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

March 15, 2023 updated by: Alnylam Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

26

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, UK NW3 2PF
        • Clinical Trial Site
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02155
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10034
        • Clinical Trial Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ALN-TTRSC (revusiran)
Drug: ALN-TTRSC (revusiran) for subcutaneous administration

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)
Time Frame: Up to 90 days
Up to 90 days
Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)
Time Frame: Up to 90 days
Up to 90 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2013

Primary Completion (Actual)

October 1, 2014

Study Completion (Actual)

January 1, 2015

Study Registration Dates

First Submitted

November 6, 2013

First Submitted That Met QC Criteria

November 6, 2013

First Posted (Estimate)

November 13, 2013

Study Record Updates

Last Update Posted (Actual)

March 20, 2023

Last Update Submitted That Met QC Criteria

March 15, 2023

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC-002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on TTR-mediated Amyloidosis

Clinical Trials on ALN-TTRSC (revusiran) for subcutaneous administration

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Spain

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe