SNPMiner Trials by Shray Alag


SNPMiner Trials: Mutation Report


Report for Mutation V299L

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

There are 9 clinical trials

Clinical Trials


1 Multicenter, Open-label Single Arm Phase II Study Testing the Tolerability and the Efficacy of Bosutinib step-in Dosing in Chronic Phase CML Patients Intolerant or Refractory to Previous Imatinib, Nilotinib or Dasatinib Therapy

Bosutinib is a 2nd generation tyrosine kinase inhibitor that has shown promising results from first up to fourth line treatment in patients with in chronic phase of chronic myelogenous leukaemia. Most patients discontinuing the treatment with Bosutinib do so because of side effects occuring early after starting the treatment. A step in dosing scheme could improve these early toxicities. The aim of this study therefore is to demonstrate that temporary lowering of the Bosutinib dose during early treatment may help to reduce or prevent side effects while preserving efficacy.

NCT03205267 Chronic Myelogenous Leukaemia Drug: Bosulif
MeSH: Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Chronic-Phase
HPO: Chronic myelogenous leukemia Myeloid leukemia

Exclusion Criteria: - Hypersensitivity against Bosutinib or other ingredients of the medicinal product - Evidence of features of accelerated (AP) or blast phase (BC) at any time before inclusion - Patients with BCR-ABL negative CML - Patients having received Imatinib for more than 6 weeks prior to initiation of 2nd generation TKI (either Nilotinib or Dasatinib) - Patients with known T315I or V299L mutation - Concomitant medications known to be strong inducers or inhibitors of P450 isoenzyme CYP3A4 - History of pancreatitis, inflammatory bowel disease requiring systemic or topical immunosuppressive therapy within the last 12 months - Impaired cardiac function, including any of the following: 1. History of or presence of complete left bundle branch block, right bundle branch block plus left anterior hemiblock, bifascicular block in screening ECG 2. ST depression of >1mm in 2 or more leads and/or T wave inversions in 2 or more contiguous leads in screening ECG 3. Congenital long QT syndrome 4. QTc> 450 msec in the screening ECG 5. QT-prolonging concomitant medication 6. History of or presence of significant ventricular or atrial tachyarrhythmias in screening ECG 7. History of or presence of clinically significant resting bradycardia (< 50 beats per minute) 8. Myocardial infarction within 6 months prior to inclusion 9. Unstable angina diagnosed or treated during the past 12 months 10. --- T315I --- --- V299L ---

Primary Outcomes

Description: calculation of the incidence rate of grade 2 to 4 GI toxicity with and without regard to causality

Measure: Rate of GI-Toxicity (i.e. incidence and severity of grade 2 to 4 toxicities)

Time: within the first 6 months of treatment

Secondary Outcomes

Description: Apart from grade 2 to 4 GI toxicity, the occurrence of toxicity will be analyzed in general. This regards all grade toxicity, 2 to 4 grade and 3 to 4 grade toxicity (NCI CTCAE v4.0).

Measure: overall Tolerability (i.e. all grade, grade 2 to 4 and grade 3 and 4 toxicities)

Time: at month 6, 12 and 24

Description: Rating of CCyR, MMR, MR4 and MR4.5 after bone marrow aspiration and biopsy

Measure: Molecular response mesured by efficacy parametern

Time: at month 3, 6, 12, 18 and 24

Description: The EORTC QLQ-CML30 will be scored according to the respective user's guides.

Measure: Patient-reported outcome measures (QoL)

Time: at month 3 and 6

Description: Progression will be assessed according to the visit schedule at any visit.

Measure: Progression-free survival (PFS)

Time: at month 3, 6, 9, 12, 15, 18, 21 and 24

Description: Survival will be assessed according to the visit schedule at any visit.

Measure: Overall Survival (OS)

Time: at month 3, 6, 9, 12, 15, 18, 21 and 24

Description: The rate and type of mutations will be described. The rate will be given as percentage of patients developing mutations.

Measure: The rate of emerging mutations during Bosutinib treatment

Time: at month 3, 6, 9, 12, 15, 18, 21 and 24

Other Outcomes

Description: Ankle Brachial Index (ABI) will be prospectively evaluated followed by analysis of various biomarkers for vascular damage

Measure: Vascular biology substudy: analysis of clinical and laboratory vascular and metabolic risk factors

Time: baseline, at months 6, 12 and 24

Description: It is planned to analyze PK parameters sequentially by taking serum from PB and subsequent HPLC-MS/MS technology. Pharmacodynamics in different compartments will be analyzed by means of flow-cytometry of PB and BM samples.

