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

TTR-mediated Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01981837
• Other study ID #: ALN-TTRSC-002
• Status: Completed
• Phase: Phase 2
• Start date: December 2013
• Est. completion date: January 2015

Study information

• Verified date: March 2018
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: January 2015
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Amyloidosis
• TTR-mediated Amyloidosis

Intervention

Drug:
• ALN-TTRSC (revusiran) for subcutaneous administration

Locations

Country	Name	City	State
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Cleveland	Ohio
United States	Clinical Trial Site	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.		Up to 63 days
Secondary	Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)		Up to 90 days
Secondary	Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)		Up to 90 days