Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

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

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01559077

Enrollment

Registered

2012-03-21

Start date

2012-03-31

Completion date

2012-11-30

Last updated

2013-01-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

TTR-mediated Amyloidosis

Keywords

ATTR

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.

Interventions

DRUGALN-TTR02

Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion

DRUGSterile Normal Saline (0.9% NaCl)

Calculated volume to match active comparator

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

ALL

Age

18 Years to 45 Years

Healthy volunteers

Yes

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

Design outcomes

Primary

Measure	Time frame
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.	Up to 28 days

Secondary

Measure	Time frame
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

Countries

United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 10, 2026