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 GTC0147
Trial ID NCT01617967
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
PhasePhase2
Recruitment statusCompleted
TitleA Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis
Year2012
CountryBrazil|France|Germany|Portugal|Spain|Sweden|United States
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR02-002|2012-000467-24
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort1: Patisiran_dose level 1_Q4W
Administration route intravenous infusion
Dosage patisiran, 0.010 mg/kg, every four weeks (Q4W)
Pts 4
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:1 25.0%; At Least 1 SAE:0 0.0%; Study Drug Discontinuation For Any Reason:1 25.0%
Adverse reactions No serious clinical adverse events
References PMID: 26338094
Cohort2: Patisiran_dose level 2_Q4W
Administration route intravenous infusion
Dosage patisiran, 0.050 mg/kg, every four weeks (Q4W)
Pts 3
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:3 100.0%; At Least 1 SAE:0 0.0%; Study Drug Discontinuation For Any Reason:0 0.0%
Adverse reactions No serious clinical adverse events
References PMID: 26338094
Cohort3: Patisiran_dose level 3_Q4W
Administration route intravenous infusion
Dosage patisiran, 0.150 mg/kg, every four weeks (Q4W)
Pts 3
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:2 66.7%; At Least 1 SAE:0 0.0%; Study Drug Discontinuation For Any Reason:0 0.0%
Adverse reactions No serious clinical adverse events
References PMID: 26338094
Cohort4: Patisiran_dose level 4_Q4W
Administration route intravenous infusion
Dosage patisiran, 0.300 mg/kg, every four weeks (Q4W)
Pts 7
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:7 100.0%; At Least 1 SAE:1 14.3%; Study Drug Discontinuation For Any Reason:0 0.0%
Adverse reactions 0/7(All-cause mortality); 1/7(Infections and infestations)
References PMID: 26338094
Cohort5: Patisiran_dose level 4_Q3W
Administration route intravenous infusion
Dosage patisiran, 0.300 mg/kg, every three weeks (Q3W)
Pts 3
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:3 100.0%; At Least 1 SAE:0 0.0%; Study Drug Discontinuation For Any Reason:0 0.0%
Adverse reactions No serious clinical adverse events
References PMID: 26338094
Cohort6: Patisiran_dose level 4_Q3W_alternative premedication regimen
Administration route intravenous infusion
Dosage patisiran, 0.300 mg/kg, every three weeks (Q3W); alternative premedication regimen
Pts 9
Age Adult, Older_Adult
Outcome Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At Least 1 AE Measure Type: Count of Participants:7 77.8%; At Least 1 SAE:1 11.1%; Study Drug Discontinuation For Any Reason:1 11.1%
Adverse reactions 0/9(All-cause mortality); 1/9(Gastrointestinal disorders; Infections and infestations)
References PMID: 26338094

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