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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02292186
Recruitment Status : Completed
First Posted : November 17, 2014
Results First Posted : June 15, 2018
Last Update Posted : July 9, 2020
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
TTR-mediated Amyloidosis Drug: Revusiran (ALN-TTRSC) Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
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
Study Start Date : October 2014
Actual Primary Completion Date : February 22, 2017
Actual Study Completion Date : February 22, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Revusiran (ALN-TTRSC) Drug: Revusiran (ALN-TTRSC)


Outcome Measures
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Primary Outcome Measures :
  1. Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients. [ Time Frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months ]
    The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study [drug] discontinuation.

  2. Serum TTR Levels [ Time Frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months ]
    Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR


Secondary Outcome Measures :
  1. Mortality [ Time Frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months ]
    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.

  2. Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization [ Time Frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months ]
    Hospitalization events were adjudicated by an independent committee as cardiovascular (CV) or non-CV events

  3. 6-minute Walk Test Performance [ Time Frame: Baseline, Month 6, and Month 12 ]
    Distance in meters walked in 6 minutes


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02292186


Locations
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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
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Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alnylam Pharmaceuticals:
Study Protocol  [PDF] March 25, 2016
Statistical Analysis Plan  [PDF] October 23, 2014

More Information
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02292186    
Other Study ID Numbers: ALN-TTRSC-003
First Posted: November 17, 2014    Key Record Dates
Results First Posted: June 15, 2018
Last Update Posted: July 9, 2020
Last Verified: July 2020
Keywords provided by Alnylam Pharmaceuticals:
Cardiomyopathy
Heart Failure
FAC
Amyloid
 Transthyretin
TTR
RNAi therapeutic
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases