NCT02292186

Brief Summary

The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2014

Geographic Reach
3 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2014

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

November 10, 2014

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 17, 2014

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 22, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 22, 2017

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

June 15, 2018

Completed
Last Updated

July 9, 2020

Status Verified

July 1, 2020

Enrollment Period

2.4 years

First QC Date

November 10, 2014

Results QC Date

February 22, 2018

Last Update Submit

July 7, 2020

Conditions

TTR-mediated Amyloidosis

Keywords

CardiomyopathyHeart FailureFACAmyloidTransthyretinTTRRNAi therapeutic

Outcome Measures

Primary Outcomes (2)

  • Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.

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

    Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months

  • Serum TTR Levels

    Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR

    Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months

Secondary Outcomes (3)

  • Mortality

    Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months

  • Clinical Effects of Long-term Dosing of ALN-TTRSC on Hospitalization

    Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months

  • 6-minute Walk Test Performance

    Baseline, Month 6, and Month 12

Study Arms (1)

Revusiran (ALN-TTRSC)

EXPERIMENTAL
Drug: Revusiran (ALN-TTRSC)

Interventions

Revusiran (ALN-TTRSC)DRUG
Revusiran (ALN-TTRSC)

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study
  • Adequate liver function
  • Not Pregnant or nursing

You may not qualify if:

  • Inadequate renal function
  • Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia
  • Untreated hypo- or hyperthyroidism
  • Prior major organ transplant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

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

Calgary, Alberta, Canada

Location

Clinical Trial Site

London, UK NW3 2PF, United Kingdom

Location

MeSH Terms

Conditions

CardiomyopathiesHeart FailureAlzheimer Disease

Interventions

revusiran

Condition Hierarchy (Ancestors)

Heart DiseasesCardiovascular DiseasesDementiaBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesTauopathiesNeurodegenerative DiseasesNeurocognitive DisordersMental Disorders

Limitations and Caveats

Safety and efficacy data are difficult to interpret due to the limited numbers of patients completing the study.

Results Point of Contact

Title
Chief Medical Officer
Organization
Alnylam Pharmaceuticals Inc
Clinicaltrials@alnylam.com
866-330-0326

Study Officials

  • Jared Gollob, MD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

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 10, 2014

First Posted

November 17, 2014

Study Start

October 1, 2014

Primary Completion

February 22, 2017

Study Completion

February 22, 2017

Last Updated

July 9, 2020

Results First Posted

June 15, 2018

Record last verified: 2020-07

Locations

US(3)CA(1)GB(1)