TTR-mediated AmyloidosisPhase 2NCT01981837
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
A Phase 2 study testing ALN-TTRSC (revusiran) for subcutaneous administration in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 4 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- TTR-mediated Amyloidosis
- What is being tested?
- ALN-TTRSC (revusiran) for subcutaneous administration
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Dec 2013to Jan 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 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 Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis
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
- Being tested
- ALN-TTRSC (revusiran) for subcutaneous administration
- Keywords
- RNAi therapeutic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-TTRSC (revusiran) for subcutaneous administrationDrug
| Study group | Role | What they receive |
|---|---|---|
| ALN-TTRSC (revusiran) | Experimental | Not described Receives: Drug: ALN-TTRSC (revusiran) for subcutaneous administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Measured: Up to 63 days
Secondary outcomes (2)
Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)
Measured: Up to 90 days
Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)
Measured: Up to 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2013 (estimated)
- Study startDec 2013
- Primary completionOct 2014
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 26 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
- NCT01981837
- Sponsor's protocol ID
- ALN-TTRSC-002
- Status last verified
- Mar 2018
- First submitted
- 6 Nov 2013
- Last updated
- 20 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01981837
- Acronym / sponsor ID
- ALN-TTRSC-002
- Conditions
- TTR-mediated Amyloidosis
- Keywords
- RNAi therapeutic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2013
- Primary completion
- Oct 2014 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 13 Nov 2013 (estimated)
- Results
- Not posted in the source record
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 is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALN-TTRSC (revusiran) | Experimental | Drug: ALN-TTRSC (revusiran) for subcutaneous administration |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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;
Key exclusion criteria (7 listed)
- 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;
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 |
|---|---|---|
| ALN-TTRSC (revusiran) for subcutaneous administration | Drug | ALN-TTRSC (revusiran) |
Primary outcome
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Time frame: Up to 63 days
2 secondary outcomes
Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC, CL, Vss, Vz)
Time frame: Up to 90 days
Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels)
Time frame: Up to 90 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2023.
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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
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALN-TTRSC (revusiran) for subcutaneous administration in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 4 sites in 2 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 9 inclusion and 7 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 20 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. (time frame: Up to 63 days). 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 20 Mar 2023.