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

Primary Purpose

TTR-mediated Amyloidosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Revusiran (ALN-TTRSC)
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for TTR-mediated Amyloidosis focused on measuring Cardiomyopathy, Heart Failure, FAC, Amyloid, Transthyretin, TTR, RNAi therapeutic

Eligibility Criteria

18 Years - 90 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Revusiran (ALN-TTRSC)

Arm Description

Outcomes

Primary Outcome Measures

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.
Serum TTR Levels
Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR

Secondary Outcome Measures

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.
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
6-minute Walk Test Performance
Distance in meters walked in 6 minutes

Full Information

First Posted
November 10, 2014
Last Updated
July 7, 2020
Sponsor
Alnylam Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02292186
Brief Title
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2020
Overall Recruitment Status
Completed
Study Start Date
October 2014 (undefined)
Primary Completion Date
February 22, 2017 (Actual)
Study Completion Date
February 22, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
TTR-mediated Amyloidosis
Keywords
Cardiomyopathy, Heart Failure, FAC, Amyloid, Transthyretin, TTR, RNAi therapeutic

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jared Gollob, MD
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02155
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10034
Country
United States
Facility Name
Clinical Trial Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Clinical Trial Site
City
Calgary
State/Province
Alberta
Country
Canada
Facility Name
Clinical Trial Site
City
London
ZIP/Postal Code
UK NW3 2PF
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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