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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

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

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

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:
Anemia
Genes 722
GTF2H5 PIEZO1 ERCC6L2 TNFRSF11A HBA2 MECOM RPL18 EPHB4 SBDS RPL35A BIRC3 FERMT3 COX15 ATRX OPA1 NFKB1 CTC1 JAK2 CASP10 NLRP3 TET2 TYMP GREM1 ZBTB24 ATRX LAMA3 FAM111A RFX5 SLC40A1 RMRP SF3B1 SBDS HBB RASGRP1 KIT HYOU1 CASR PRKCD ENG SURF1 HBB HBA2 COQ2 PIGA NPHP4 SLC19A3 IRF2BP2 HMGCL FANCG TNFRSF4 PEPD CA2 EPB41 HBA1 TERC ERCC4 ABCA1 WAS MARS1 REN KLF1 TCIRG1 BMPR1A HBB PARN GBA COX6B1 ENG COL7A1 PLA2G4A TBCE IFNGR1 PKLR RPS14 FANCF RPL15 TNFAIP3 TINF2 NRAS LARS2 WFS1 RFXAP ADA2 RPS7 IL2RB SURF1 COX8A MMAB RPS7 IL12B RPS17 SMAD4 ALG8 SLC7A7 SLC19A2 SLCO2A1 HLA-B CD3G ORAI1 TBL1XR1 DHFR STEAP3 FIP1L1 SLC4A1 SPTA1 COX14 PRF1 AGXT TF COL7A1 SPTA1 SAMD9 ABCD3 KCNN4 PGK1 NDUFS2 CIITA SEC23B BCOR NDUFS1 PET100 NPM1 ITK SRP54 BPGM STX11 SLC4A1 SLC46A1 DNASE1 COX20 PLEC PLEC RPS28 NABP1 CALR CLCN7 RAG2 CDAN1 PHKG2 TNFRSF13C HBG1 CFH RPL31 FANCG FCGR2A IL2RG NDUFAF2 COG6 CCND1 SLC11A2 PHGDH NUMA1 IFNG SPTB ITGB4 RPS26 GATA1 HPRT1 ELANE RPS27 SLC29A3 ISCU MMP1 HELLS CA2 FLI1 SPTB STAT3 POLG KRAS RHAG SPTA1 STIM1 ATP7B SLC7A7 ITGA2B RPL11 SHPK HBB-LCR GP1BA FOXP1 EPB42 COA8 GYPC MYSM1 MPL RPS26 FANCA LAMB3 ALAS2 ACVRL1 MPLKIP AMN ABCB7 GATA1 NHEJ1 TPP2 GBA HBB LRBA DNMT3B SDHA ALAS2 CP FANCB EFL1 FECH RAD51 PRKACG C1R MMUT RMRP DBH FANCD2 PFKM CLCN7 SMAD4 TACO1 XRCC4 LCAT NPHP1 PRF1 NLRP3 SMAD4 SNX10 TTC7A FANCB RPL5 ADAMTS13 BRCA1 COL17A1 RTEL1 AMMECR1 PACS2 FASTKD2 ALAD AK1 HBB NDUFAF5 SMPD1 SPP1 HBA2 NDUFB8 IDH1 HBB ATP7B CTLA4 GATA1 GATA1 CAD NPM1 SPTB SLX4 XRCC2 FTCD MPL SRD5A3 FOXP3 CD59 NHLRC2 DCLRE1C PSMB8 PSMB4 GCLC KIT MTR TMPRSS6 SP110 SMAD4 HBA1 RPL5 NDUFA12 EWSR1 NDUFA13 PGM3 FANCC IDH2 SLC46A1 LPIN2 FANCL TP53 UNC13D AGGF1 STK11 GTF2E2 C1QA TGFB1 RPL35 APC GSS GPI WRAP53 BTK DGKE PDGFRA DNAJC21 FAS SCO1 MTFMT TINF2 ABCB7 RPS29 ITGB3 CD81 EPB42 DKC1 OSTM1 STK11 IFT140 PRKCD RPS19 SAMD9L BTNL2 SPTA1 COX10 RPL15 NLRP1 HBG2 NLRC4 RPS15A CRIPT SDHA PHGDH GLA TNFSF12 ACVRL1 ADA SPTA1 ZBTB16 FASLG CTSK STIM1 FOXRED1 KLF1 COL7A1 CLCNKB SFXN4 HBB COX4I2 SLC25A13 RAD51C DNAJC19 RMRP ANK1 STAT5B C3 GATA1 PRDX1 G6PC3 COL7A1 RPL35A YARS2 DDX41 BCL10 NDUFA4 NPHP4 ERCC2 AK2 CLPX HBA1 COA8 ECHS1 RPL11 