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

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

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01814839

Enrollment

Registered

2013-03-20

Start date

2013-03-31

Completion date

2015-05-31

Last updated

2016-02-05

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

Conditions

TTR-mediated Amyloidosis

Keywords

RNAi therapeutic

Brief summary

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

Interventions

DRUGALN-TTRSC (revusiran)

Ascending doses of ALN-TTRSC (revusiran) by subcutaneous (SC) injection

DRUGSterile Normal Saline (0.9% NaCl)

Calculated volume to match active comparator

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 55 Years

Healthy volunteers

Yes

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.

Design outcomes

Primary

Measure	Time frame
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.	Up to 63 days

Secondary

Measure	Time frame
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 vitamin A (Determination of % Lowering of vitamin A 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

Countries

United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026