Study to Evaluate the Long-term Safety and Efficacy of Patisiran (ALN-TTR02) in Patients with Familial Amyloidotic Polyneuropathy (FAP)

A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with Patisiran

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-003877-40-ES

Enrollment

228

Registered

2015-03-03

Start date

2015-05-11

Completion date

Unknown

Last updated

2015-08-24

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

Conditions

Transthyretin mediated amyloidosis (ATTR) MedDRA version: 17.1 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders MedDRA version: 17.1 Level: PT Classification code 10019889 Term: Hereditary neuropathic amyloidosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Patisiran Product Code: ALN-TTR02 Pharmaceutical Form: Solution for injection INN or Proposed INN: Patisiran Current Sponsor code: Patisiran Other descriptive name: ALN-TTR02 Concentrati

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Have completed a patisiran study 2. Adequate liver function and renal function 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 114 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 114

Exclusion criteria

Exclusion criteria: 1. Pregnant or nursing 2. Have uncontrolled clinically significant cardiac arrhythmia or unstable angina

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP);Secondary Objective: None;Primary end point(s): To assess the safety and efficacy of long-term dosing with patisiran;Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through 28 days post last dose. Efficacy assessments will be performed at Day 0, annually and 28 days post last dose	—

Measure

Time frame

Main Objective: To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP);Secondary Objective: None;Primary end point(s): To assess the safety and efficacy of long-term dosing with patisiran;Timepoint(s) of evaluation of this end point: The review of AEs will be done at each visit from Day 0 through 28 days post last dose. Efficacy assessments will be performed at Day 0, annually and 28 days post last dose

—

Secondary

Measure	Time frame
Secondary end point(s): None;Timepoint(s) of evaluation of this end point: None	—

Countries

Argentina, Brazil, Bulgaria, Canada, France, Germany, Italy, Japan, Korea, Republic of, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactMonica Bermejo

Medpace Spain, S.L

m.bermejo@medpace.com0034918534105

Outcome results

None listed

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