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



Status:Completed
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - 80
Updated:1/31/2018
Start Date:December 2013
End Date:January 2015

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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

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.


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.
We found this trial at
4
sites
Cleveland, Ohio 44195
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Boston, Massachusetts 02114
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Boston, MA
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London,
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New York, New York 10032
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New York, NY
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