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.
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
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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.
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
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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jared Gollob, MD, Study director, Alnylam Pharmaceuticals
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
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02292186
- Acronym / sponsor ID
- ALN-TTRSC-003
- Conditions
- TTR-mediated Amyloidosis
- Keywords
- Cardiomyopathy, Heart Failure, FAC, Amyloid, Transthyretin, TTR, RNAi therapeutic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- 22 Feb 2017 (actual)
- Study completion
- 22 Feb 2017 (actual)
- First posted
- 17 Nov 2014 (estimated)
- Results
- Posted 15 Jun 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Revusiran (ALN-TTRSC) | Experimental | Drug: Revusiran (ALN-TTRSC) |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Subjects who previously received and tolerated revusiran (ALN-TTRSC) in the ALN-TTRSC-002 study
- Adequate liver function
- Not Pregnant or nursing
Key exclusion criteria (4 listed)
- Inadequate renal function
- Uncontrolled hypertension, ischemic heart disease or clinically significant cardiac arrhythmia
- Untreated hypo- or hyperthyroidism
- Prior major organ transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Revusiran (ALN-TTRSC) | Drug | Revusiran (ALN-TTRSC) |
Primary outcome
Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients.
Time frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study [drug] discontinuation.
Serum TTR Levels
Time frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months
Pharmacodynamic (PD) effect of long-term dosing of ALN-TTRSC on serum levels of TTR
3 secondary outcomes
Mortality
Time frame: 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
Time frame: 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
Time frame: Baseline, Month 6, and Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jared Gollob, MD study director
Alnylam Pharmaceuticals
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Revusiran (ALN-TTRSC) in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Jun 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients. (time frame: Day 0 up to 90 days post modified early termination visit (end of study); Mean duration of collection was 16 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2020.