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

Phase 1

• Conditions: Transthyretin mediated amyloidosis (ATTR); MedDRA version: 20.0Level: PTClassification code 10007509Term: Cardiac amyloidosisSystem Organ Class: 10007541 - Cardiac disorders; MedDRA version: 20.0Level: PTClassification code 10019889Term: Hereditary neuropathic amyloidosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2014-003877-40-IT
• Lead Sponsor: ALNYLAM PHARMACEUTICALS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-18
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 228

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety and efficacy of long-term dosing with patisiran in patients with transthyretin-mediated amyloidosis (ATTR) amyloidosis;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 <br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): None;Timepoint(s) of evaluation of this end point: None