Measure: Pharmacokinetic (PK), pharmacodynamic (PD) substudy

Time: at day 1, months 1, 2, 3, 12, 18, 24

Description: Assessment of telomere length in normal and leukemic cells as potential new biomarker for prognosis, prediction of response under Bosutinib

Measure: Telomere substudy

Time: at months 1, 2, 3, 12 and 24

Description: Documentation of subclone evolution or elimination during Bosutinib treatment

Measure: Ultra-deep next-generation sequencing (UD-NGS)

Time: at months 1, 2, 3, 12 and 24

Description: Documentation of patient´s comorbidity profile using 3 different comorbidity scales

Measure: Assessment of patients comorbidities and correlation to individual patient´s adverse side effect profile substudy

Time: through study completion, an average of 2 years

Description: Investigation the role of the 5-HT pathway in directing bosutinib induced diarrhea by assessment of 5-HT and certain cytokine levels and genetic analysis including SNP and GWAS

Measure: Transport mechanisms of Bosutinib and mechanisms of diarrhea substudy

Time: every 14 days month 1-3

2 Bosutinib Efficacy, Safety, Tolerability (BEST) Study in Elderly Chronic Myeloid Leukemia Patients Failing Front-line Treatment With Other Tyrosine Kinase Inhibitors

The objective of the present study is to evaluate a new drug called bosutinib as it is believed that this agent may be able to predict an excellent prognosis in patients that did not obtain any benefit with other drugs before. Still, this needs to be proved and we hope this study is able to do so.

NCT02810990 Chronic Myeloid Leukemia Drug: Bosutinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

Exclusion Criteria: 1. Accelerated or blastic phase CML (according to ELN 2013 criteria) 2. Patients with the T315I or the V299L mutation 3. Patients previously treated with 2 TKIs or more 4. Compelled to take medications that are known to be associated with Torsades de Pointes and/or with significant QTc prolongation 5. Any condition or illness that, in the opinion of the Investigator, would compromise patient safety or interfere with the evaluation of the drug 6. --- T315I --- --- V299L ---

Primary Outcomes

Measure: Number of patients who are in major molecular response (MMR)

Time: One year treatment

Secondary Outcomes

Measure: Number of patients who obtain molecular response

Time: At 6 and 12 months from treatment start

Measure: Number of patients discontinuing treatment for failure, adverse events or other reasons

Time: At 12 and 36 months

Measure: Number of Adverse Events (AEs)

Time: At 36 months

Measure: Number of patients alive

Time: At 36 months

Measure: Number of patients on treatment at 200, 300 and 400 mg or more daily

Time: At 6, 12 and 36 months

Measure: Number and type of BCR-ABL1 mutations

Time: At 36 months

Measure: Patient reported quality of life

Time: At 3, 6, and 12 months

3 A Study of Complete Molecular Response for Chronic Myeloid Leukemia in Chronic Phase Patients, Treated With Dasatinib

The purpose of this study is to evaluate complete molecular response of Dasatinib in patients for Philadelphia chromosome-positive chronic myeloid leukemia

NCT01342679 Chronic Myeloid Leukemia Drug: dasatinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

Inclusion Criteria: - Chronic Myeloid Leukemia in the Chronic Phase - 20 years old over - ECOG performance status (PS) score 0-2 - Patients for major molecular response (MMR) with no CMR - Adequate organ function (hepatic, renal and lung) - Signed written informed consent Exclusion Criteria: - A case with the double cancer of the activity - Women who are pregnant or breastfeeding - The case of Pleural effusion clearly - Patients with complications or a history of severe or uncontrolled cardiovascular failure following - have a Myocardial infarction within 6 months - have an Angina within 3 months - have a Congestive heart failure within 3 months - have a suspected congenital QT syndrome - have a QTc interval of more than 450msec at baseline - A serious uncontrolled medical disorder that would impair the ability of the subjects to receive protocol therapy - Prior treatment with dasatinib - Subjects with T315I, F317L and V299L BCR-ABL point mutations Inclusion Criteria: - Chronic Myeloid Leukemia in the Chronic Phase - 20 years old over - ECOG performance status (PS) score 0-2 - Patients for major molecular response (MMR) with no CMR - Adequate organ function (hepatic, renal and lung) - Signed written informed consent Exclusion Criteria: - A case with the double cancer of the activity - Women who are pregnant or breastfeeding - The case of Pleural effusion clearly - Patients with complications or a history of severe or uncontrolled cardiovascular failure following - have a Myocardial infarction within 6 months - have an Angina within 3 months - have a Congestive heart failure within 3 months - have a suspected congenital QT syndrome - have a QTc interval of more than 450msec at baseline - A serious uncontrolled medical disorder that would impair the ability of the subjects to receive protocol therapy - Prior treatment with dasatinib - Subjects with T315I, F317L and V299L BCR-ABL point mutations Chronic Myeloid Leukemia Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive null --- T315I --- --- F317L --- --- V299L ---

