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 GTC1356
Trial ID NCT01148953
Disease Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTR01
PhasePhase1
Recruitment statusCompleted
TitleA Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis
Year2010
CountryFrance|Portugal|Sweden|United Kingdom
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR01-001|2009-017383-16
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort1: ALN-TTR01_dose level 1
Administration route intravenous injection
Dosage ALN-TTR01, 0.01 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort2: ALN-TTR01_dose level 2
Administration route intravenous injection
Dosage ALN-TTR01, 0.03 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort3: ALN-TTR01_dose level 3
Administration route intravenous injection
Dosage ALN-TTR01, 0.1 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort4: ALN-TTR01_dose level 4
Administration route intravenous injection
Dosage ALN-TTR01, 0.2 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort5: ALN-TTR01_dose level 5
Administration route intravenous injection
Dosage ALN-TTR01, 0.4 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort6: ALN-TTR01_dose level 6
Administration route intravenous injection
Dosage ALN-TTR01, 0.7 mg/kg
Pts 3
Age Adult, Older_Adult
References PMID: 23984729
Cohort7: ALN-TTR01_dose level 7
Administration route intravenous injection
Dosage ALN-TTR01, 1.0 mg/kg
Pts 6
Age Adult, Older_Adult
References PMID: 23984729
Cohort8: Sterile Normal Saline
Administration route intravenous injection
Pts 8
Age Adult, Older_Adult
References PMID: 23984729

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