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Report for Clinical Trial NCT01523171

Developed by Shray Alag, 2020.
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: Hematologic Neoplasms Primary Myelofibrosis
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 349
ATM LIG4 KIT NF1 MPL RPL35A RPL18 DDX41 BCL10 SBDS LPP BIRC3 ETV6 F13A1 MS4A1 TP63 ATRX LIG4 JAK2 NUP214 NFKB2 FANCD2 JAK2 SF3B1 PIGL NRAS TCIRG1 RFWD3 TET2 ATRX TREX1 NTHL1 PTPN11 CALR GINS1 WIPF1 SF3B1 NOP10 RASGRP1 GATA2 GLI1 TNFRSF1B XRCC4 PRKCD NHP2 PIGA CBL MPL MPL PIGL THPO FANCG CASP10 TERC ERCC4 FANCI GATA2 CDKN2A NPM1 CBL ERBB3 PTPN11 PARN GFI1 NBN HSPA9 TET2 KIT GFI1B MAD2L2 BCR DYNC2LI1 ICOS TNFSF12 CHEK2 ELANE RPS14 FANCF USB1 RPL15 TINF2 SBDS NRAS RPL26 ADA2 RPS7 TYROBP MSH2 BCR MLH1 FANCA SLX4 CASP10 TET2 TERT TRIP13 MYD88 NUTM1 GNB1 TNFRSF13B MYC TP53 DNMT3A MDM2 SAMD9L BCL10 POT1 RPS10 EP300 BUB1B DNAJC21 CALR LIG4 BCL2 TET2 JAK2 SAMD9 SMPD1 PALB2 BUB3 PRSS1 NPM1 KIF11 ITK BCR MSH2 TAL2 SRP54 IFIH1 ABL1 FANCE RAD54B FANCM RFWD3 MLLT10 RAF1 BRAF LIG4 CBFB BLM THPO AAGAB CALR CALR TNFRSF1B NBN JAK2 TNFRSF13C DNASE1L3 RPL31 FANCG IL2RG MLF1 PIK3CA DNAJC21 CTLA4 CD28 PNP RUNX1 SETBP1 HAX1 POLE RPS26 CR2 ADA RPS27 ZAP70 ABL1 NBN KRAS RPS15A NAGS CD19 RPL11 IGH FOXP1 BUB1B RAG1 DNMT3A MSH6 FANCA TERC BUB1 FLT3 TAL1 RECQL4 PDGFRA SRP54 RPS29 MCM4 FANCE CTC1 GBA LYST STS SH3GL1 CD70 MPL FANCB SH2B3 EFL1 MAGT1 RAD51 TNFRSF13B IGH TREM2 RMRP RUNX1 FANCD2 CTLA4 DCLRE1C FAS ICOS NSD1 BRD4 RNF43 NSUN2 XIAP IL7R SH2B3 RARA BCL6 GATA2 SRP72 ATM CHIC2 IL2RG BRCA1 RTEL1 TERT ARHGAP26 TNFRSF13C TCF4 EVC SCN10A GATA1 RNASEH2B CCND1 SH2B3 XRCC2 CD19 CR2 JAK2 RPS19 MPL BRCA2 RPL27 F13B WAS TERC BCL10 TP53 COL14A1 PRKCD RPS24 TERT NUMA1 NUP214 RPL5 SH2D1A CCND1 TET2 MSH6 CFTR CD27 PGM3 FANCC EVC2 CEBPA BRCA2 TCF4 CHD7 SCN9A IGH FANCL ADAR BLM ASXL1 HLA-DRB1 RPS14 RNASEH2C SCN11A SMARCD2 GATA2 RPL35 WRAP53 TET2 PDGFRB KRAS RNASEH2A TET2 TINF2 SRP54 DNAJC21 FAS DKC1 RUNX1 APC LIG4 CD81 RECQL4 CD28 DKC1 NBEAL2 RAG2 MLH1 RPS19 ELANE SAMD9L NFKB1 PMS2 ASXL1 FANCC TSR2 PRF1 PTEN MALT1 RAD54L BRIP1 GFI1 SRSF2 TNFSF12 STAT3 ADA RPS17 FLT3 FASLG PIK3R1 KRAS CEP57 PICALM CREBBP GATA2 TP53 CTRC MYD88 TET2 RPS28 RAD51C RB1 UBE2T RMRP SPINK1 TERT KLHDC8B GATA1 JAK2 SAMHD1 RHOH
Leukemia
Genes 190
ATM KRAS KIT RPS15A NF1 MPL RPL35A RPL11 RPL18 DDX41 SBDS LPP ETV6 BUB1B F13A1 DNMT3A JAK2 NUP214 FANCD2 MSH6 FANCA PIGL NRAS TCIRG1 BUB1 TET2 FLT3 TAL1 ATRX TREX1 PDGFRA SRP54 RPS29 PTPN11 CALR FANCE WIPF1 SF3B1 GATA2 GLI1 STS SH3GL1 XRCC4 MPL SH2B3 EFL1 PIGA TREM2 CBL MPL RUNX1 MPL PIGL THPO FANCG NSD1 BRD4 NSUN2 SH2B3 RARA GATA2 NPM1 CBL CHIC2 ERBB3 GFI1 TERT KIT BCR ARHGAP26 DYNC2LI1 EVC SCN10A GATA1 ELANE RNASEH2B RPS14 SH2B3 JAK2 RPL15 SBDS NRAS RPL26 RPL27 ADA2 RPS7 TYROBP F13B MSH2 BCR WAS TERC TP53 TRIP13 MYD88 NUTM1 RPS24 GNB1 TP53 DNMT3A TERT NUMA1 NUP214 RPL5 SAMD9L TET2 CFTR POT1 FANCC RPS10 EP300 EVC2 BUB1B CEBPA BRCA2 DNAJC21 CALR SCN9A LIG4 ADAR BLM TET2 JAK2 SMPD1 RNASEH2C SCN11A GATA2 BUB3 PRSS1 KIF11 BCR RPL35 TAL2 SRP54 IFIH1 ABL1 PDGFRB KRAS RNASEH2A TET2 MLLT10 SRP54 DNAJC21 DKC1 RUNX1 APC LIG4 CBFB BLM THPO CALR NBN JAK2 MLH1 RPS19 ELANE SAMD9L RPL31 PMS2 ASXL1 TSR2 MLF1 PIK3CA DNAJC21 RUNX1 GFI1 SRSF2 SETBP1 HAX1 RPS26 RPS17 FLT3 RPS27 PIK3R1 KRAS CEP57 PICALM CREBBP GATA2 CTRC MYD88 TET2 RPS28 ABL1 RB1 SPINK1 GATA1 NBN JAK2 SAMHD1