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The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-TTRSC04 | Experimental | Participants will be administered a single dose of ALN-TTRSC04. |
|
| Placebo | Placebo Comparator | Participants will be administered a single dose of placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-TTRSC04 | Drug | ALN-TTRSC04 will be administered by subcutaneous (SC) injection. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of Adverse Events | Up to 20 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Serum Transthyretin (TTR) Levels Over Time | Up to 18 months | |
| Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04 | Day 1 up to Day 4 | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | London | SE1 1YR | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41388232 | Derived | 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. |
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| Placebo |
| Drug |
Placebo will be administered by SC injection. |
|
| Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04 |
| Day 1 up to Day 4 |
| Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04 | Day 1 up to Day 4 |
| Fraction of ALN-TTRSC04 Excreted in the Urine (fe) | Up to Day 2 |