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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-003877-40 | EudraCT Number |
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The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prior Placebo Group of Study 004 | Experimental | Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months. |
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| Prior Patisiran Group of Study 004 | Experimental | Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months. |
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| Prior Patisiran Group of Study 003 | Experimental | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Patisiran | Drug | Patisiran was administered IV. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation | 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. | First dose up to 28 days after last dose of study drug (approximately 5.6 years) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | The NIS assessment is a 244-point composite measure of neurologic impairment which includes a physical exam of lower limbs, upper limbs, and cranial nerves to assess the components: motor strength/weakness (NIS-W), reflexes (NIS-R), and sensation (NIS-S). NIS total score is obtained by combining all the component scores, ranging from 0 to 244. Higher scores represent a greater severity of disease. A positive change from baseline indicates the worsening of neuropathy. |
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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 | La Mesa | California | United States | |||
| Clinical Trial Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39804640 | Derived | Adams D, Wixner J, Polydefkis M, Berk JL, Conceicao IM, Dispenzieri A, Peltier A, Ueda M, Bender S, Capocelli K, Jay PY, Yureneva E, Obici L; patisiran Global OLE study group. Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension. JAMA Neurol. 2025 Mar 1;82(3):228-236. doi: 10.1001/jamaneurol.2024.4631. | |
| 38548524 |
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A total of 211 participants who completed either ALN-TTR02-003 (NCT01961921) or ALN-TTR02-004 (NCT01960348) studies were enrolled into the study to receive at least 1 dose of patisiran.
Participants took part in the study at investigational sites in Asia, Europe, Canada, the United Kingdom, and the United States from 16 July 2015 to 23 November 2022.
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| ID | Title | Description |
|---|---|---|
| FG000 | Prior Placebo Group of Study 004 | Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months. |
| FG001 | Prior Patisiran Group of Study 004 |
| 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 2, 2017 | Nov 16, 2023 |
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All the participants received only patisiran but divided into 3 arm groups based on administration in the parent study (placebo or patisiran in 003 [NCT01961921] or 004 [NCT01960348] studies).
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| Baseline, Year 5 |
| Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3 | The mNIS+7 is a composite measure of neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs, and cranial nerves to assess motor strength/weakness (192 points), reflexes (20 points), electrophysiologic measurement of small and large nerve fiber function (10 points), sensory testing (80 points), and postural blood pressure (2 points). The total mNIS+7 composite score is obtained by combining all the component scores, ranging from 0 (no impairment) to 304 (maximum impairment). A negative change from baseline indicates an improvement in neuropathy. | Baseline, Year 3 |
| Change From Baseline in the NIS+7 Total Score at Week 52 | The NIS+7 provides additional, objective measures of nerve fibre function and autonomic nerve function in participants with diabetic neuropathy. The NIS+7 includes the full NIS, sum of 5 nerve conduction studies (NCS) (Sural sensory nerve action potential [SNAP], tibial motor nerve distal latency, peroneal compound motor action potential [CMAP], motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing. The total NIS+7 score is obtained by combining all the component scores, ranging from 0 (no impairment) to 270 points (maximum impairment). A positive change from baseline indicates worsening. | Baseline, Week 52 |
| Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5 | The Norfolk QoL-DN questionnaire is a standardized 47-item patient-reported outcomes measure, sensitive to the perception of the effects of diabetic neuropathy by the participant. The scores range from -4 (best possible QOL) to 136 (worst possible QOL). A negative change from baseline represents improved QOL. | Baseline, Year 5 |
| Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5 | The EQ-5D-5L is a patient-reported measure of QoL based on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The overall score is rated on a scale from 0 (worst) to 1 (no impairment). Higher scores indicate a higher QoL. A negative change from baseline indicates worsening of QoL. | Baseline, Year 5 |
| Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5 | EQ-VAS measures the participant's self-rated health on a vertical scale evaluated on a scale of 0 ("worst health you can imagine") to 100 ("best health you can imagine"). Higher scores indicate a higher QOL. A negative change from baseline indicates worsening of QoL. | Baseline, Year 5 |
| Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52 | COMPASS 31 questionnaire measures autonomic symptoms in participants with neuropathy. The questionnaire consists of 31 clinically selected questions evaluating 6 autonomic domains (orthostatic intolerance, secretomotor, gastrointestinal, bladder, and pupillomotor). COMPASS 31 is measured on a scale from 0 to 100, with 100 representing maximum impairment. | Baseline, Week 52 |
| Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5 | Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kilograms per square meter [kg/m^2]) multiplied by the concentration of serum albumin (grams per liter [g/L]). A positive change from baseline indicates improvement in nutritional status. | Baseline, Year 5 |
| Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5 | The R-ODS is a 24-item patient-reported questionnaire that specifically captures activity and social participation limitations. It measures the level of disability on a scale of 0 (worst) to 48 (best, no limitations), higher score indicates a better outcome. A negative change from baseline indicates worsening of disability. | Baseline, Year 5 |
| Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5 | The NIS+7 provides additional, objective measures of nerve fiber function and autonomic nerve function in participants with diabetic neuropathy. The NIS+7 includes the full NIS (NIS-W, NIS-R, NIS-S), sum of 5 nerve conduction studies (NCS) (Sural SNAP, tibial motor nerve distal latency, peroneal CMAP, motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing. NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The score ranges from 0 to 192. A higher score indicates greater severity of disease. | Baseline, Year 5 |
| Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5 | 10-MWT is a measure of ambulatory ability and walk speed. It measures the speed (in meters per second [m/s]) of a participant to walk 10 meters. A negative change from baseline represents decreased ambulatory ability. | Baseline, Year 5 |
| Change From Baseline in the Hand Grip Strength at Week 52 | Hand grip strength was measured by dynamometer. Grip strength in the dominant arm is a measure of motor function, with a higher grip strength indicating better motor function. The mean change from baseline in the hand grip strength was reported. | Baseline, Week 52 |
| Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage | PND measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (sensory disturbances but preserved walking capability), II (impaired walking capability but ability to walk without a stick or crutches), IIIA (walking with help of 1 stick/crutch), IIIB (with help of 2 sticks/crutches), and IV (confined to wheelchair or bedridden). Lower scores indicate greater ambulatory function. The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Baseline, Year 5 |
| Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage | FAP measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (unimpaired ambulation; mostly mild sensory, motor, and autonomic neuropathy in the lower limbs), II (assistance with ambulation required; moderate impairment of the lower limbs, upper limbs, and trunk), and III (wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs). Lower scores indicate greater ambulatory function. The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Baseline, Year 5 |
| Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification | NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea). The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Baseline, Year 5 |
| Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5 | IENFD (fibers/millimeter [mm]) is a measure for the pathologic evaluation of sensory and autonomic innervation. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening. | Baseline, Year 5 |
| Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5 | SGNFD (meter/cubic millimeter [m/mm^3]) is a measure for the pathologic evaluation of sensory and autonomic innervation. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening. | Baseline, Year 5 |
| Change From Baseline in the Dermal Amyloid Burden at Year 5 | Dermal Amyloid Burden is a measure for the pathologic evaluation of sensory and autonomic innervation and reported as % congo red stain. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. | Baseline, Year 5 |
| Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5 | Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: troponin (micrograms per liter [µg/L]). The troponin I values <0.1 μg/L were imputed to 0.1 thus the actual changes cannot be calculated for values <0.1 μg/L. | Baseline, Year 5 |
| Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5 | Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: NT-proBNP (nanograms per liter [ng/L]). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5 | The echocardiogram parameters analyzed included measures of systolic function: Average peak longitudinal strain (percentage [%]). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: LV mass (grams [g]). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5 | The echocardiogram parameters analyzed included measures of diastolic function: LV end-diastolic volume (milliliters [mL]). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: LV relative wall thickness (ratio). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: Mean LV wall thickness (centimeters [cm]). | Baseline, Year 5 |
| Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5 | The echocardiogram parameters analyzed included measures of systolic function: Cardiac output (liters per minute [L/min]). | Baseline, Year 5 |
| Percent Change From Baseline in Serum TTR Levels at Year 5 | Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA). | Baseline, Year 5 |
| Aurora |
| Colorado |
| United States |
| Clinical Trial Site | Jacksonville | Florida | 32224 | United States |
| Clinical Trial Site | Chicago | Illinois | United States |
| Clinical Trial Site | Baltimore | Maryland | United States |
| Clinical Trial Site | Boston | Massachusetts | United States |
| Clinical Trial Site | Detroit | Michigan | United States |
| Clinical Trial Site | Rochester | Minnesota | United States |
| Clinical Trial Site | Joplin | Missouri | 64804 | United States |
| Clinical Trial Site | St Louis | Missouri | United States |
| Clinical Trial Site | Cooperstown | New York | United States |
| Clinical Trial Site | New York | New York | 10029 | United States |
| Clinical Trial Site | New York | New York | 10032 | United States |
| Clinical Trial Site | Columbus | Ohio | 43210 | United States |
| Clinical Trial Site | Buenos Aires | Argentina |
| Clinical Trial Site | Westmead | Australia |
| Clinical Trial Site | Rio de Janeiro | Brazil |
| Clinical Trial Site | São Paulo | Brazil |
| Clinical Trial Site | Sofia | Bulgaria |
| Clinical Trial Site | Vancouver | British Columbia | Canada |
| Clinical Trial Site | Nicosia | Cyprus |
| Clinical Trial Site | Fort de France | Martinique | 97261 | France |
| Clinical Trial Site | Saint-Pierre | Reunion Island | 97448 | France |
| Clinical Trial Site | Créteil | France |
