Basic Information

Gene symbol PCSK9 Synonyms FH3, FHCL3, HCHOLA3, LDLCQ1, NARC-1, NARC1, PC9 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description proprotein convertase subtilisin/kexin type 9

GTO ID GTC1383
Trial ID NCT01350960
Disease Familial Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyASO
Treatment SPC5001
PhasePhase1
Recruitment statusTerminated
TitleA First-in-Human (FIH), Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SPC5001 Administered to Healthy Subjects and Subjects With Familial Hypercholesterolemia (FH)
Year2011
CountryNetherlands
Company sponsorSantaris Pharma A/S
Other ID(s)SPC5001-901|EudraCT 2011-000489-36
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Saline
Administration route subcutaneous injection
Dosage saline
Pts 6
Age Adult, Older_Adult
References PMID: 26261033
Cohort2: SPC5001_dose level 1
Administration route subcutaneous injection
Dosage SPC5001, 0.5 mg/kg, 3 weekly
Pts 6
Age Adult, Older_Adult
References PMID: 26261033
Cohort3: SPC5001_dose level 2
Administration route subcutaneous injection
Dosage SPC5001, 1.5 mg/kg, 3 weekly
Pts 6
Age Adult, Older_Adult
References PMID: 26261033
Cohort4: SPC5001_dose level 3
Administration route subcutaneous injection
Dosage SPC5001, 5.0 mg/kg, 3 weekly
Pts 6
Age Adult, Older_Adult
References PMID: 26261033

Relationship Graph

Overview of Knowledge Graph