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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-005803-98 | EudraCT Number |
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The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-TTRSC02 | Active Comparator |
| |
| Sterile normal saline 0.9% for SC administration | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-TTRSC02 | Drug | Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) | Day 1 through to Day 314 |
| Measure | Description | Time Frame |
|---|---|---|
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Cmax | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | tmax |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| John Vest, MD, PhD | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | London | 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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| ID | Term |
|---|---|
| D028226 | Amyloidosis, Familial |
| D028227 | Amyloid Neuropathies, Familial |
| D017772 | Amyloid Neuropathies |
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| ID | Term |
|---|---|
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
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| Sterile Normal Saline (0.9% NaCl) |
| Drug |
Calculated volume to match active comparator |
|
| Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | AUC | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | t½ | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | CL/F | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Vss/F | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | Vz/F | Day 1 through to Day 3 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | fe | Day 1 through to Day 314 |
| Profile of pharmacokinetics (PK) of ALN-TTRSC02 | CLR | Day 1 through to Day 314 |
| Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR | 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 | Screening through to Day 314 |
| D009750 | Nutritional and Metabolic Diseases |
| D000686 | Amyloidosis |
| D057165 | Proteostasis Deficiencies |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |