TTR-mediated AmyloidosisPhase 2NCT02292186

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

A Phase 2 study testing Revusiran (ALN-TTRSC) in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 5 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 to 90 years
Conditions the study is for: TTR-mediated Amyloidosis
What is being tested?: Revusiran (ALN-TTRSC)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 25 participants
Where: 5 sitesin 3 countries
Study dates: Oct 2014to 22 Feb 2017
Phase: Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 90 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

5 sites in 3 countries.

Country

City, postcode or hospital

5 sites in 3 countries

United States3 sites

Clinical Trial Site
Boston, Massachusetts, 02155
Clinical Trial Site
Cleveland, Ohio, 44195
Clinical Trial Site
New York, New York, 10034

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Canada1 site

Clinical Trial Site
Calgary, Alberta

Other trials in Canada

United Kingdom1 site

Clinical Trial Site
London, UK NW3 2PF

Other trials in United Kingdom

About this study

Official title: A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC

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.

Conditions: TTR-mediated Amyloidosis
Being tested: Revusiran (ALN-TTRSC)
Keywords: Cardiomyopathy, Heart Failure, FAC, Amyloid, Transthyretin, TTR, RNAi therapeutic
Medical subject headings: Cardiomyopathies, Heart Failure, Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Revusiran (ALN-TTRSC)Drug

Study groups in NCT02292186
Study group	Role	What they receive
Revusiran (ALN-TTRSC)	Experimental	Not described Receives: Drug: Revusiran (ALN-TTRSC)

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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
Measured: 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
Total number of deaths in the study and total number of deaths adjudicated as being related to cardiovascular causes. Deaths were adjudicated by an independent adjudication committee as cardiovascular (CV) or non-CV events.
Measured: 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
Hospitalization events were adjudicated by an independent committee as cardiovascular (CV) or non-CV events
Measured: 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
Distance in meters walked in 6 minutes
Measured: Baseline, Month 6, and Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 201422 Feb 2017

First posted17 Nov 2014 (estimated)
Study startOct 2014
Primary completion22 Feb 2017
Study completion22 Feb 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for TTR-mediated AmyloidosisStudies of the same condition, open and under way.
More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02292186
Sponsor's protocol ID: ALN-TTRSC-003
Status last verified: Jul 2020
First submitted: 10 Nov 2014
Last updated: 9 Jul 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No