A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

TTR-mediated Amyloidosis Clinical Trial

Official title:

A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02292186
• Other study ID #: ALN-TTRSC-003
• Status: Completed
• Phase: Phase 2
• Start date: October 2014
• Est. completion date: February 22, 2017

Study information

• Verified date: July 2020
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: February 22, 2017
• Est. primary completion date: February 22, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 90 Years

Eligibility

Inclusion Criteria:

• Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study

• Adequate liver function

• Not Pregnant or nursing

Exclusion Criteria:

• Inadequate renal function

• Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia

• Untreated hypo- or hyperthyroidism

• Prior major organ transplant

Related Conditions & MeSH terms

• Amyloidosis
• TTR-mediated Amyloidosis

Intervention

Drug:
• Revusiran (ALN-TTRSC)

Locations

Country	Name	City	State
Canada	Clinical Trial Site	Calgary	Alberta
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Cleveland	Ohio
United States	Clinical Trial Site	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.	The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study [drug] discontinuation.	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Primary	Serum TTR Levels	Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Secondary	Mortality	Total number of deaths in the study and total number of deaths adjudicated as being related to cardiovascular causes. Deaths were adjudicated by an independent adjudication committee as cardiovascular (CV) or non-CV events.	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Secondary	Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization	Hospitalization events were adjudicated by an independent committee as cardiovascular (CV) or non-CV events	Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Secondary	6-minute Walk Test Performance	Distance in meters walked in 6 minutes	Baseline, Month 6, and Month 12