A Phase 1, Randomized, Double-blind, Placebo-controlled, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC in Healthy Volunteers

Alnylam Pharmaceuticals1 site in 1 country85 target enrollmentMarch 2013

ConditionsTTR-mediated Amyloidosis

InterventionsALN-TTRSC (revusiran)Sterile Normal Saline (0.9% NaCl)

DrugsALN-TTRSC (revusiran)Sterile Normal Saline (0.9% NaCl)

Overview

Phase: Phase 1
Intervention: ALN-TTRSC (revusiran)
Conditions: TTR-mediated Amyloidosis
Sponsor: Alnylam Pharmaceuticals
Enrollment: 85
Locations: 1
Primary Endpoint: The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Status: Completed
Last Updated: 10 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

March 2013

End Date

May 2015

Last Updated

10 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

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.

Arms & Interventions

ALN-TTRSC (revusiran)

Intervention: ALN-TTRSC (revusiran)

Sterile Normal Saline (0.9% NaCl)

Intervention: Sterile Normal Saline (0.9% NaCl)

Outcomes

Primary Outcomes

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.

Time Frame: Up to 63 days

Secondary Outcomes

Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC0-last, CL).(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)
Effect of ALN-TTRSC (revusiran) on retinol binding protein (RBP) (Determination of % Lowering of RBP to pretreatment/Baseline Levels).(Up to 90 days)
Effect of ALN-TTRSC (revusiran) on vitamin A (Determination of % Lowering of vitamin A to pretreatment/Baseline Levels).(Up to 90 days)