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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02510261
Registration number
NCT02510261
Ethics application status
Date submitted
16/07/2015
Date registered
29/07/2015
Titles & IDs
Public title
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
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Scientific title
A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
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Secondary ID [1]
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0
2014-003877-40
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Secondary ID [2]
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ALN-TTR02-006
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Amyloidosis
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0
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Condition category
Condition code
Metabolic and Endocrine
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0
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0
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Metabolic disorders
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Metabolic and Endocrine
0
0
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0
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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0
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Patisiran
Experimental: 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.
Experimental: 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.
Experimental: 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.
Treatment: Drugs: Patisiran
Patisiran was administered IV.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation
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Assessment method [1]
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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.
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Timepoint [1]
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First dose up to 28 days after last dose of study drug (approximately 5.6 years)
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Secondary outcome [1]
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Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5
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Assessment method [1]
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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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Timepoint [1]
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Baseline, Year 5
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Secondary outcome [2]
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Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline, Year 3
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Secondary outcome [3]
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Change From Baseline in the NIS+7 Total Score at Week 52
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline, Week 52
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Secondary outcome [4]
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Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline, Year 5
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Secondary outcome [5]
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Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline, Year 5
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Secondary outcome [6]
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Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline, Year 5
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Secondary outcome [7]
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Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52
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Assessment method [7]
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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.
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Timepoint [7]
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Baseline, Week 52
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Secondary outcome [8]
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Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5
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Assessment method [8]
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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.
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Timepoint [8]
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Baseline, Year 5
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Secondary outcome [9]
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Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5
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Assessment method [9]
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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.
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Timepoint [9]
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Baseline, Year 5
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Secondary outcome [10]
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Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5
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Assessment method [10]
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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.
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Timepoint [10]
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Baseline, Year 5
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Secondary outcome [11]
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Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5
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Assessment method [11]
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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.
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Timepoint [11]
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Baseline, Year 5
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Secondary outcome [12]
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Change From Baseline in the Hand Grip Strength at Week 52
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Assessment method [12]
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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.
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Timepoint [12]
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Baseline, Week 52
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Secondary outcome [13]
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Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage
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Assessment method [13]
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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.
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Timepoint [13]
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Baseline, Year 5
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Secondary outcome [14]
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Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage
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Assessment method [14]
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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.
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Timepoint [14]
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Baseline, Year 5
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Secondary outcome [15]
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Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification
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Assessment method [15]
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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.
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Timepoint [15]
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Baseline, Year 5
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Secondary outcome [16]
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Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5
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Assessment method [16]
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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.
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Timepoint [16]
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Baseline, Year 5
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Secondary outcome [17]
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Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5
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Assessment method [17]
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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.
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Timepoint [17]
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Baseline, Year 5
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Secondary outcome [18]
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Change From Baseline in the Dermal Amyloid Burden at Year 5
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Assessment method [18]
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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.
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Timepoint [18]
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Baseline, Year 5
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Secondary outcome [19]
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Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5
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Assessment method [19]
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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.
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Timepoint [19]
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Baseline, Year 5
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Secondary outcome [20]
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Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5
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Assessment method [20]
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Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: NT-proBNP (nanograms per liter \[ng/L\]).
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Timepoint [20]
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Baseline, Year 5
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Secondary outcome [21]
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Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5
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Assessment method [21]
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The echocardiogram parameters analyzed included measures of systolic function: Average peak longitudinal strain (percentage \[%\]).
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Timepoint [21]
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Baseline, Year 5
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Secondary outcome [22]
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Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5
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Assessment method [22]
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The echocardiogram parameters analyzed included measures of cardiac structure: LV mass (grams \[g\]).
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Timepoint [22]
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Baseline, Year 5
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Secondary outcome [23]
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Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5
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Assessment method [23]
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The echocardiogram parameters analyzed included measures of diastolic function: LV end-diastolic volume (milliliters \[mL\]).
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Timepoint [23]
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Baseline, Year 5
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Secondary outcome [24]
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Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5
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Assessment method [24]
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The echocardiogram parameters analyzed included measures of cardiac structure: LV relative wall thickness (ratio).
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Timepoint [24]
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Baseline, Year 5
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Secondary outcome [25]
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Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5
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Assessment method [25]
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The echocardiogram parameters analyzed included measures of cardiac structure: Mean LV wall thickness (centimeters \[cm\]).
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Timepoint [25]
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Baseline, Year 5
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Secondary outcome [26]
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Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5
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Assessment method [26]
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The echocardiogram parameters analyzed included measures of systolic function: Cardiac output (liters per minute \[L/min\]).
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Timepoint [26]
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Baseline, Year 5
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Secondary outcome [27]
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Percent Change From Baseline in Serum TTR Levels at Year 5
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Assessment method [27]
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Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA).
