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 |
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.
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.
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