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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
Group Labels: 1

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

Allocation: N/A

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:
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