A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers

Alnylam Pharmaceuticals

Status and phase

Completed

Phase 1

Conditions

TTR-mediated Amyloidosis

Treatments

Drug: Sterile Normal Saline (0.9% NaCl)

Drug: ALN-TTRSC (revusiran)

Study type

Interventional

Funder types

Industry

Identifiers

NCT01814839

ALN-TTRSC-001

Details and patient eligibility

About

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC (revusiran) in healthy volunteer subjects.

Enrollment

85 patients

Sex

All

Ages

18 to 55 years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

Body mass index (BMI) must be ≥ 16.5 kg/m2 and ≤ 35.0 kg/m2;
Female subjects must be of non-childbearing potential; e.g., postmenopausal or pre-menopausal with surgical sterilization;
Male subjects agree to use appropriate contraception;
Adequate blood counts, liver and renal function;
Non-smokers for at least 3 months;
Willing to give written informed consent and are willing to comply with the study requirements;
Subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan (cohorts 19 and 20).

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;
History of drug abuse and/or alcohol abuse;
Receiving an investigational agent within 3 months prior to study drug administration;
Considered unfit for the study by the Principal Investigator.