A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3
Phase 1
- Conditions
- Familial Amyloid PolyneuropathyMedDRA version: 20.0 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy System Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2017-005115-13-GB
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 164
Inclusion Criteria
1. Patient or representative must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
2. Patient must be part of the ISIS 420915-CS2 safety population (defined as all patients that received at least 1 dose of Study Drug (placebo or inotersen)) or representative(s) of patient in ISIS 420915-CS2 safety population
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 82
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 82
Exclusion Criteria
None
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To monitor the survival of patients who are no longer actively participating in ISIS 420915-CS2 or ISIS 420915-CS3 and were in the ISIS 420915-CS2 safety population (defined as all patients that received at least 1 dose of Study Drug (placebo or inotersen));Secondary Objective: To collect information about the cause of death and related circumstances;<br> Primary end point(s): - Time to death from first dose of Study Drug in ISIS 420915-CS2 (inotersen or placebo)<br> - Time to death from first dose of inotersen<br> - Percentage of patients that died within 66 weeks, as well as 3 years, of first dose of Study Drug in ISIS 420915-CS2<br> - Percentage of patients whose survival status is unknown 66 weeks, as well as 3 years, after first dose of Study Drug in ISIS 420915-CS2<br> ;Timepoint(s) of evaluation of this end point: 2 years
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Cause of death;Timepoint(s) of evaluation of this end point: 2 years