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

NCT01981837 | Completed Phase 2

• 1 other identifier: ALN-TTRSC-002
• Study Type: interventional
• Target: 26
• Locations: 2 countries
• Sites: 4

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.

Conditions

TTR-mediated Amyloidosis

Keywords

RNAi therapeutic

Outcome Measures

Primary Outcomes (1)

• The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.

  Up to 63 days

Secondary Outcomes (2)

• 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

Study Arms (1)

ALN-TTRSC (revusiran)

EXPERIMENTAL

Drug: ALN-TTRSC (revusiran) for subcutaneous administration

Interventions

ALN-TTRSC (revusiran) for subcutaneous administration DRUG

ALN-TTRSC (revusiran)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (4)

Clinical Trial Site

Boston, Massachusetts, 02155, United States

Location

Clinical Trial Site

New York, New York, 10034, United States

Location

Clinical Trial Site

Cleveland, Ohio, 44195, United States

Location

Clinical Trial Site

London, UK NW3 2PF, United Kingdom

Location

MeSH Terms

Interventions

revusiran

Study Officials

• Jared Gollob, MD

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2013
• First Posted: November 13, 2013
• Study Start: December 1, 2013
• Primary Completion: October 1, 2014
• Study Completion: January 1, 2015
• Last Updated: March 20, 2023

Record last verified: 2018-03

Locations

GB (1); US (3)