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Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01981837
First Posted: November 13, 2013
Last Update Posted: February 5, 2016
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.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
  Purpose
The purpose of this study is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.

Condition Intervention Phase
TTR-mediated Amyloidosis Drug: ALN-TTRSC (revusiran) for subcutaneous administration Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. [ Time Frame: Up to 63 days ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz) [ Time Frame: Up to 90 days ]
  • Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels) [ Time Frame: Up to 90 days ]

Enrollment: 26
Study Start Date: December 2013
Study Completion Date: January 2015
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ALN-TTRSC (revusiran) Drug: ALN-TTRSC (revusiran) for subcutaneous administration

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • TTR cardiac amyloidosis;
  • Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception;
  • Male subjects agree to use appropriate contraception;
  • Adequate blood counts, liver, renal and heart function;
  • Adequate Karnofsky performance status;
  • Adequate New York Heart Association (NYHA) Classification Score;
  • Clinically stable on heart medications;
  • Adequate 6-minute walk test;
  • Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion Criteria:

  • Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
  • Subjects with a history of multiple drug allergies or intolerance to SC injection;
  • Received an investigational agent other than tafamidis, diflunisal, doxycycline or tauroursodeoxycholic acid, or an investigational device within 30 days prior to first dose of study;
  • Uncontrolled hypertension, ischemic heart disease or cardiac arrhythmia;
  • Untreated hypo- or hyperthyroidism;
  • Prior major organ transplant;
  • Considered unfit for the study by the Principal Investigator.
  Contacts and Locations
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): NCT01981837


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
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: NCT01981837     History of Changes
Other Study ID Numbers: ALN-TTRSC-002
First Submitted: November 6, 2013
First Posted: November 13, 2013
Last Update Posted: February 5, 2016
Last Verified: February 2016

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases