Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01981837

Enrollment

Registered

2013-11-13

Start date

2013-12-31

Completion date

2015-01-31

Last updated

2023-03-20

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 pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.

Interventions

DRUGALN-TTRSC (revusiran) for subcutaneous administration

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 80 Years

Healthy volunteers

Inclusion criteria

* TTR cardiac amyloidosis; * Women of child-bearing potential (WOCBP) must have a negative pregnancy test, cannot be breast feeding, and must be willing to use 2 highly effective methods of contraception; * Male subjects agree to use appropriate contraception; * Adequate blood counts, liver, renal and heart function; * Adequate Karnofsky performance status; * Adequate New York Heart Association (NYHA) Classification Score; * Clinically stable on heart medications; * Adequate 6-minute walk test; * Willing to give written informed consent and are willing to comply with the study requirements.

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; * Received an investigational agent other than tafamidis, diflunisal, doxycycline or tauroursodeoxycholic acid, or an investigational device within 30 days prior to first dose of study; * Uncontrolled hypertension, ischemic heart disease or cardiac arrhythmia; * Untreated hypo- or hyperthyroidism; * Prior major organ transplant; * 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, AUC, CL, Vss, Vz)	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

Countries

United Kingdom, United States

Outcome results

None listed

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