxalate Corrected for BSA in Part B
Time frame: Part B (MAD): 24 hour urine collections on Days 29, 57, 85, 113, 141, 169, 197
Baseline 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of 24 Hour Urine Glycolate:Creatinine Ratio in Part B - Initial 85 Days
Time frame: Part B (MAD): 24 hour urine collections on Days 29, 57 and 85
Baseline Creatinine Clearance Corrected for BSA in Part B
Time frame: Part B (MAD): Baseline
Percentage Change From Baseline of Creatinine Clearance Corrected for BSA in Part B
Time frame: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41388232Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
- Derived from this study: PubMed 33985991Frishberg Y, Deschenes G, Groothoff JW, Hulton SA, Magen D, Harambat J, Van't Hoff WG, Lorch U, Milliner DS, Lieske JC, Haslett P, Garg PP, Vaishnaw AK, Talamudupula S, Lu J, Habtemariam BA, Erbe DV, McGregor TL, Cochat P; study collaborators. Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial. Clin J Am Soc Nephrol. 2021 Jul;16(7):1025-1036. doi: 10.2215/CJN.14730920. Epub 2021 May 13.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tracy McGregor, MD, MSCI study director
Alnylam Pharmaceuticals
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Lumasiran in people with Primary Hyperoxaluria Type 1 (PH1), run by Alnylam Pharmaceuticals at 9 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in France, Israel, United Kingdom, Germany, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jan 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) (time frame: Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02706886, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2020.