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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-003134-24 | EudraCT Number |
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The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | Experimental | Participants enrolling from study 001B (NCT02706886), received lumasiran, subcutaneous (SC) injection, at a starting dose of 1.0 milligrams per kilograms (mg/kg) once monthly (QM) or 3.0 mg/kg once every 3 months [Q3M]) from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose 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 Safety Review Committee (SRC). |
|
| Lumasiran (ALN-GO1): 3.0 mg/kg QM | Experimental | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lumasiran | Drug | Multiple doses of lumasiran by SC injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With at Least One Adverse Event (AE) | 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. | Baseline (Day -1) up to 54 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Bordeaux | France | ||||
| Clinical Trial Site |
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.
Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
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The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg once monthly (QM) & 3 mg/kg once every 3 months (Q3M), these participants were pooled into one arm as recommended by the Safety Review Committee (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 took part in the study at 9 study centers in France, Germany, Israel, Netherlands and the United Kingdom from 04 April 2018 to 07 February 2023. A total of 20 participants previously treated in Study 001B (NCT02706886) were enrolled and treated in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | Participants enrolling from study 001B, received lumasiran, subcutaneous (SC) injection, at a starting dose of 1.0 milligrams per kilograms (mg/kg) QM or 3.0 mg/kg (Q3M) from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 4, 2020 | Feb 7, 2024 |
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| Baseline (Day -1) up to 54 months |
| Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 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. | Baseline (Day -1) up to 54 months |
| Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 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. | Baseline (Day -1) up to 54 months |
| Lyon |
| France |
| Clinical Trial Site | Paris | France |
| Clinical Trial Site | Bonn | Germany |
| Clinical Trial Site | Haifa | Israel |
| Clinical Trial Site | Jerusalem | Israel |
| Clinical Trial Site | Amsterdam | Netherlands |
| Clinical Trial Site | Birmingham | United Kingdom |
| Clinical Trial Site | London | United Kingdom |
| FG001 | Lumasiran (ALN-GO1): 3.0 mg/kg QM | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
| Safety Analysis Set | Safety analysis set included all participants who received any amount of study drug. |
|
| Pharmacodynamic Analysis Set | Pharmacodynamic (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. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
Safety Analysis Set. 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.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose 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. |
| BG001 | Lumasiran (ALN-GO1): 3.0 mg/kg QM | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With at Least One Adverse Event (AE) | 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. | 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. | Posted | Count of Participants | Participants | Baseline (Day -1) up to 54 months |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 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. | 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. | Posted | Mean | Standard Error | mmol/24hr/1.73m^2 | Baseline (Day -1) up to 54 months |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 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. | 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. | Posted | Mean | Standard Deviation | mmol/mmol | Baseline (Day -1) up to 54 months |
| ||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 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. | 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. | Posted | Mean | Standard Error | mL/min/1.73m^2 | Baseline (Day -1) up to 54 months |
|
Baseline (Day -1) up to 54 months
Safety analysis set. 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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose 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. | 0 | 13 | 4 | 13 | 13 | 13 |
| EG001 | Lumasiran (ALN-GO1): 3.0 mg/kg QM | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. | 0 | 7 | 3 | 7 | 7 | 7 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thyroid Mass | Endocrine disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Bone Contusion | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Craniocerebral Injury | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Glomerular Filtration Rate Decreased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Renal Colic | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Renal Stone Removal | Surgical and medical procedures | MedDRA (25.0) | Systematic Assessment |
| |
| Thyroid Operation | Surgical and medical procedures | MedDRA (25.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Atrioventricular Block | Cardiac disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Ear Pain | Ear and labyrinth disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperacusis | Ear and labyrinth disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Thyroid Mass | Endocrine disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Eye Allergy | Eye disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Periorbital Swelling | Eye disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Diverticulum Intestinal | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Food Poisoning | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Large Intestine Polyp | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Feeling Cold | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Influenza Like Illness | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Injection Site Reaction | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Non-cardiac Chest Pain | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Vaccination Site Pain | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Vessel Puncture Site Haematoma | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Immunisation Reaction | Immune system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Seasonal Allergy | Immune system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Asymptomatic COVID-19 | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Cystitis Escherichia | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Ear Infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Tinea Versicolour | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Head Injury | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Ligament Sprain | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Limb Injury | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Skin Abrasion | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Skin Wound | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Stoma Site Pain | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Tibia Fracture | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Bilirubin Conjugated Increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Blood Bilirubin Increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Blood Creatinine Increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Electrocardiogram QRS Complex Prolonged | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Glycolic Acid Increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| SARS-CoV-2 Test Positive | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Ultrasound Breast Abnormal | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Ultrasound Kidney Abnormal | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Increased Appetite | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Iron Deficiency | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Vitamin D Deficiency | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Weight Gain Poor | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Flank Pain | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Device Breakage | Product Issues | MedDRA (25.0) | Systematic Assessment |
| |
| Affective Disorder | Psychiatric disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nightmare | Psychiatric disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Sleep Disorder | Psychiatric disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Renal Cyst | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Renal Pain | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Genital Pain | Reproductive system and breast disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Heavy Menstrual Bleeding | Reproductive system and breast disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Upper-airway Cough Syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gastrostomy Closure | Surgical and medical procedures | MedDRA (25.0) | Systematic Assessment |
| |
| Tooth Extraction | Surgical and medical procedures | MedDRA (25.0) | Systematic Assessment |
| |
| Wisdom Teeth Removal | Surgical and medical procedures | MedDRA (25.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Orthostatic Hypotension | Vascular disorders | MedDRA (25.0) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Alnylam Pharmaceuticals Inc. | 866-330-0326 | clinicaltrials@alnylam.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 23, 2019 | Feb 7, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006960 | Hyperoxaluria, Primary |
| D006959 | Hyperoxaluria |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D008659 | Metabolic Diseases |
| ID | Term |
|---|---|
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000716350 | lumasiran |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| White |
|
| Other |
|
| OG001 | Lumasiran (ALN-GO1): 3.0 mg/kg QM | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
|
|
| Lumasiran (ALN-GO1): 3.0 mg/kg QM |
Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
|
|
| OG001 | Lumasiran (ALN-GO1): 3.0 mg/kg QM | Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. |
|
|