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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-017383-16 | EudraCT Number |
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The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALN-TTR01 | Active Comparator |
| |
| Sterile Normal Saline (0.9% NaCl) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALN-TTR01 | Drug | Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion |
| |
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation. | Up to 28 Days |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL) | Up to 70 days | |
| Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level) | up to 70 days |
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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 | Le Kremlin-Bicêtre | 94275 | France | |||
| 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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| Sterile Normal Saline (0.9% NaCl) |
| Drug |
Calculated volume to match active comparator |
|
| Porto |
| 4099-001 |
| Portugal |
| Clinical Site | Umeå | SE 901 85 | Sweden |
| Clinical Site | London | SE1 1YR | United Kingdom |