MS4A1 FASLG LIG4 TFR2 NFKB2 FANCD2 PUS1 TBXAS1 TARS1 AASS IREB2 KIF15 RFWD3 ATRX TERT NDUFAF3 PLEKHM1 HBA1 PTEN UROS WIPF1 HBA1 ATRX NDUFS3 RHAG NOP10 LIPA LMBRD1 THRA GBA TRNT1 HLA-B HBD TACO1 IRX5 RPL27 SEC61A1 TNFSF11 SLC19A2 HBG1 NHP2 PCNT ALX4 BMPR1A NDUFS4 PTPN22 TRNN CD55 STAT4 HK1 PML ANK1 FANCI NT5C3A BMPR1A UMPS NDUFS7 G6PD CFB LAT LYRM7 ERBB3 PARN NBN HSPA9 TET2 RARA LARS1 RNF113A MAD2L2 HELLPAR TEK ATP11C BRCA1 ICOS RFXANK ELMO2 GATA1 TNFSF12 ELANE PTH1R GLRX5 USB1 EPO CFH FAS SMARCAL1 SBDS LYST HPRT1 FMO3 RPL26 TTC7A ENG FANCA STAT1 MUC1 PCCB TALDO1 HPGD SLX4 CASP10 TET2 RAG2 TERT SLC4A1 MYD88 GLA PKLR ACD TNFRSF13B KDM6A CLCN7 PTF1A ACVR1 RPS10 HAMP SAMD9L FARSB CPOX RPS10 NDUFS2 DNAJC21 SMARCAL1 VPS45 KMT2D TGFB1 GBA PALB2 CFI GCLC PUS1 TALDO1 HBB SRD5A3 PIGT CD46 FANCE FANCM TMEM67 SLC4A1 NPHP1 DNM1L SLC2A1 RPL26 CFI WFS1 RAG1 LIG4 UMPS CASK PGM3 ADAR PSMB8 PDHA1 SNX10 MTRR DKC1 PNP WT1 HMOX1 COG1 CR2 STEAP3 ADA COL7A1 FARS2 LAMC2 ZAP70 CUBN ALPL F2 ETV6 TCN2 STAT3 NBN RPS15A CFHR1 GDF2 ITGB4 CD19 HMGCL UROD TNFSF11 MMADHC TCIRG1 FECH NDUFA2 ABCD4 TREX1 RAG1 PSMB9 COL4A1 CDCA7 TF RBM8A KRT14 SDHC TERC EXT2 LPIN2 RAG1 TFRC EFL1 TSR2 PFKM HBB RPS29 FOXP3 FANCE CLCN7 CTC1 SDHB LYST KIF1B PNPO TBXAS1 HAVCR2 ABCG8 MMAA NDUFAF6 TRNW FAM111A DCLRE1C FAS HBB TERT RPS27 NSUN2 CTLA4 XIAP PLEKHM1 IL7R NOD2 GSS VPS33A HBG2 NDUFA10 PRKAR1A FCGR2B EPB41 RAG1 NDUFV2 HBA2 THBD CISD2 ERCC3 TERC NDUFV1 SLC4A1 GNA14 GALT SLC2A1 ZBTB20 CFHR3 TRNS1 RPS19 STAT1 ANK1 BRCA2 AIRE RPL27 SLC25A38 WAS IL2RA PGK1 HLA-DRB1 LIPT1 HBB RPS24 FDX2 ALDOA IKZF1 PRKCD RPS24 LAMA3 ATRX KCNQ1 DAXX UBE2T SH2D1A SLC12A3 ACAD8 MVK DNAJC19 MTHFD1 UBR1 ADA2 RHAG LAMC2 SCO2 MPIG6B SCARB2 CHD7 IGH OCRL NDUFA9 ALAS2 C15ORF41 RPS14 GPX1 SMARCD2 STING1 HBA2 CD40LG CD46 CBLIF TINF2 SRP54 DKC1 TPI1 ABCB6 GATA1 ACTN4 RECQL4 PSAP RAG2 IRAK1 BMPR1A HBB MAD2L2 NFKB1 PET100 FANCC TSR2 MALT1 UROS BRIP1 FANCL PHKA2 TCIRG1 YARS2 ABCA1 SARS2 MTRR FERMT1 RPS17 PCCA PNPO GBA MMP1 TRNT1 APOA1 MMACHC CP LAMB3 TET2 RPS28 MMUT STIM1 UBE2T RTEL1 RRM2B KIF23 TERT KCNE1 NDUFS8 PHF21A MLX
Splenomegaly
Genes 478
