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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT02292186
First received: November 10, 2014
Last updated: May 23, 2017
Last verified: May 2017
  Purpose
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.

Condition Intervention Phase
TTR-mediated Amyloidosis
Drug: Revusiran (ALN-TTRSC)
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • Safety and tolerability of long-term dosing with revusiran (ALN-TTRSC) as measured by number of adverse events in patients with transthyretin (TTR) cardiac amyloidosis [ Time Frame: Up to 35 days post last dose ]

Secondary Outcome Measures:
  • Assessment of changes from baseline in serum TTR [ Time Frame: From Baseline to Day 1372 ]
  • Mortality [ Time Frame: From Baseline to Day 1344 ]
    Total number of deaths in the study and total number of deaths adjudicated as being related to cardiovascular causes

  • Frequency of cardiovascular-related and heart failure-related hospitalizations [ Time Frame: From Baseline to Day 1344 ]
    The number of times that a subject is hospitalized for cardiovascular- or heart failure- related causes

  • 6-minute walk test performance [ Time Frame: From Baseline to Day 1344 ]
    Change from Baseline to Day 1344 in the total distance walked


Enrollment: 25
Study Start Date: October 2014
Study Completion Date: February 2017
Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Revusiran (ALN-TTRSC) Drug: Revusiran (ALN-TTRSC)

  Eligibility

Ages Eligible for Study:   18 Years to 90 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02292186

Locations
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States, 02155
United States, New York
Clinical Trial Site
New York, New York, United States, 10034
United States, Ohio
Clinical Trial Site
Cleveland, Ohio, United States, 44195
Canada, Alberta
Clinical Trial Site
Calgary, Alberta, Canada
United Kingdom
Clinical Trial Site
London, United Kingdom, UK NW3 2PF
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02292186     History of Changes
Other Study ID Numbers: ALN-TTRSC-003
Study First Received: November 10, 2014
Last Updated: May 23, 2017

Keywords provided by Alnylam Pharmaceuticals:
Cardiomyopathy
Heart Failure
FAC
Amyloid
Transthyretin
TTR
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on May 25, 2017