| Clinical Trial Site | Le Kremlin-Bicêtre | France |
| Clinical Trial Site | Lille | France |
| Clinical Trial Site | Marseille | France |
| Clinical Trial Site | Cologne | 50937 | Germany |
| Clinical Trial Site | Heidelberg | Germany |
| Clinical Trial Site | Münster | Germany |
| Clinical Trial Site | Pavia | Italy |
| Clinical Trial Site | Rome | Italy |
| Clinical Trial Site | Sicily | Italy |
| Clinical Trial Site | Ehime | Japan |
| Clinical Trial Site | Fukuoka | Japan |
| Clinical Trial Site | Hiroshima | Japan |
| Clinical Trial Site | Kumamoto | Japan |
| Clinical Trial Site | Nagano | Japan |
| Clinical Trial Site | Okawasuji | Japan |
| Clinical Trial Site | Ono | Japan |
| Clinical Trial Site | Ōita | Japan |
| Clinical Trial Site | Kuala Lumpur | Malaysia |
| Clinical Trial Site | Tlalpan | Mexico |
| Clinical Trial Site | Groningen | Netherlands |
| Clinical Trial Site | Lisbon | Portugal |
| Clinical Trial Site | Porto | Portugal |
| Clinical Trial Site | Seoul | South Korea |
| Clinical Trial Site | Barcelona | Spain |
| Clinical Trial Site | Huelva | Spain |
| Clinical Trial Site | Madrid | Spain |
| Clinical Trial Site | Palma de Mallorca | Spain |
| Clinical Trial Site | Umeå | Sweden |
| Clinical Trial Site | Taipei | 11217 | Taiwan |
| Clinical Trial Site | Taipei | Taiwan |
| Clinical Trial Site | Istanbul | Turkey (Türkiye) |
| Clinical Trial Site | London | United Kingdom |
| Derived |
| Lin KP, Yang CC, Lee YC, Lee MJ, Vest J, Sweetser MT, White MT, Badri P, Hsieh ST, Chao CC. Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study. J Formos Med Assoc. 2024 Sep;123(9):975-984. doi: 10.1016/j.jfma.2024.03.008. Epub 2024 Mar 27. |
| 33212063 | Derived | Adams D, Polydefkis M, Gonzalez-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada Lopez IA, Dispenzieri A, Quan D, Conceicao IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Plante-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. |
Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months. |
| FG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
| Full Analysis Set | Full analysis set included all participants who were enrolled in this study. |
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| Safety Analysis Set | Safety analysis set included all the enrolled participants who received at least 1 dose of patisiran in this study. |
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| Pharmacodynamic (PD) Analysis Set | PD analysis set included all participants who received at least 1 dose of patisiran in this study and have had both baseline and at least 1 post-baseline PD assessment (either transthyretin [TTR] or vitamin A). |
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| COMPLETED |
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| NOT COMPLETED |
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Full analysis set included all participants who were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Prior Placebo Group of Study 004 | Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 65.5 months. |
| BG001 | Prior Patisiran Group of Study 004 | Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months. |
| BG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
| BG003 | 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 |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Serum Transthyretin (TTR) Level | 'Number analyzed' indicates the number of participants with data available for analysis of the specified measure. | Mean | Standard Deviation | mg/L |
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| 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 | Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation | 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 the enrolled participants who received at least 1 dose of patisiran in this study. Percentages are rounded off to the nearest decimal point. | Posted | Number | percentage of participants | First dose up to 28 days after last dose of study drug (approximately 5.6 years) |
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| Secondary | Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | The NIS assessment is a 244-point composite measure of neurologic impairment which includes a physical exam of lower limbs, upper limbs, and cranial nerves to assess the components: motor strength/weakness (NIS-W), reflexes (NIS-R), and sensation (NIS-S). NIS total score is obtained by combining all the component scores, ranging from 0 to 244. Higher scores represent a greater severity of disease. A positive change from baseline indicates the worsening of neuropathy. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3 | The mNIS+7 is a composite measure of neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs, and cranial nerves to assess motor strength/weakness (192 points), reflexes (20 points), electrophysiologic measurement of small and large nerve fiber function (10 points), sensory testing (80 points), and postural blood pressure (2 points). The total mNIS+7 composite score is obtained by combining all the component scores, ranging from 0 (no impairment) to 304 (maximum impairment). A negative change from baseline indicates an improvement in neuropathy. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | score on a scale | Baseline, Year 3 |
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| Secondary | Change From Baseline in the NIS+7 Total Score at Week 52 | The NIS+7 provides additional, objective measures of nerve fibre function and autonomic nerve function in participants with diabetic neuropathy. The NIS+7 includes the full NIS, sum of 5 nerve conduction studies (NCS) (Sural sensory nerve action potential [SNAP], tibial motor nerve distal latency, peroneal compound motor action potential [CMAP], motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing. The total NIS+7 score is obtained by combining all the component scores, ranging from 0 (no impairment) to 270 points (maximum impairment). A positive change from baseline indicates worsening. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | score on a scale | Baseline, Week 52 |