CLDN1 MPL PIEZO1 TNFRSF11A GUSB CCDC115 FERMT3 AGPAT2 B2M JAK2 CASP10 NLRP3 ATRX LYZ IDUA SF3B1 RASGRP1 CASR PRKCD HBB HBA2 KCNN3 LMNA MPL MPL RPGRIP1L TNFRSF4 PEPD EPB41 NGLY1 IFT172 ABCA1 KLF1 GPC1 GBA HSD3B7 TREX1 SUMF1 IFNGR1 PKLR NRAS IL2RB NPC2 TMEM67 CYP7B1 IFIH1 ASAH1 HIRA GBA SLCO2A1 GNPTAB BRAF TP53 BMP2 DHCR24 LMNA CYBA STEAP3 KCNH1 USB1 SPTA1 CALR PRF1 ABCB4 SMPD1 ABCD3 KCNN4 SEC23B ICOS NPM1 ITK BCR MEFV JAK2 BPGM IL10 STX11 SLC4A1 PTPRC SLC17A5 COG7 IL12A-AS1 THPO FAH PIK3C2A ARVCF CALR BACH2 ATP6V1B2 CDAN1 JAK2 PHKG2 TNFRSF13C ABCA1 ITCH MST1 SEC24C IDUA IL2RG COG6 CCND1 CD28 RUNX1 IDUA FUCA1 SPTB SCYL1 JMJD1C GNE COMT CA2 SPTB KRAS GPC3 SMPD1 TTC37 RHAG SPTA1 ATP7B SLC7A7 IGH GP1BA EPB42 GYPC HFE GPD1 GNS NEU1 FOS NCF1 PDGFRA TPP2 RNU4ATAC GBA IL1RN GBA HBB CAV1 PSAP SH2B3 PIGM TNFRSF13B MIF MMUT LIPA GP1BB SKIV2L VPS13A INPPL1 AP3B1 NOTCH2 NLRP3 GATA2 SNX10 ATM JAK2 RTEL1 NLRP12 HBB APOE SMPD1 SPTB CD19 CR2 JAK2 MPL AKR1D1 CYBB GPC4 DCLRE1C PSMB8 PSMB4 APOC2 DDRGK1 SGSH ARSB BTNL2 HSD3B7 ASAH1 CCBE1 CD27 PSAP LPIN2 HLA-DRB1 RREB1 PEPD DOLK TGFB1 GPI WRAP53 KLRC4 TET2 HLA-B FAS APOA1 ZAP70 ADAMTS3 PHKG2 CD81 EPB42 DKC1 OSTM1 PRKCD DGUOK ATP6AP1 GPC3 NLRP1 SOX10 NLRC4 TNFSF12 DYNC2LI1 ADA PTEN SLC30A10 FASLG PIK3R1 CTSK KLF1 ALG1 PIK3CA PPARG COX4I2 RMRP FGA ANK1 GATA1 G6PC3 PHYH ERCC6 HJV CCR1 LACC1 HBA1 MVK MS4A1 NBEAL2 BSCL2 FASLG LIG4 NFKB2 TBXAS1 APOE NEU1 CALR PLEKHM1 UROS HBA1 NOP10 LIPA GBA TNFRSF1B SUMF1 HBG1 NHP2 TBX1 THPO IDS MEFV HK1 ANK1 GBA RPGRIP1L DCDC2 G6PD LAT HLA-DRB1 ERBB3 PARN TET2 BSCL2 SLC29A3 GFI1B LCAT PEX7 GBA ICOS GATA1 TNFSF12 GLRX5 USB1 FAS LYST FMO3 SLC39A4 AGA GPR35 CTNS CYBC1 HPGD CASP10 RAG2 MYD88 CCDC47 IDUA DPM1 PKLR TNFRSF13B CLCN7 HAMP BTD SAMD9L CC2D2A MKS1 NPC1 CPOX RNASEH2A GLIS3 VPS45 BCL2 AKT1 CAVIN1 PTEN NOTCH1 JAK2 GBA TNFRSF1A IL7R TALDO1 HBB NAGLU COG4 ABL1 IDUA SLC4A1 SLC2A1 RAG1 CYP7B1 UMPS CASK CALR IL12A PSMB8 TNFRSF1B IL23R SNX10 DNASE1L3 IL6 CTLA4 CLCN7 PNP PPARG HGSNAT CR2 ADA AP3D1 UFD1 CAVIN1 FAT4 CC2D2A GBA TMEM67 CD19 TNFSF11 CYBB F5 TCIRG1 AKT1 GAA CYBA ATP8B1 RAG1 JAK2 PSMB9 NCF1 WDR35 TERC CAV1 CARD11 OCLN HBB MCM4 NLRC4 PIEZO1 FOXP3 IRF8 ERAP1 CLCN7 CTC1 LYST INPP5E HAVCR2 ABCG8 MPL CASR IGH KCNH1 CTLA4 DCLRE1C FAS ICOS DCDC2 CTLA4 XIAP IL7R LIPA SH2B3 BCL6 NOD2 VPS33A HBG2 CASP8 EPB41 RAG1 CFAP410 ALMS1 NCF2 TNFRSF13C GALE ABCB11 SLC4A1 SH2B3 ANK1 FAS NCF2 AKR1D1 IL2RA TLR4 ALDOA GPC4 STAT4 PRKCD DDRGK1 HFE SH2B3 SH2D1A CCND1 TET2 MVK ADA2 RHAG XIAP MPIG6B SCARB2 CHD7 LBR LPL ALAS2 HLA-DRB1 C15ORF41 FAH TCF4 HBA2 OTC CD40LG PEX2 UBAC2 TINF2 RAB27A TPI1 GATA1 GNPTAB C4A CD28 NBEAL2 RAG2 PTEN GLB1 AGPAT2 DGUOK NFKB1 GPIHBP1 NCF4 UROS PHKA2 TCIRG1 MEFV PKHD1 MAN2B1 ERCC8 GBA MVK GUSB PIK3CD APOA1 TBX1 TET2 TERT JAK2