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

• Register: EUCTR
• Last refreshed on: 11 June 2018
• Main ID: EUCTR2017-005115-13-ES
• Date of registration: 19/02/2018
• Prospective Registration: Yes
• Primary sponsor: Ionis Pharmaceuticals, Inc.
• Public title: A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3
• Scientific title: A Non-Interventional Study to Monitor the Survival Status of Patients that Discontinued from ISIS 420915-CS2 or ISIS 420915-CS3
• Date of first enrolment: 25/04/2018
• Target sample size: 164
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-005115-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: yes; Other trial design description: Patient follow-up to determine survival status; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Matthew Rael
• Affiliation: Ionis Pharmaceuticals, Inc

• Name: Matthew Rael
• Affiliation: Ionis Pharmaceuticals, Inc

Key inclusion & exclusion 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:
None


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

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 Sponsor code: ISIS 420915
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-

Primary Outcome(s)

Secondary Objective: To collect information about the cause of death and related circumstances

Timepoint(s) of evaluation of this end point: 2 years

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))

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

Secondary Outcome(s)

Secondary end point(s): - Cause of death

Timepoint(s) of evaluation of this end point: 2 years

Secondary ID(s)

ISIS420915-CS4

Source(s) of Monetary Support

Ionis Pharmaceuticals