The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
27
Clinical Trial Site
Boston, Massachusetts, United States
Clinical Trial Site
Rio de Janeiro, Brazil
Clinical Trial Site
Le Kremlin-Bicêtre, France
Clinical Trial Site
Marseille, France
The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
An AE is any untoward medical occurrence in a patient or clinical investigational patient administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.
Time frame: From Baseline up to 56 days post last dose
Percentage Change From Baseline in Serum TTR Levels
TTR levels, measured using the enzyme-linked immunosorbent assay (ELISA) method.
Time frame: From Baseline up to 56 days post last dose
Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7)
The mNIS+7 assessment is a composite measure of neurologic impairment that provides a comprehensive measure of large and small fiber function that encompasses the totality of the motor, sensory, and autonomic deficits seen in hereditary transthyretin-mediated amyloidosis (hATTR) patients with polyneuropathy. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
Time frame: Baseline, Month 24
Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS)
The overall EQ-5D is measured on a scale from 0 to 1, with 0 being worst and 1 best. The EQ-VAS is measured on a scale of 0-100, with 0 being the worst and 100 the best. The R-ODS captures activity and social participation limitations in patients. The R-ODS score ranges from 0 (most severe activity and social participation limitations) to 100 (no activity and social participation limitations).
Time frame: Baseline, Month 24
Change in Gait Speed With 10-meter Walk Test
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Clinical Trial Site
Münster, Germany
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Clinical Trial Site
Palma de Mallorca, Spain
Clinical Trial Site
Umeå, Sweden
The 10-meter walk test measures the time (in seconds) that it takes a patient to walk 10 meters.
Time frame: Baseline, Month 24
Mean Change From Baseline in Hand Grip Strength
The mean (SEM) hand grip strength change from baseline at 24 months between patients who used patisiran with or without a concomitant TTR stabilizer.
Time frame: Baseline, Month 24
Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI)
Nutritional status of patients was evaluated using the mBMI, calculated as BMI (kg/m\^2) multiplied by albumin (g/L). An increase from baseline in mBMI suggests improvement, and a decrease from baseline suggests worsening.
Time frame: Baseline, Month 24