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Report for Clinical Trial NCT03580655

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)

NCT03580655 Advanced Systemic Mastocytosis Aggressive Systemic Mastocytosis Systemic Mastocytosis With an Associated Hematologic Neoplasm Mast Cell Leu Mast Cell Leukemia
MeSH:Mastocytosis Mastocytosis, Systemic Hematologic Neoplasms Leukemia, Mast-Cell
HPO:Hematological neoplasm Leukemia Mastocytosis

1 Interventions

Name: Avapritinib

Description: Avapritinib tablet
Type: Drug
Group Labels: 1

Avapritinib


Primary Outcomes

Measure: Objective response rate (ORR) based on modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) response criteria

Time: 10 Months

Secondary Outcomes

Description: 0 - 80 points (higher value represents worse symptom outcomes)

Measure: Mean Change from Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score

Time: 10 Months

Description: Months

Measure: Time-to-response (TTR)

Time: 10 Months

Description: Months

Measure: Duration of Response (DOR)

Time: 10 Months

Description: Months

Measure: Progression-free Survival (PFS)

Time: 10 Months

Description: Months

Measure: Overall Survival (OS)

Time: 10 Months

Description: percentage

Measure: Changes in bone marrow mast cells

Time: 10 Months

Description: ng/mL

Measure: Change in serum tryptase

Time: 10 Months

Description: percentage

Measure: Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) mutation burden

Time: 10 Months

Description: mL

Measure: Change in liver volume by imaging

Time: 10 Months

Description: mL

Measure: Change in spleen volume by imaging

Time: 10 Months

Measure: Clinical benefit based on modified IWG-MRT-ECNM consensus criteria

Time: 10 Months

Description: 0 - 10 points (higher value represents worse symptom outcomes)

Measure: Change in PGIS

Time: 10 Months

Description: 0 - 100 points (lower value represents worse quality of life)

Measure: Change in EORTC QLQ-C30

Time: 10 Months

Description: CTCAE version 4.0

Measure: Safety of Avapritinib as assessed by incidence of adverse events

Time: 10 Months

Description: h•ng/mL

Measure: Area Under Curve (0 to Tau) for Avapritinib

Time: 4 Months

Purpose: Treatment

Allocation: N/A

Single Group Assignment


There is one SNP

SNPs


1 D816V

Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) mutation burden. --- D816V ---



HPO Nodes


HPO:
HP:0004377: Hematological neoplasm
Genes 347
KRAS PDGFRA IL2RG SRP54 IL2RG NPM1 TET2 FLT3 ITK SMPD1 PRKCD GATA1 KIT RPL11 RUNX1 CALR ATRX TNFRSF1B DCLRE1C SAMD9L EVC2 CD70 BRAF TSR2 SPINK1 RNF43 FASLG ATM CASP10 NRAS BCL10 BUB1 SH2B3 RPS29 CEBPA TP53 CHD7 TINF2 MYSM1 ASXL1 UBE2T CR2 CALR SETBP1 RNASEH2B KLHDC8B MALT1 TINF2 RAF1 STS TERT BCR SRP54 SRP54 TNFSF12 MLH1 RPL18 ELANE DNASE1L3 CBL MAGT1 TYROBP ASXL1 FOXP1 IL7R TCF4 FANCE DNAJC21 RPL15 FANCL KIF11 TP53 COL14A1 MPL HSPA9 IFIH1 SCN11A RPS10 JAK2 DNMT3A CTRC BCL2 LIG4 GINS1 TERC FANCG SF3B1 ELANE NBN NHP2 NUP214 MYC ETV6 CD81 TNFSF12 RNASEH2A BRIP1 BCR BUB1B RPL5 NBN FANCF IGH DNAJC21 TNFRSF1B CCND1 RMRP XRCC2 MSH6 FAS IGH NFKB2 RECQL4 CD28 BUB1B CTLA4 ARHGAP26 RPL35 CBL RAD54L POLE PALB2 GNB1 MDM2 CBFB ADAR ADA RPS17 PARN RPS14 BCR POT1 ABL1 TNFRSF13C FANCE TNFRSF13B ABL1 ICOS TET2 CD27 NFKB1 CALR LIG4 SH3GL1 CD28 PNP F13B USB1 RPL26 RPL31 PTPN11 TP63 RAD51 MPL TET2 PTPN11 ADA NUTM1 STAT3 RAG2 PIGL PRSS1 TET2 NRAS PRF1 FANCA GFI1 ATRX FANCD2 SCN9A TET2 EVC TNFRSF13C BLM RB1 FANCM SH2B3 RTEL1 HAX1 FANCC RAD54B BIRC3 GFI1 RPS15A RPS14 GFI1B RPS19 RPL35A MSH2 TCF4 RFWD3 NF1 TRIP13 PIGA GATA2 ATM SAMD9L DDX41 IGH FANCD2 RASGRP1 CASP10 MLLT10 ADA2 LIG4 MPL RUNX1 BRCA2 CD19 ERCC4 TP53 RMRP RAD51C NUP214 JAK2 SBDS SH2D1A WAS SMARCD2 THPO ZAP70 TREM2 RPS24 KIT JAK2 BLM BRCA1 RNASEH2C MYD88 RFWD3 SAMD9 SLX4 TNFRSF13B SH2B3 GLI1 CHEK2 NPM1 TET2 DKC1 GATA2 MS4A1 ICOS CALR RPS19 DKC1 LIG4 SRSF2 CR2 GATA2 RAG1 PRKCD BCL6 TREX1 RPS27 RPS26 FANCG GBA FANCI LYST SAMHD1 TET2 GATA2 LIG4 PICALM DNAJC21 NTHL1 JAK2 BUB3 TCIRG1 RPS28 FANCA MPL DYNC2LI1 DNMT3A AAGAB FANCC RARA PIK3CA TERC PGM3 MAD2L2 MYD88 MLH1 FANCB MSH6 KRAS TERT RPS7 RUNX1 MCM4 BCL10 XIAP LPP BRCA2 CD19 BRD4 PIK3R1 PTEN NAGS NBEAL2 F13A1 CTC1 SBDS HLA-DRB1 WRAP53 TAL1 EFL1 NOP10 JAK2 FLT3 CEP57 KRAS APC PMS2 WIPF1 JAK2 TERC CTLA4 GATA1 NSUN2 BCL10 XRCC4 NBN SF3B1 FAS PDGFRB RHOH MSH2 RECQL4 TAL2 PIGL RPL27 EP300 CHIC2 CCND1 TET2 GATA2 NUMA1 TERT MPL THPO CREBBP SCN10A CDKN2A ERBB3 SRP72 TERT
Protein Mutations 0
SNP 0