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| Secondary | Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5 | The Norfolk QoL-DN questionnaire is a standardized 47-item patient-reported outcomes measure, sensitive to the perception of the effects of diabetic neuropathy by the participant. The scores range from -4 (best possible QOL) to 136 (worst possible QOL). A negative change from baseline represents improved QOL. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5 | The EQ-5D-5L is a patient-reported measure of QoL based on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The overall score is rated on a scale from 0 (worst) to 1 (no impairment). Higher scores indicate a higher QoL. A negative change from baseline indicates worsening of QoL. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5 | EQ-VAS measures the participant's self-rated health on a vertical scale evaluated on a scale of 0 ("worst health you can imagine") to 100 ("best health you can imagine"). Higher scores indicate a higher QOL. A negative change from baseline indicates worsening of QoL. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52 | COMPASS 31 questionnaire measures autonomic symptoms in participants with neuropathy. The questionnaire consists of 31 clinically selected questions evaluating 6 autonomic domains (orthostatic intolerance, secretomotor, gastrointestinal, bladder, and pupillomotor). COMPASS 31 is measured on a scale from 0 to 100, with 100 representing maximum impairment. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 52 |
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| Secondary | Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5 | Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kilograms per square meter [kg/m^2]) multiplied by the concentration of serum albumin (grams per liter [g/L]). A positive change from baseline indicates improvement in nutritional status. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | kg.g/m^2.L | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5 | The R-ODS is a 24-item patient-reported questionnaire that specifically captures activity and social participation limitations. It measures the level of disability on a scale of 0 (worst) to 48 (best, no limitations), higher score indicates a better outcome. A negative change from baseline indicates worsening of disability. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5 | The NIS+7 provides additional, objective measures of nerve fiber function and autonomic nerve function in participants with diabetic neuropathy. The NIS+7 includes the full NIS (NIS-W, NIS-R, NIS-S), sum of 5 nerve conduction studies (NCS) (Sural SNAP, tibial motor nerve distal latency, peroneal CMAP, motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing. NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The score ranges from 0 to 192. A higher score indicates greater severity of disease. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Year 5 |
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| Secondary | Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5 | 10-MWT is a measure of ambulatory ability and walk speed. It measures the speed (in meters per second [m/s]) of a participant to walk 10 meters. A negative change from baseline represents decreased ambulatory ability. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | m/s | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Hand Grip Strength at Week 52 | Hand grip strength was measured by dynamometer. Grip strength in the dominant arm is a measure of motor function, with a higher grip strength indicating better motor function. The mean change from baseline in the hand grip strength was reported. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Error | kg | Baseline, Week 52 |
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| Secondary | Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage | PND measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (sensory disturbances but preserved walking capability), II (impaired walking capability but ability to walk without a stick or crutches), IIIA (walking with help of 1 stick/crutch), IIIB (with help of 2 sticks/crutches), and IV (confined to wheelchair or bedridden). Lower scores indicate greater ambulatory function. The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline, Year 5 |
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| Secondary | Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage | FAP measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (unimpaired ambulation; mostly mild sensory, motor, and autonomic neuropathy in the lower limbs), II (assistance with ambulation required; moderate impairment of the lower limbs, upper limbs, and trunk), and III (wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs). Lower scores indicate greater ambulatory function. The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline, Year 5 |
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| Secondary | Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification | NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea). The number of participants with change in the stage from baseline was reported as: Improved or worsened. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Count of Participants | Participants | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5 | IENFD (fibers/millimeter [mm]) is a measure for the pathologic evaluation of sensory and autonomic innervation. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of the specified parameter at specified timepoint. | Posted | Mean | Standard Deviation | fibers/mm | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5 | SGNFD (meter/cubic millimeter [m/mm^3]) is a measure for the pathologic evaluation of sensory and autonomic innervation. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number of participants analyzed' indicates the number of participants with data available for analysis of the specified parameter at specified timepoint. | Posted | Mean | Standard Deviation | m/mm^3 | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Dermal Amyloid Burden at Year 5 | Dermal Amyloid Burden is a measure for the pathologic evaluation of sensory and autonomic innervation and reported as % congo red stain. It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg. | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis of the specified parameter at specified timepoint. | Posted | Mean | Standard Deviation | percent congo red stain | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5 | Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: troponin (micrograms per liter [µg/L]). The troponin I values <0.1 μg/L were imputed to 0.1 thus the actual changes cannot be calculated for values <0.1 μg/L. | Full analysis set included all participants who were enrolled in this study. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | µg/L | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5 | Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: NT-proBNP (nanograms per liter [ng/L]). | Full analysis set included all participants who were enrolled in this study. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | ng/L | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5 | The echocardiogram parameters analyzed included measures of systolic function: Average peak longitudinal strain (percentage [%]). | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | percentage | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: LV mass (grams [g]). | Full analysis set included all participants who were enrolled in this study. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | grams | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5 | The echocardiogram parameters analyzed included measures of diastolic function: LV end-diastolic volume (milliliters [mL]). | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | mL | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: LV relative wall thickness (ratio). | Full analysis set included all participants who were enrolled in this study. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | ratio | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5 | The echocardiogram parameters analyzed included measures of cardiac structure: Mean LV wall thickness (centimeters [cm]). | Full analysis set included all participants who were enrolled in this study. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | cm | Baseline, Year 5 |
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| Secondary | Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5 | The echocardiogram parameters analyzed included measures of systolic function: Cardiac output (liters per minute [L/min]). | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | Posted | Mean | Standard Deviation | L/min | Baseline, Year 5 |
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| Secondary | Percent Change From Baseline in Serum TTR Levels at Year 5 | Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA). | PD analysis set included all participants who received at least 1 dose of patisiran in this study and have had both baseline and at least 1 post-baseline PD assessment (either TTR or vitamin A). 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. | Posted | Mean | Standard Deviation | percent change | Baseline, Year 5 |
|
First dose up to 28 days after last dose of study drug (approximately 5.6 years)
Safety analysis set included all the enrolled participants who received at least 1 dose of patisiran in this study.
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 | Prior Placebo Group of Study 004 | Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 65.5 months. | 19 | 49 | 39 | 49 | 47 | 49 |
| EG001 | Prior Patisiran Group of Study 004 | Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months. | 21 | 137 | 82 | 137 | 135 | 137 |
| EG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. | 1 | 25 | 12 | 25 | 25 | 25 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Myocardial Infarction | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrial Fibrillation | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrial Flutter | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrial Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrioventricular Block | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrioventricular Block Complete | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrioventricular Block Second Degree | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Amyloidosis | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Arrest | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Failure | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Failure Congestive | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Flutter | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Tamponade | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiogenic Shock | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Conduction Disorder | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Coronary Artery Stenosis | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Myocardial Infarction | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulseless Electrical Activity | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Restrictive Cardiomyopathy | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sinus Node Dysfunction | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ventricular Arrhythmia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ventricular Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Familial Amyloidosis | Congenital, familial and genetic disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hereditary Neuropathic Amyloidosis | Congenital, familial and genetic disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sudden Hearing Loss | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vertigo Positional | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperthyroidism | Endocrine disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Corneal Perforation | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Deposit Eye | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Eye Haemorrhage | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Glaucoma | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Keratitis | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Retinal Artery Occlusion | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Retinal Detachment | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Retinal Ischaemia | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rhegmatogenous Retinal Detachment | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ulcerative Keratitis | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vitreous Haemorrhage | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vitreous Opacities | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Distension | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea Haemorrhagic | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Faeces Pale | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrointestinal Disorder | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrointestinal Haemorrhage | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Inguinal Hernia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Large Intestine Polyp | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pancreatitis Acute | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Umbilical Hernia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Chest Discomfort | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Extravasation | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gait Disturbance | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| General Physical Health Deterioration | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Generalised Oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Implant site injury | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Polyp | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sudden Cardiac Death | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sudden Death | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Systemic Inflammatory Response Syndrome | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bile Duct Stone | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cholecystitis Acute | Hepatobiliary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| COVID-19 Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Clostridium Difficile Colitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gangrene | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Herpes Zoster | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Infected Skin Ulcer | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Infusion Site Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Parotitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Peritonitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia Bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pyelonephritis Acute | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Septic Shock | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Soft Tissue Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Vascular Device Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Viral Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Wound Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Ankle Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Burns Second Degree | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Facial Bones Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Femur Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Fibula Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Fractured Sacrum | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Hip Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Joint Dislocation | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Limb Injury | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Lower Limb Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Lumbar Vertebral Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Rib Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Road Traffic Accident | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Spinal Compression Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Stoma Site Extravasation | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Tibia Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Traumatic Haematoma | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Blood Creatine Phosphokinase Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Intraocular Pressure Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Weight Decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Cachexia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Electrolyte Imbalance | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fluid Overload | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Metabolic Acidosis | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Amyloid Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Foot Deformity | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Groin Pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lumbar Spinal Stenosis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neck Pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rotator Cuff Syndrome | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Benign Renal Neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Bladder Cancer Recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Bone Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Brain Cancer Metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Breast Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Cholangiocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Gastric Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Hypopharyngeal Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Intraductal Proliferative Breast Lesion | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Invasive Ductal Breast Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Metastases to Lymph Nodes | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Metastases to Meninges | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Metastatic Malignant Melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Myelodysplastic Syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Ocular Surface Squamous Neoplasia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Oncocytoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
| |
| Autonomic Nervous System Imbalance | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Basal Ganglia Infarction | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Carpal Tunnel Syndrome | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cerebral Infarction | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cerebrovascular Accident | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoxic-ischaemic Encephalopathy | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Psychomotor Skills Impaired | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Subarachnoid Haemorrhage | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Device Capturing Issue | Product Issues | MedDRA 23.0 | Systematic Assessment |
