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Report for Clinical Trial NCT02730884

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Phase II Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia

The goal of this clinical research study is to learn if rigosertib can help to control MF in patients with anemia. The safety of this drug will also be studied. This is an investigational study. Rigosertib is not FDA-approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work. Up to 35 participants will be enrolled in this study. All will be enrolled at MD Anderson.

NCT02730884 Leukemia Myelofibrosis Anemia Splenomegaly
MeSH:Anemia Primary Myelofibrosis Splenomegaly
HPO:Anemia Splenomegaly

2 Interventions

Name: Rigosertib

Description: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon.
Type: Drug
Group Labels: 1

Rigosertib

Name: Questionnaire

Description: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Type: Behavioral
Group Labels: 1

Rigosertib


Primary Outcomes

Description: Spleen response defined as ≥ 35% spleen volume reduction from Baseline, which must be confirmed by MRI or CT measurement per revised International Working Group for Myelofibrosis Research and Treatment (IWG MRT) response criteria.

Measure: Change in Spleen Volume

Time: Baseline and 48 weeks

Description: Anemia response defined as the proportion of transfusion-independent patients with Hgb increase of at least 2 g/dL from Baseline or the proportion of transfusion-dependent patients becoming transfusion independent for at least 12 weeks as defined in 2013 International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria.

Measure: Change in Anemia Response

Time: Baseline and 48 weeks

Secondary Outcomes

Description: Symptoms response defined as the proportion of patients achieving ≥ 50% reduction in the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at any time before Week 48.

Measure: Symptoms Response

Time: 48 weeks

Purpose: Treatment

Allocation: N/A

Single Group Assignment


There is one SNP

SNPs


1 V617F

Measurement of JAK2 V617F allele burden in BM samples, if not done within 6 months prior to Screening, must be provided with the Screening BM biopsy/aspirate report (patients are eligible regardless of JAK2 mutation status); 3. Anemia or RBC-transfusion dependence defined as follows: a) Anemia: defined for the purpose of this protocol as 1) a hemoglobin level <10 g/L on every determination over 84 days before study-entry, without RBC-transfusions, or 2) a hemoglobin level <10 g/L on a patient that is receiving RBC-transfusions periodically but not meeting criteria for transfusion-dependent patient as defined below. --- V617F ---



