SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT01523171

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

A Phase II, Multicenter, Open Label, Single Arm Study of SAR302503 in Subjects Previously Treated With Ruxolitinib and With a Current Diagnosis of Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis

Primary Objective: - To evaluate the efficacy of once daily dose of SAR302503 in subjects previously treated with ruxolitinib and with a current diagnosis of intermediate-1 with symptoms, Intermediate-2 or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (Post-PV MF), or post-essential thrombocythemia myelofibrosis (Post-ET MF) based on the reduction of spleen volume at the end of 6 treatment cycles; Secondary Objectives: - To evaluate the effect of SAR302503 on Myelofibrosis (MF) associated symptoms as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) diary - To evaluate the durability of splenic response - To evaluate the splenic response to SAR302503 by palpation at the end of Cycle 6 - To evaluate the splenic response to SAR302503 at the end of Cycle 3 - To evaluate the effect of SAR302503 on the Janus kinase 2 (JAK2) V617F allele burden - To evaluate the safety and tolerability of SAR302503 in this population - To evaluate plasma concentrations of SAR302503 for population PK analysis, if warranted

NCT01523171 Hematopoietic Neoplasm
MeSH: Primary Myelofibrosis Hematologic Neoplasms
HPO: Hematological neoplasm Leukemia

1 Interventions

Name: SAR302503

Description: Pharmaceutical form:capsule Route of administration: oral

Type: Drug

SAR302503 400 mg


Primary Outcomes

Measure: Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction from baseline in volume of spleen at the end of Cycle 6 as measured by Magnetic Resonance Imaging (MRI) (or CT scan in subjects with contraindications for MRI)

Time: 6 months

Secondary Outcomes

Measure: Symptom Response Rate (SRR): Proportion of subjects with a ≥50% reduction from baseline to the end of Cycle 6 in the total symptom score using the modified MFSAF

Time: 6 months

Measure: Duration of spleen response, measured by MRI (or CT scan in subjects with contraindications for MRI)

Time: 6 months

Measure: Proportion of subjects with a ≥50% reduction in length of spleen by palpation from baseline at the end of Cycle 6

Time: 6 months

Measure: Response Rate at the end of Cycle 3, defined as the proportion of subjects who have a ≥35% reduction from baseline in volume of spleen at the end of Cycle 3 as measured by MRI (or CT scan in subjects with contraindications for MRI)

Time: 6 months

Measure: Percent change of spleen volume at the end of Cycles 3 and 6 from baseline as measured by MRI (or CT scan in subjects with contraindications for MRI)

Time: 6 months

Measure: Safety, as assessed by clinical, laboratory, ECG, and vital sign events; graded by the NCI CTCAE v4.03

Time: approximately 5 years

Measure: Plasma concentrations of SAR302503

Time: 4 months

Measure: The effect of SAR302503 on the JAK2V617F allele burden

Time: 2 years

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 V617F

Phase II, Open Label, Single Arm Study of SAR302503 In Myelofibrosis Patients Previously Treated With Ruxolitinib Primary Objective: - To evaluate the efficacy of once daily dose of SAR302503 in subjects previously treated with ruxolitinib and with a current diagnosis of intermediate-1 with symptoms, Intermediate-2 or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (Post-PV MF), or post-essential thrombocythemia myelofibrosis (Post-ET MF) based on the reduction of spleen volume at the end of 6 treatment cycles; Secondary Objectives: - To evaluate the effect of SAR302503 on Myelofibrosis (MF) associated symptoms as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) diary - To evaluate the durability of splenic response - To evaluate the splenic response to SAR302503 by palpation at the end of Cycle 6 - To evaluate the splenic response to SAR302503 at the end of Cycle 3 - To evaluate the effect of SAR302503 on the Janus kinase 2 (JAK2) V617F allele burden - To evaluate the safety and tolerability of SAR302503 in this population - To evaluate plasma concentrations of SAR302503 for population PK analysis, if warranted Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction from baseline in volume of spleen at the end of Cycle 6 as measured by Magnetic Resonance Imaging (MRI) (or CT scan in subjects with contraindications for MRI). --- V617F ---



