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

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ClinicalTrials.gov Identifier: NCT01981837
Recruitment Status : Completed
First Posted : November 13, 2013
Last Update Posted : January 29, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 6, 2013
First Posted Date  ICMJE November 13, 2013
Last Update Posted Date January 29, 2018
Study Start Date  ICMJE December 2013
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 6, 2013)
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. [ Time Frame: Up to 63 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01981837 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 25, 2015)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 6, 2013)
  • Pharmacokinetics (PK) of ALN-TTRSC (Cmax, tmax, t1/2, AUC, CL, Vss, Vz) [ Time Frame: Up to 90 days ]
  • Effect of ALN-TTRSC on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels) [ Time Frame: Up to 90 days ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE TTR-mediated Amyloidosis
Intervention  ICMJE Drug: ALN-TTRSC (revusiran) for subcutaneous administration
Study Arms  ICMJE Experimental: ALN-TTRSC (revusiran)
Intervention: Drug: ALN-TTRSC (revusiran) for subcutaneous administration
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 4, 2016)
26
Original Estimated Enrollment  ICMJE
 (submitted: November 6, 2013)
15
Actual Study Completion Date  ICMJE January 2015
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01981837
Other Study ID Numbers  ICMJE ALN-TTRSC-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP