Basic Information

Gene symbol TTR Synonyms ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description transthyretin

GTO ID GTC1417
Trial ID NCT01559077
Disease Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTR02|ONPATTRO|patisiran
Location approved US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
PhasePhase1
Recruitment statusCompleted
TitleA Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers
Year2012
CountryUnited Kingdom
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR02-001
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort1: ALN-TTR02_dose level 1
Administration route intravenous infusion
Dosage ALN-TTR02, 0.01 mg/kg
Pts 3
Age Adult
References PMID: 23984729
Cohort2: ALN-TTR02_dose level 2
Administration route intravenous infusion
Dosage ALN-TTR02, 0.05 mg/kg
Pts 3
Age Adult
References PMID: 23984729
Cohort3: ALN-TTR02_dose level 3
Administration route intravenous infusion
Dosage ALN-TTR02, 0.15 mg/kg
Pts 3
Age Adult
References PMID: 23984729
Cohort4: ALN-TTR02_dose level 4
Administration route intravenous infusion
Dosage ALN-TTR02, 0.3 mg/kg
Pts 3
Age Adult
References PMID: 23984729
Cohort5: ALN-TTR02_dose level 5
Administration route intravenous infusion
Dosage ALN-TTR02, 0.5 mg/kg
Pts 1
Age Adult
References PMID: 23984729
Cohort6: ALN-TTR02_dose level 6
Administration route intravenous infusion
Dosage ALN-TTR02, 0.4 mg/kg
Pts 6
Age Adult
References PMID: 23984729
Cohort7: Sterile Normal Saline
Administration route intravenous infusion
Dosage Calculated volume to match active comparator
Pts 4
Age Adult
References PMID: 23984729

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