A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-005115-13-ES

Enrollment

164

Registered

2018-02-19

Start date

2018-04-25

Completion date

Unknown

Last updated

2018-06-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Amyloid Polyneuropathy MedDRA version: 20.0 Level: LLT Classification code 10057949 Term: Familial amyloid polyneuropathy System Organ Class: 100000004850

Interventions

Product Name: Human Transthyretin Antisense Oligonucleotide Product Code: ISIS 420915 Pharmaceutical Form: Solution for injection INN or Proposed INN: ISIS 420915 CAS Number: 1432726-13-0 Current Spon

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria: None

Design outcomes

Primary

Measure	Time frame
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;Primary end point(s): - Time to death from first dose of Study Drug in ISIS 420915-CS2 (inotersen or placebo) - Time to death from first dose of inotersen - Percentage of patients that died within 66 weeks, as well as 3 years, of first dose of Study Drug in ISIS 420915-CS2 - 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;Timepoint(s) of evaluation of this end point: 2 years	—

Measure

Time frame

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;Primary end point(s): - Time to death from first dose of Study Drug in ISIS 420915-CS2 (inotersen or placebo) - Time to death from first dose of inotersen - Percentage of patients that died within 66 weeks, as well as 3 years, of first dose of Study Drug in ISIS 420915-CS2 - 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;Timepoint(s) of evaluation of this end point: 2 years

—

Secondary

Measure	Time frame
Secondary end point(s): - Cause of death;Timepoint(s) of evaluation of this end point: 2 years	—

Countries

Argentina, Brazil, France, Germany, Italy, New Zealand, Portugal, Spain, United Kingdom, United States

Contacts

Public ContactMatthew Rael

Ionis Pharmaceuticals, Inc

mrael@ionisph.com011760603 2387

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026