HPO Nodes


HPO:
HP:0001903: Anemia
Genes 730
SRD5A3 NDUFS7 CTC1 KLF1 TBXAS1 SRP54 IL2RG TET2 RPS27 AMMECR1 SLC25A38 MLX CD40LG ERCC6L2 RPS24 YARS2 CFI DCLRE1C SCARB2 ALX4 UBE2T MPL TSR2 EXT2 MUC1 XRCC4 VPS33A FAM111A EFL1 WT1 FASLG TSR2 PNPO RPS29 SDHC BCL10 LAMC2 OCRL CHD7 TINF2 FOXP3 NT5C3A ENG PRKCD CR2 PLEKHM1 CASK IRAK1 DAXX MALT1 PKLR GBA TINF2 PSMB8 ZBTB20 SLC4A1 WFS1 NHEJ1 TACO1 RPL35A TCIRG1 FLI1 BMPR1A TNFSF12 CFI BMPR1A SMAD4 LAT ACVR1 COX10 ALAS2 MTRR FOXP1 LYRM7 DHFR TERT DNAJC21 RPL15 HBA1 CTSK KLF1 PGK1 HBB HBB CLCNKB RPS10 MTHFD1 FASTKD2 G6PC3 AASS BRCA1 ANK1 UROS EPB42 TEK FASLG SBDS NHP2 MYSM1 CD81 TNFSF12 RHAG CFH SMAD4 HBB LRBA NDUFS3 STEAP3 CDCA7 DNAJC21 CP DCLRE1C HMGCL RMRP XRCC2 RAG1 PGK1 F8 HELLPAR NFKB2 RECQL4 NPHP4 EPO RHAG COX6B1 REN COL7A1 SPTB RPL35 CD46 PALB2 RAG2 NDUFB8 SRD5A3 FTCD FCGR2A COX8A HPRT1 PTF1A WAS TBCE SPTA1 KDM6A FANCE ICOS SLC11A2 CLPX NFKB1 FCGR2B CA2 KIF23 HLA-DRB1 FAS ALAD RPL26 RAD51 EWSR1 MMAA SLC4A1 CAT NDUFS2 STAT3 SLC2A1 ADA2 EPB41 C1R APOA1 GBA ATRX FANCD2 TET2 UMPS SLCO2A1 ATP11C ACVRL1 KRT14 RTEL1 RPL5 ITGB3 GLA TMEM67 FANCC HBA2 DNMT3B HBA1 LAMA3 TNFSF11 BIRC3 SDHA RPS14 IFNG HAVCR2 IRF2BP2 RPS19 RPL35A PEPD TCIRG1 SPTA1 ACVRL1 GLA PIGA FANCL YARS2 STK11 NDUFA12 GPI SNX10 ECHS1 THRA RPL27 APC NDUFV2 ACD BCOR MTRR GBA ALAS2 ERCC4 PIEZO1 RPL26 HBG1 MVK AK1 STAT1 MTFMT CFB SBDS WAS NFKB1 AK2 IFT140 PFKM MMACHC RPS24 RNF113A HMGCL UMPS SLC4A1 HBB GATA1 BRCA1 TRNS1 PARN ZBTB24 STIM1 RFWD3 KIF15 PCCA HBA1 NDUFS2 ITGB4 PSMB4 DNASE1 HBA2 ABCD3 ALDOA RPS28 TET2 COL7A1 ACTN4 SMPD1 RPS17 ABCB7 ABCA1 ZBTB16 NUMA1 TINF2 DKC1 BMPR1A LIG4 RPS7 AGGF1 NDUFAF6 RAG1 UROS KCNN4 IREB2 NPHP1 RPL15 SLC2A1 FMO3 RPS27 RPS26 KIT ALPL HYOU1 STAT5B HPGD GBA C1QA TGFB1 SLX4 RAG2 TERC BMPR1A MMUT KCNN4 HMOX1 FANCB LAMA3 RPS28 SLC19A3 FANCA MPL STIM1 ELANE FANCC CD46 PET100 PGM3 SLC4A1 MAD2L2 ERCC2 EPHB4 KRAS FOXP3 RPS7 MMUT TGFB1 CD19 HBG2 KIT SARS2 SBDS WRAP53 GCLC COX14 EFL1 COX4I2 ORAI1 STING1 NABP1 CRIPT PUS1 CA2 FARS2 ERCC3 SLC19A2 WIPF1 SLC25A13 TERC DKC1 NSUN2 TRNN PHKG2 ETV6 NBN NDUFV1 STAT3 SP110 UBR1 PUS1 PRF1 RARA PHGDH IL2RB FERMT3 ATP7B RPL27 PET100 LCAT LAMB3 C3 COL7A1 HLA-B NLRC4 ENG CD59 COX15 NDUFA10 TERT TF TNFAIP3 LPIN2 MECOM PIGT DNAJC19 NDUFS1 PRF1 DNAJC19 GALT ITK PTEN PRKCD LIPA ABCD4 IDH1 STK11 RPL11 ATRX LPIN2 GATA1 NPM1 TPP2 SAMD9L ALAS2 KIF1B CD3G NDUFAF3 EPB41 ABCG8 SLC12A3 HBB