Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)Phase 1NCT02797847
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
A Phase 1 study testing ALN-TTRSC02 and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin-mediated Amyloidosis (ATTR Amyloidosis), run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
Trial summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
- What is being tested?
- ALN-TTRSC02 and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 7 Jun 2016to 12 Jan 2018
- Phase
- Phase 1
- 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male and female subjects, age 18 to 65 years, inclusive.
- Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening.
- No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator.
- Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent.
- For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years.
Who cannot join
- Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders.
- Active serious mental illness or psychiatric disorder.
- Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant.
- Known history of allergic reaction to an oligonucleotide or GalNAc.
- History of intolerance to subcutaneous injection.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Single-Blind, Placebo Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Being tested
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Keywords
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Amyloidosis, RNAi therapeutic
- Medical subject headings
- Amyloidosis, Familial, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-TTRSC02Drug
Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)Drug
Calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| ALN-TTRSC02 | Active comparator | Not described Receives: Drug: ALN-TTRSC02 |
| Sterile normal saline 0.9% for SC administration | Placebo comparator | Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)
Measured: Day 1 through to Day 314
Secondary outcomes (11)
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Cmax
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
tmax
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
AUC
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
t½
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
CL/F
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Vss/F
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Vz/F
Measured: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
fe
Measured: Day 1 through to Day 314
Profile of pharmacokinetics (PK) of ALN-TTRSC02
CLR
Measured: Day 1 through to Day 314
Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR
Measured: Day 1 through to Day 314
Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A
Measured: Screening through to Day 314
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2016 (estimated)
- Study start7 Jun 2016
- Primary completion12 Jan 2018
- Study completion12 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Vest, MD, PhD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02797847
- Sponsor's protocol ID
- ALN-TTRSC02-001
- Other IDs
- 2015-005803-98, 2015
- Status last verified
- Sep 2018
- First submitted
- 18 May 2016
- Last updated
- 21 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Publications
Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
PubMed 41388232
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Single-Blind, Placebo Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy Subjects
- ClinicalTrials.gov ID
- NCT02797847
- Acronym / sponsor ID
- ALN-TTRSC02-001
- Keywords
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Amyloidosis, RNAi therapeutic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2016 (actual)
- Primary completion
- 12 Jan 2018 (actual)
- Study completion
- 12 Jan 2018 (actual)
- First posted
- 14 Jun 2016 (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 safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-TTRSC02 | Active comparator | Drug: ALN-TTRSC02 |
| Sterile normal saline 0.9% for SC administration | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male and female subjects, age 18 to 65 years, inclusive.
- Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening.
- No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator.
- Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent.
- For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years.
Key exclusion criteria (5 listed)
- Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders.
- Active serious mental illness or psychiatric disorder.
- Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant.
- Known history of allergic reaction to an oligonucleotide or GalNAc.
- History of intolerance to subcutaneous injection.
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-TTRSC02Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection | Drug | ALN-TTRSC02 |
| Sterile Normal Saline (0.9% NaCl)Calculated volume to match active comparator | Drug | Sterile normal saline 0.9% for SC administration |
Primary outcome
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)
Time frame: Day 1 through to Day 314
11 secondary outcomes
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 3
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 314
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Time frame: Day 1 through to Day 314
Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR
Time frame: Day 1 through to Day 314
Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A
Time frame: Screening through to Day 314
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 21 Sep 2018.
- Derived from this study: PubMed 41388232Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.
- Individual participant dataSharing planned: Undecided
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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.
John Vest, MD, PhD study director
Alnylam Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-TTRSC02 and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin-mediated Amyloidosis (ATTR Amyloidosis), run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
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 5 inclusion and 5 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 21 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) (time frame: Day 1 through to Day 314). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02797847, which also lists 1 linked publication.
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 21 Sep 2018.