HPO Nodes


HPO:
Hematological neoplasm
Genes 228
MYC CDKN2A TET2 MYD88 TSR2 RPL26 RPL27 TREX1 ASXL1 SCN11A RPL35A BRIP1 ERCC4 ABL1 SAMD9 CEBPA ATRX PDGFRA TNFSF12 RTEL1 PDGFRB HLA-DRB1 ARHGAP26 RPS7 RPS10 GATA1 GATA2 RPS14 RPS15A GBA ETV6 TCIRG1 DNAJC21 EVC SRP54 RPS17 NBN SRP72 MCM4 RPS19 CCND1 BCL2 RPS24 RPS26 RPS27 RPS28 RPS29 BCL6 MAGT1 PTEN MDM2 ADA BCR ADAR TRIP13 ADA2 NSUN2 LYST PICALM GINS1 RNF43 GFI1 F13A1 NAGS F13B ITK STAT3 FANCA FANCC BLM FANCD2 FANCE NUTM1 JAK2 IFIH1 GFI1B TYROBP FANCB FANCF MALT1 FANCG PTPN11 SAMD9L NF1 ICOS PIGA BRCA1 BRAF BRCA2 TINF2 PIK3CA GLI1 PIK3R1 NBEAL2 NFKB1 NFKB2 RNASEH2C RECQL4 BUB1 RAD54B BUB1B SCN9A SCN10A MLF1 MLH1 DKC1 COL14A1 NOP10 BCL10 HAX1 MAD2L2 GNB1 TAL1 KIT TAL2 DNASE1L3 HSPA9 RAD54L KIF11 DNMT3A PNP MPL RAD51 RAD51C RNASEH2B KRAS NPM1 RAF1 RAG1 RAG2 TCF4 EFL1 PIGL FLT3 PMS2 EVC2 RARA NRAS WAS WIPF1 SH2B3 USB1 RB1 RNASEH2A CTC1 CALR NTHL1 POLE SH3GL1 NUMA1 APC NSD1 BIRC3 XIAP CASP10 FANCL SAMHD1 MSH2 CHD7 NUP214 XRCC2 MLLT10 RUNX1 XRCC4 WRAP53 FANCM CBFB FAS CBL NHP2 FASLG CR2 TERC AAGAB TERT CREBBP ZAP70 PALB2 RFWD3 RMRP MSH6 RASGRP1 LIG4 THPO SF3B1 STS UBE2T CD19 MS4A1 IGH DCLRE1C DYNC2LI1 CD27 CD28 SBDS PRF1 FANCI TP63 BRD4 SETBP1 LPP CHEK2 TREM2 TNFRSF13C ELANE TNFRSF13B CD70 SMARCD2 PRKCD FOXP1 CD81 PARN CTLA4 ATM RPL35 TNFRSF1B BUB3 SH2D1A CEP57 IL2RG RPL5 EP300 SLX4 TP53 IL7R RPL11 RPL15 RPL18
Leukemia
Genes 125
MPL RNASEH2B KRAS NPM1 TET2 MYD88 TSR2 RPL26 RPL27 TREX1 EFL1 PIGL SCN11A FLT3 PMS2 RPL35A EVC2 ABL1 CEBPA RARA NRAS WAS WIPF1 ATRX SH2B3 PDGFRA RB1 RNASEH2A PDGFRB CALR ARHGAP26 SH3GL1 RPS7 RPS10 NUMA1 GATA1 GATA2 RPS15A APC NSD1 ETV6 TCIRG1 DNAJC21 EVC SRP54 RPS17 NBN RPS19 SAMHD1 MSH2 RPS24 NUP214 RPS26 RPS27 RPS28 RPS29 MLLT10 RUNX1 XRCC4 CBFB CBL BCR ADAR TRIP13 ADA2 NSUN2 CREBBP PICALM GFI1 F13A1 F13B FANCA FANCC BLM FANCD2 FANCE NUTM1 JAK2 IFIH1 TYROBP MSH6 FANCG LIG4 PTPN11 SAMD9L THPO NF1 STS PIGA BRCA2 DYNC2LI1 PIK3CA SBDS GLI1 PIK3R1 BRD4 SETBP1 RNASEH2C LPP BUB1 BUB1B SCN9A SCN10A TREM2 MLF1 MLH1 ELANE DKC1 ATM HAX1 RPL35 GNB1 BUB3 CEP57 TAL1 KIT TAL2 RPL5 EP300 TP53 RPL11 KIF11 RPL15 DNMT3A RPL18