POLG PRDX1 WFS1 NRAS CISD2 GLRX5 PRKAR1A LAMC2 COA8 OSTM1 RPS29 ATRX SCO2 NLRP3 SLC46A1 DGKE FIP1L1 MYSM1 UBE2T CPOX SLC29A3 LAMB3 TRNT1 TACO1 COL7A1 HBD SPTB NDUFA9 MTR PML GSS SMAD4 TRNT1 TBL1XR1 HBA1 SRP54 SHPK RPL18 SMARCAL1 SPTA1 ISCU DNM1L ELANE MARS1 IDH2 RPS26 GNA14 CAD IL7R COX20 GREM1 FANCE MMP1 HBA1 NDUFAF2 FANCL RFX5 NPHP1 ATP7B ATRX HBB HSPA9 CLCN7 JAK2 TPI1 MPIG6B SLC40A1 PSAP FOXRED1 LIG4 PHGDH SURF1 TERC FANCG SF3B1 KMT2D NOD2 BRIP1 SLC19A2 ADAMTS13 HBB RPL5 GATA1 NBN FANCF PDGFRA PSMB9 ALG8 FECH CP GYPC TALDO1 ABCB7 FAS PTH1R CLCN7 PCCB FAM111A HAMP CDAN1 PGM3 STIM1 DBH LMBRD1 ADA HBG2 GTF2E2 SMAD4 RPS17 STAT1 PARN RPS14 RFXANK RHAG TNFRSF13C TNFRSF13B STX11 CALR UROD ACAD8 NLRP3 PNP USB1 SEC23B RPL31 CIITA HBG1 RFXAP ADA LARS1 RAG2 RMRP COL4A1 FDX2 COQ2 PFKM COG1 CLCN7 BTK FANCA AMN NDUFA13 TF SEC61A1 NDUFS4 F2 SLC4A1 STEAP3 CASP10 ATRX GTF2H5 FANCM TARS1 KCNE1 RTEL1 SLC46A1 TNFSF11 ABCA1 CD55 PIEZO1 RBM8A ITGA2B CTLA4 PNPO SLC7A7 KCNQ1 RPS15A HELLS PLEC NDUFAF5 RPS10 SDHB ELMO2 HPRT1 HBB ANK1 MPLKIP ITGB4 RAG1 SAMD9L DDX41 IGH RAG1 SPTA1 IRX5 FANCD2 SDHA GDF2 COA8 GBA HBB TFRC RASGRP1 CASP10 TCIRG1 ADA2 NHLRC2 CASR COL7A1 PDHA1 BRCA2 PNP NPHP4 TNFRSF11A STAT4 RMRP PSMB8 RAD51C PCNT PLEC HBA2 HLA-B SH2D1A EPB42 SMARCD2 LARS2 ZAP70 RPL11 C15ORF41 SPTA1 IL2RA TCN2 PTPN22 SMARCAL1 TYMP MYD88 TFR2 GPX1 TREX1 SAMD9 BTNL2 PACS2 NDUFA4 SLX4 OPA1 GCLC NPM1 GATA1 COG6 IKZF1 PHKA2 ADAR SLC7A7 DKC1 NLRP1 MS4A1 VPS45 CFHR1 SCO1 RPS19 NDUFA2 PRKCD TMPRSS6 PLEKHM1 TP53 AIRE BPGM GATA1 FANCG RPS15A FANCI LYST TET2 TNFRSF4 CFHR3 MMP1 HBB SLC4A1 COL17A1 HBA2 LYST SNX10 HBB RRM2B CTLA4 PKLR IL12B PHF21A FERMT1 FANCB NDUFS8 SFXN4 HBB-LCR TERT GATA1 PRKACG XIAP SPP1 CUBN SPTB GSS CTC1 CCND1 TTC7A TTC7A NOP10 FARSB LIPT1 ABCB6 UNC13D IFNGR1 TRNW MMAB GATA1 PLA2G4A FAS HBB ANK1 CFH CLCN7 VPS33A SURF1 CBLIF TALDO1 AGXT HBA2 MMADHC HK1 GP1BA MAD2L2 ENG FECH G6PD TBXAS1 THBD TERT ERBB3
Protein Mutations 4
C282Y C677T H63D V617F
HP:0001744: Splenomegaly
Genes 481
MEFV PDGFRA KLF1 IL2RG GPC4 ABCB4 CD40LG TCF4 B2M TNFRSF1B TTC37 DCLRE1C SCARB2 BSCL2 LYZ NEU1 SOX10 PSAP IDUA HSD3B7 IL12A FASLG ERCC6 NOTCH1 DPM1 ATP8B1 CHD7 TINF2 FOXP3 PRKCD CR2 LACC1 ABCB11 RNU4ATAC PLEKHM1 CASK COG7 GBA MST1 PKLR GBA PSMB8 GPIHBP1 IL12A-AS1 AGA ERAP1 GLIS3 TNFSF12 GPC4 LAT DNASE1L3 DOLK LCAT TBX1 PIEZO1 CTSK KLF1 HBB AKT1 HFE G6PC3 ANK1 IRF8 UROS EPB42 PIK3CD