Transthyretin-Mediated AmyloidosisPhase 1NCT05661916
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
A Phase 1 study testing ALN-TTRSC04 in people with Transthyretin-Mediated Amyloidosis, run by Alnylam Pharmaceuticals at 1 site in United Kingdom.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04. 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
- What is being tested?
- ALN-TTRSC04
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 96 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 16 Jan 2023to 30 Sep 2025
- Phase
- Phase 1
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
- Body mass index (BMI) ≥18.0 kg/m^2 and ≤30 kg/m^2 assessed at Screening.
Who cannot join
- Has an estimated glomerular filtration rate (eGFR) <90 mL/min/1.73m^2 at screening.
- Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin above the upper limit of normal (ULN).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Randomized, Double-Blind, Placebo Controlled, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcutaneously Administered ALN-TTRSC04 in Healthy Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
- Conditions
- Transthyretin-Mediated Amyloidosis
- Being tested
- ALN-TTRSC04, Placebo
- Keywords
- ATTR amyloidosis, siRNA
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-TTRSC04Drug
ALN-TTRSC04 will be administered by subcutaneous (SC) injection.
PlaceboDrug
Placebo will be administered by SC injection.
| Study group | Role | What they receive |
|---|---|---|
| ALN-TTRSC04 | Experimental | Participants will be administered a single dose of ALN-TTRSC04. Receives: Drug: ALN-TTRSC04 |
| Placebo | Placebo comparator | Participants will be administered a single dose of placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events
Measured: Up to 20 months
Secondary outcomes (5)
Change from Baseline in Serum Transthyretin (TTR) Levels Over Time
Measured: Up to 18 months
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04
Measured: Day 1 up to Day 4
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04
Measured: Day 1 up to Day 4
Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04
Measured: Day 1 up to Day 4
Fraction of ALN-TTRSC04 Excreted in the Urine (fe)
Measured: Up to Day 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2022
- Study start16 Jan 2023
- Primary completion30 Sep 2025
- Study completion30 Sep 2025
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 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05661916
- Sponsor's protocol ID
- ALN-TTRSC04-001
- Status last verified
- Feb 2026
- First submitted
- 15 Dec 2022
- Last updated
- 17 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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