SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT00931762

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

Phase II Trial of Oral Panobinostat (LBH589), a Novel Deacetylase Inhibitor (DACi) in Patients With Primary Myelofibrosis (PMF), Post Essential Thrombocythemia (ET) Myelofibrosis and Post- Polycythemia Vera (PV) Myelofibrosis

This study will assess the safety and efficacy of Panobinostat as a single agent in the treatment of Primary Myelofibrosis, Post-Polycythemia Vera and Post-Essential Thrombocythemia. There will be two cohorts - patients with JAK2 mutation and patients without JAK2 mutation.

NCT00931762 Primary Myelofibrosis Post-Polycythemia Vera Post-Essential Thrombocytopenia
MeSH: Thrombocytopenia Primary Myelofibrosis Polycythemia Polycythemia Vera Purpura, Thrombocytopenic
HPO: Polycythemia Thrombocytopenia

1 Interventions

Name: Panobinostat

Description: Panobinostat will be administered orally once a day on Monday, Wednesday and Friday at a fixed dose of 40 mg. A cycle is defined as 28 days of treatment.

Type: Drug

Panobinostat


Primary Outcomes

Measure: To evaluate the overall response (CR, PR, and clinical improvement) to oral panobinostat as a single agent at 40 mg daily every Monday, Wednesday and Friday in patients with myelofibrosis.

Time: Upon enrollment of 13 participants into each cohort of the study and at the end of the study.

Secondary Outcomes

Measure: To compare the response to panobinostat in patients with the JAK2 V617F mutation to those without the JAK2 V617F mutation

Time: Upon enrollment of 13 participants into the study and at the end of the study

Measure: To evaluate the symptomatic improvement of myelofibrosis patients treated with panobinostat using the Myelofibrosis Symptom Assessment Form (MF-SAF) at baseline and after 2 and 4 months of treatment

Time: Upon enrollment of 13 participants in each cohort and at the end of the study

Measure: To evaluate the symptomatic improvement of myelofibrosis patients treated with panobinostat using the Myelofibrosis Symptom Assessment Form (MF-SAF) at baseline and after 2 and 4 months of treatment

Time: throughout the study

Measure: To assess compliance to panobinostat treatment as assessed by monthly capsule counts

Time: at the end of the study

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 V617F

To compare the response to panobinostat in patients with the JAK2 V617F mutation to those without the JAK2 V617F mutation. --- V617F ---

To compare the response to panobinostat in patients with the JAK2 V617F mutation to those without the JAK2 V617F mutation. --- V617F --- --- V617F ---

(The presence of a JAK2 V617F mutation is not required for study entry) 2. Patients must meet the following laboratory criteria: - Patients can be either JAK2 V617F mutated or wild type - Serum potassium, magnesium, phosphorous, sodium, total calcium (corrected for serum albumin) or ionized calcium within normal limits (WNL) for the institution Note: Potassium, magnesium, phosphorous, sodium, and/or calcium supplements maybe given to correct values that are < LLN. --- V617F ---

(The presence of a JAK2 V617F mutation is not required for study entry) 2. Patients must meet the following laboratory criteria: - Patients can be either JAK2 V617F mutated or wild type - Serum potassium, magnesium, phosphorous, sodium, total calcium (corrected for serum albumin) or ionized calcium within normal limits (WNL) for the institution Note: Potassium, magnesium, phosphorous, sodium, and/or calcium supplements maybe given to correct values that are < LLN. --- V617F --- --- V617F ---



HPO Nodes


HPO:
Polycythemia
Genes 15
PKLR SH2B3 VHL JAK2 ENG EPO EPOR SLC30A10 EPAS1 EGLN1 BPGM ACVRL1 CYB5R3 FH GATA1
Thrombocytopenia
Genes 264
CFH TET2 TPP2 USP18 TREX1 ABCA1 MYH9 GALC BRIP1 EOGT ERCC4 ZBTB16 ABL1 SAMD9 ATP7B PSAP ACD ATRX HLA-B PDGFB TNFSF12 RTEL1 PDGFRB VPS45 TMEM165 HLA-DRB1 ACP5 SRC VPS33A PSMB4 CD109 GATA1 GATA2 PEPD PSMB8 PSMB9 GBA HLCS ETV6 DNAJC21 SRP54 MECOM NBN RPS19 IKZF1 SLC46A1 CD46 ACTN1 TNFRSF11A ITGA2 ITGA2B RREB1 SLFN14 ADA BCR ADAR NSUN2 LYST ITGB3 MYSM1 PHGDH STAT1 ITK STAT3 STAT5B HELLPAR HIRA FANCA FANCC IVD FANCD2 FANCE STIM1 TUBB1 JAK2 HOXA11 IFIH1 GFI1B FANCB FANCF FANCG BLOC1S6 PTPN11 SARS2 ICOS BRCA1 BRAF CLCN7 BRCA2 TINF2 CIITA SC5D COG1 COG6 ARHGAP31 POMP NBEAL2 DGUOK NFKB1 BTK DHFR NFKB2 RNASEH2C UFD1 SNX10 DIAPH1 ABCD4 DKC1 FIP1L1 ALG8 PLAU NOP10 RBM8A MAD2L2 UROS DOCK6 TALDO1 KDM6A NIPBL COG4 NOTCH1 TBX1 CA2 GP1BA PML GP1BB PNP FOXP3 GP9 MPL RAD51 RAD51C RNASEH2B TBXAS1 KRAS NPM1 COL4A5 RAG1 FLI1 RAG2 FLNA EFL1 RARA VWF NRAS WAS COMT WIPF1 OSTM1 USB1 TCN2 RNASEH2A DLL4 CTC1 WFS1 CALR ACAD9 SMARCAL1 NUMA1 SLC35A1 SLC19A2 ANKRD26 XIAP CASP10 DGKE SALL4 FANCL SAMHD1 ADAMTS13 OCRL LBR NABP1 APOE XRCC2 RUNX1 WRAP53 SLC7A7 FANCM AP3B1 CFI MPIG6B FAS NHP2 LARS2 FASLG CR2 TERC SP110 TBL1XR1 TERT RFX5 RFXAP JMJD1C ARPC1B ZAP70 PALB2 MMACHC RFWD3 STT3B TFRC ESCO2 IFNG MMAA RASGRP1 LIG4 FCGR2C KMT2D TNFSF11 GNA14 SEC24C LAT UBE2T CD19 G6PC3 MS4A1 ARVCF DCLRE1C GUCY1A1 FARS2 MTOR SBDS PRF1 FANCI SLC20A2 RFXANK CD36 BTNL2 SCARB2 RBPJ PRDX1 CYCS CD40LG PRKACG ERCC6L2 TNFRSF13C ANKRD11 PRKAR1A AGK ELANE TNFRSF13B SMARCD2 PRKCD CD81 PARN CTLA4 TNFAIP3 LRBA OCLN NHEJ1 HPS5 STX11 SH2D1A MAP2K1 FYB1 MMAB CDC42 PCCA SPATA5 PCCB LMBRD1 SLX4 MMUT MVK XPR1