Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
Phase 2
Completed
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Drug: ALN-TTRSC (revusiran) for subcutaneous administration
- Registration Number
- NCT01981837
- Lead Sponsor
- Alnylam Pharmaceuticals
- 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 available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 26
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ALN-TTRSC (revusiran) ALN-TTRSC (revusiran) for subcutaneous administration -
- Primary Outcome Measures
Name Time Method The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. Up to 63 days
- Secondary Outcome Measures
Name Time Method Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz) Up to 90 days Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels) Up to 90 days
Trial Locations
- Locations (1)
Clinical Trial Site
🇬🇧London, United Kingdom