| |
| Device Physical Property Issue | Product Issues | MedDRA 23.0 | Systematic Assessment |
| |
| Device Power Source Issue | Product Issues | MedDRA 23.0 | Systematic Assessment |
| |
| Confusional State | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Conversion Disorder | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Kidney Injury | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Calculus Bladder | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Chronic Kidney Disease | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| End Stage Renal Disease | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neurogenic Bladder | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Urethral Haemorrhage | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary Bladder Haemorrhage | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary Retention | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Benign Prostatic Hyperplasia | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Pulmonary Oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Respiratory Failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoventilation | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary Oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory Acidosis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory Arrest | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory Disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sleep Apnoea Syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Decubitus Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Disability | Social circumstances | MedDRA 23.0 | Systematic Assessment |
| |
| Rehabilitation Therapy | Social circumstances | MedDRA 23.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypovolaemic Shock | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neurogenic Shock | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Orthostatic Hypotension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Phlebitis | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Poor Peripheral Circulation | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Shock | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Shock Haemorrhagic | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Varicose Vein | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrial Fibrillation | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Atrial Flutter | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cardiac Failure | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Ear Pain | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vertigo Positional | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Conjunctival Haemorrhage | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Visual Impairment | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal Pain Upper | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dental Caries | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrooesophageal Reflux Disease | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gait Disturbance | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Peripheral Swelling | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Infusion Related Reaction | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Infected Skin Ulcer | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Localised Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Respiratory Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Wound Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Foot Fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Ligament Sprain | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Limb Injury | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Post-traumatic Pain | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Skin Abrasion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Thermal Burn | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| Traumatic Haematoma | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
| |
| N-terminal Prohormone Brain Natriuretic Peptide Increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Weight Decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vitamin D Deficiency | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Musculoskeletal Pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute Kidney Injury | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Renal Failure | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Urinary Retention | Renal and urinary disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Benign Prostatic Hyperplasia | Reproductive system and breast disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Decubitus Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin Lesion | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Skin Ulcer | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Orthostatic Hypotension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
|
Not provided
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 | Jul 12, 2017 | Nov 16, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000686 | Amyloidosis |
| D028227 | Amyloid Neuropathies, Familial |
| ID | Term |
|---|---|
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D017772 | Amyloid Neuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028226 | Amyloidosis, Familial |
| D008661 | Metabolism, Inborn Errors |
Not provided
Not provided
| ID | Term |
|---|---|
| C000606954 | patisiran |
Not provided
Not provided
Not provided
|
|
|
|
|
| OG002 |
| Prior Patisiran Group of Study 003 |
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
| OG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
| OG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
| OG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
|
|
|
|
| Prior Patisiran Group of Study 003 |
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
| OG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.
| OG002 | Prior Patisiran Group of Study 003 | Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
| Prior Patisiran Group of Study 003 |
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months. |
|
|
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|