A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers

Alnylam Pharmaceuticals1 site in 1 country17 target enrollmentMarch 2012

ConditionsTTR-mediated Amyloidosis

InterventionsALN-TTR02 Sterile Normal Saline (0.9% NaCl)

DrugsALN-TTR02 Sterile Normal Saline (0.9% NaCl)

Overview

Phase: Phase 1
Intervention: ALN-TTR02
Conditions: TTR-mediated Amyloidosis
Sponsor: Alnylam Pharmaceuticals
Enrollment: 17
Locations: 1
Primary Endpoint: The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Status: Completed
Last Updated: 13 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 a single dose of ALN-TTR02 in healthy volunteer subjects.

Registry

clinicaltrials.gov

Start Date

March 2012

End Date

November 2012

Last Updated

13 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
•Non-smokers for at least 3 months preceding screening;
•Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
•Males agree to use appropriate contraception;
•Medical history must be verified by either a personal physician or medical practitioner as appropriate;
•Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion Criteria

•Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
•Multiple drug allergies or know sensitivity to oligonucleotide
•History of drug abuse and/or alcohol abuse
•Receiving an investigational agent within 3 months prior to study drug administration
•Subjects with safety laboratory test results deemed clinical significant by the Investigator;
•Received prescription drugs within 4 weeks of first dosing
•Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
•Subjects who have used prescription drugs within 4 weeks of first dosing
•Considered unfit for the study by the Principal Investigator
•Employee or family member of the sponsor or the clinical study site personnel

Arms & Interventions

ALN-TTR02

Intervention: ALN-TTR02

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 28 days

Secondary Outcomes

Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).(Up to 180 days)
Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)(Up to 56 days)