FASLG UFD1 NHP2 BSCL2 CD81 TNFSF12 RHAG HBB DCDC2 NCF2 STEAP3 IGH TNFRSF1B DCLRE1C RMRP FOS RAG1 SLC29A3 NFKB2 CASR SMPD1 JMJD1C SPTB RAG2 FAT4 DGUOK RPGRIP1L MEFV ABL1 ICOS INPP5E NFKB1 CA2 SCYL1 FAS PTEN NCF1 ARSB MKS1 ALMS1 GALE MPL SLC4A1 F5 ABCA1 SEC24C ADA2 EPB41 APOA1 GBA ADAMTS3 TET2 LBR UMPS SLCO2A1 PIK3CA KCNH1 SH2B3 IDUA CAV1 TNFSF11 HAVCR2 PEPD ASAH1 ATP6V1B2 TCIRG1 SPTA1 STAT4 LPL GNPTAB PHKG2 IDUA IL1RN BMP2 GPI SNX10 AKR1D1 GBA AKT1 CFAP410 MPL COMT MAN2B1 DGUOK CD19 MVK GBA ALAS2 PIEZO1 MVK JAK2 VPS13A NLRP12 PTEN GBA GBA NPC1 CTNS SLC4A1 SH2B3 HBB TNFRSF1A GATA1 SH2B3 LIPA PSMB4 HBA2 ABCD3 ALDOA GATA2 JAK2 SMPD1 ICOS CALR SUMF1 DKC1 FUCA1 LIG4 RAG1 UROS KCNN4 PTEN BCL6 GPC3 APOC2 SLC2A1 FMO3 CAV1 INPPL1 HGSNAT HPGD GBA TGFB1 RAB27A JAK2 KCNN4 CYBB RREB1 RPGRIP1L CLDN1 MPL DYNC2LI1 HJV CCR1 PEX2 PEPD KRAS RUNX1 MCM4 MMUT COG4 HLA-DRB1 PPARG CD19 HBG2 HLA-DRB1 WRAP53 OTC APOA1 GP1BB SLC17A5 COX4I2 GPD1 TREX1 ATP6AP1 DDRGK1 TERC CTLA4 PHKG2 CYBC1 LMNA IDS IL7R IL2RB MEFV FERMT3 GPC1 CCND1 NLRC4 THPO FGA GNPTAB MIF TERT CLCN7 PRF1 PIGM HLA-DRB1 ITK SMPD1 PRKCD LIPA IDUA CALR ATRX LPIN2 KLRC4 GATA1 TPP2 SUMF1 SAMD9L IFT172 ZAP70 SLC30A10 EPB41 ABCG8 GUSB PKHD1 AGPAT2 HBB CC2D2A NRAS GLRX5 SH2B3 OSTM1 GAA NLRP3 CPOX PSAP CALR SPTB TMEM67 HBA1 JAK2 AKR1D1 SLC39A4 ICOS SPTA1 BACH2 IL7R FAH HBA1 DHCR24 ATP7B OCLN MPL DCDC2 CLCN7 JAK2 TPI1 MPIG6B HIRA GNE NCF2 BCL2 PIK3C2A CCBE1 SF3B1 KCNH1 CASP8 NOD2 ERCC8 BCR USB1 HBB CAVIN1 PSMB9 CARD11 MVK GYPC FAS IGH HLA-B CLCN7 CD28 NOTCH2 CTLA4 GNS HAMP CDAN1 BRAF ADA NCF4 PARN RHAG TNFRSF13C C4A TNFRSF13B STX11 CD27 UBAC2 SGSH CALR NLRP3 CD28 PNP USB1 SEC23B TMEM67 HBG1 IDUA IL23R ADA HSD3B7 RAG2 NLRC4 WDR35 PEX7 TET2 CCDC115 NGLY1 NEU1 LIPA NBEAL2 NCF1 CC2D2A TNFRSF13C SLC4A1 CASP10 SKIV2L RTEL1 ABCA1 PIEZO1 SLC7A7 TLR4 GFI1B NPC2 AP3D1 ITCH ANK1 IL10 ATM RAG1 XIAP GBA RASGRP1 CASP10 TCIRG1 CASR ALG1 TNFRSF11A TP53 PSMB8 ARVCF SH2D1A EPB42 THPO C15ORF41 IL2RA LMNA MYD88 GUSB APOE TNFRSF13B CYBA ASAH1 NPM1 GATA1 COG6 PHKA2 KCNN3 CYBA HFE GLB1 NLRP1 MS4A1 VPS45 GPR35 TBX1 CR2 PRKCD NAGLU BTNL2 BPGM APOE LYST TET2 TNFRSF4 CCDC47 JAK2 SLC4A1 HBA2 CAVIN1 LYST SNX10 PPARG RNASEH2A CTLA4 FAH PKLR FAS CYP7B1 GPC3 CYP7B1 XIAP CYBB SPTB PIK3R1 NBEAL2 CTC1 CCND1 NOP10 JAK2 DDRGK1 IFNGR1 JAK2 FAS ANK1 PTPRC VPS33A IL6 GBA TALDO1 BTD TET2 HK1 GP1BA MPL G6PD AP3B1 IFIH1 AGPAT2 TBXAS1 ERBB3 PHYH
SNP 0