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The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-TTR02 | Active Comparator |
| |
| Sterile Normal Saline (0.9% NaCl) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-TTR02 | Drug | Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion |
| |
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. | Up to 28 days |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL). | Up to 180 days | |
| Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jared Gollob, MD | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Site | Leeds | LS2 9LH | United Kingdom | |||
| Clinical Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23984729 | Derived | Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760. |
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| ID | Term |
|---|---|
| C000606954 | patisiran |
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| Sterile Normal Saline (0.9% NaCl) |
| Drug |
Calculated volume to match active comparator |
|
| Up to 56 days |
| London |
| NW10 7EW |
| United Kingdom |