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Timepoint [27]
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Baseline, Year 5
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Eligibility
Key inclusion criteria
* Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
* Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
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Minimum age
18
Years
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Maximum age
85
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Any new or uncontrolled condition that could make the participant unsuitable for participation
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/07/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
23/11/2022
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Sample size
Target
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Accrual to date
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Final
211
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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0
Clinical Trial Site - Westmead
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Recruitment postcode(s) [1]
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- Westmead
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Recruitment outside Australia
Country [1]
0
0
United States of America
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State/province [1]
0
0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Colorado
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Florida
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Illinois
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Maryland
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Country [6]
0
0
United States of America
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State/province [6]
0
0
Massachusetts
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Country [7]
0
0
United States of America
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State/province [7]
0
0
Michigan
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Country [8]
0
0
United States of America
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State/province [8]
0
0
Minnesota
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Country [9]
0
0
United States of America
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State/province [9]
0
0
Missouri
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Country [10]
0
0
United States of America
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State/province [10]
0
0
New York
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Country [11]
0
0
United States of America
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State/province [11]
0
0
Ohio
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Country [12]
0
0
Argentina
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State/province [12]
0
0
Buenos Aires
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Country [13]
0
0
Brazil
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State/province [13]
0
0
Rio de Janeiro
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Country [14]
0
0
Brazil
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State/province [14]
0
0
São Paulo
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Country [15]
0
0
Bulgaria
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State/province [15]
0
0
Sofia
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Country [16]
0
0
Canada
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State/province [16]
0
0
British Columbia
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Country [17]
0
0
Cyprus
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State/province [17]
0
0
Nicosia
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Country [18]
0
0
France
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State/province [18]
0
0
Martinique
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Country [19]
0
0
France
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State/province [19]
0
0
Reunion Island
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Country [20]
0
0
France
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State/province [20]
0
0
Creteil
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Country [21]
0
0
France
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State/province [21]
0
0
Le Kremlin-bicetre
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Country [22]
0
0
France
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State/province [22]
0
0
Lille
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Country [23]
0
0
France
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State/province [23]
0
0
Marseille Cedex
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Country [24]
0
0
Germany
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State/province [24]
0
0
Cologne
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Country [25]
0
0
Germany
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State/province [25]
0
0
Heidelberg
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Country [26]
0
0
Germany
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State/province [26]
0
0
Muenster
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Country [27]
0
0
Italy
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State/province [27]
0
0
Pavia
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Country [28]
0
0
Italy
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State/province [28]
0
0
Rome
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Country [29]
0
0
Italy
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State/province [29]
0
0
Sicily
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Country [30]
0
0
Japan
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State/province [30]
0
0
Ehime
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Country [31]
0
0
Japan
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State/province [31]
0
0
Fukuoka
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Country [32]
0
0
Japan
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State/province [32]
0
0
Hiroshima
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Country [33]
0
0
Japan
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State/province [33]
0
0
Kumamoto
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Country [34]
0
0
Japan
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State/province [34]
0
0
Nagano
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Country [35]
0
0
Japan
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State/province [35]
0
0
Okawasuji
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Country [36]
0
0
Japan
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State/province [36]
0
0
Ono
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Country [37]
0
0
Japan
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State/province [37]
0
0
Oita
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Country [38]
0
0
Korea, Republic of
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State/province [38]
0
0
Seoul
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Country [39]
0
0
Malaysia
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State/province [39]
0
0
Kuala Lumpur
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Country [40]
0
0
Mexico
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State/province [40]
0
0
Tlalpan
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Country [41]
0
0
Netherlands
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State/province [41]
0
0
Groningen
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Country [42]
0
0
Portugal
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State/province [42]
0
0
Lisbon
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Country [43]
0
0
Portugal
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State/province [43]
0
0
Porto
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Country [44]
0
0
Spain
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State/province [44]
0
0
Barcelona
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Country [45]
0
0
Spain
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State/province [45]
0
0
Huelva
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Country [46]
0
0
Spain
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State/province [46]
0
0
Madrid
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Country [47]
0
0
Spain
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State/province [47]
0
0
Palma De Mallorca
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Country [48]
0
0
Sweden
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State/province [48]
0
0
Umeå
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Country [49]
0
0
Taiwan
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State/province [49]
0
0
Taipei
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Country [50]
0
0
Turkey
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State/province [50]
0
0
Istanbul
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Country [51]
0
0
United Kingdom
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State/province [51]
0
0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Alnylam Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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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Trial website
https://clinicaltrials.gov/study/NCT02510261
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Trial related presentations / publications
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. Erratum In: Lancet Neurol. 2021 Feb;20(2):e2. doi: 10.1016/S1474-4422(20)30458-0.
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Public notes
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Contacts
Principal investigator
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Medical Director
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Alnylam Pharmaceuticals
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Contact person for public queries
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
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Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/61/NCT02510261/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/61/NCT02510261/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02510261