The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
- Registration Number
- NCT01961921
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 27
- Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
- Adequate Karnofsky performance status, liver function, and renal function.
- Pregnant or nursing.
- Has had a liver transplant.
- Has a New York Heart Association heart failure classification >2.
- Has unstable angina.
- Has uncontrolled clinically significant cardiac arrhythmia.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ALN-TTR02 (patisiran) ALN-TTR02 (patisiran) administered by intravenous (IV) infusion -
- Primary Outcome Measures
Name Time Method The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation From Baseline up to 56 days post last dose 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.
- Secondary Outcome Measures
Name Time Method Change in Gait Speed With 10-meter Walk Test Baseline, Month 24 The 10-meter walk test measures the time (in seconds) that it takes a patient to walk 10 meters.
Mean Change From Baseline in Hand Grip Strength Baseline, Month 24 The mean (SEM) hand grip strength change from baseline at 24 months between patients who used patisiran with or without a concomitant TTR stabilizer.
Percentage Change From Baseline in Serum TTR Levels From Baseline up to 56 days post last dose TTR levels, measured using the enzyme-linked immunosorbent assay (ELISA) method.
Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Baseline, Month 24 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.
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) Baseline, Month 24 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).
Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI) Baseline, Month 24 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.
Trial Locations
- Locations (1)
Clinical Trial Site
🇸🇪Umeå, Sweden