Inclusion Criteria: - Chronic Myeloid Leukemia in the Chronic Phase - 20 years old over - ECOG performance status (PS) score 0-2 - Patients for major molecular response (MMR) with no CMR - Adequate organ function (hepatic, renal and lung) - Signed written informed consent Exclusion Criteria: - A case with the double cancer of the activity - Women who are pregnant or breastfeeding - The case of Pleural effusion clearly - Patients with complications or a history of severe or uncontrolled cardiovascular failure following - have a Myocardial infarction within 6 months - have an Angina within 3 months - have a Congestive heart failure within 3 months - have a suspected congenital QT syndrome - have a QTc interval of more than 450msec at baseline - A serious uncontrolled medical disorder that would impair the ability of the subjects to receive protocol therapy - Prior treatment with dasatinib - Subjects with T315I, F317L and V299L BCR-ABL point mutations Inclusion Criteria: - Chronic Myeloid Leukemia in the Chronic Phase - 20 years old over - ECOG performance status (PS) score 0-2 - Patients for major molecular response (MMR) with no CMR - Adequate organ function (hepatic, renal and lung) - Signed written informed consent Exclusion Criteria: - A case with the double cancer of the activity - Women who are pregnant or breastfeeding - The case of Pleural effusion clearly - Patients with complications or a history of severe or uncontrolled cardiovascular failure following - have a Myocardial infarction within 6 months - have an Angina within 3 months - have a Congestive heart failure within 3 months - have a suspected congenital QT syndrome - have a QTc interval of more than 450msec at baseline - A serious uncontrolled medical disorder that would impair the ability of the subjects to receive protocol therapy - Prior treatment with dasatinib - Subjects with T315I, F317L and V299L BCR-ABL point mutations Chronic Myeloid Leukemia Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive null --- T315I --- --- F317L --- --- V299L --- --- T315I --- --- F317L --- --- V299L ---

Primary Outcomes

Measure: Rate of complete molecular response (CMR) after treatment with dasatinib

Time: at 12 months

Secondary Outcomes

Measure: Dasatinib of dose intensity

Time: at 12 months

Measure: Expansions rate of large granular lymphocyte

Time: at 12 months

Measure: Progression free survival

Time: at 12 months

Measure: Number of Participants with Adverse Events

Time: at 12 months

4 A PHASE 4 SAFETY AND EFFICACY STUDY OF BOSUTINIB (BOSULIF (REGISTERED)) IN PATIENTS WITH PHILADELPHIA CHROMOSOME POSITIVE CHRONIC MYELOID LEUKEMIA PREVIOUSLY TREATED WITH ONE OR MORE TYROSINE KINASE INHIBITORS

The purpose of this study is to fulfill the post-authorization commitment made by Pfizer to the European Medicines Agency in providing additional safety and efficacy data in approximately 150 Philadelphia Chromosome Positive Chronic Myeloid Leukemia patients with high unmet medical need, including 75 Chronic Phase, Accelerated Phase or Blast Phase patients in the fourth or later line treatment setting (i.e., after treatment with at least 3 other Tyrosine Kinase Inhibitors).

NCT02228382 Previously Treated PH + CML Drug: Bosutinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Philadelphia Chromosome
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

- Known T315I or V299L mutation. --- T315I --- --- V299L ---

Primary Outcomes

Description: Cytogenetic response (CyR) is based on the prevalence of Philadelphia positive cells in metaphase from Bone Marrow sample.

Measure: Percentage of Participants with Major Cytogenetic Response (MCyR) by Week 52 in Chronic Phase Second-line Population and Chronic Phase Third-line Population of Philadelphia Chromosome Positive Chronic Myeloid Leukemia patients.

Time: Week 52

Description: Cytogenetic response (CyR) is based on the prevalence of Philadelphia positive cells in metaphase from Bone Marrow sample.

Measure: Percentage of Participants with Major Cytogenetic Response (MCyR) by Week 52 in Chronic Phase Fourth-line and later-line Population of Philadelphia Chromosome Positive Chronic Myeloid Leukemia patients.

Time: Week 52

Description: OHR includes Complete Hematological Response (CHR) or return to chronic phase (RCP).

Measure: Percentage of Participants with Overall Hematologic Response (OHR) by Week 52 in Advanced Leukemia Population patients.

Time: Week 52

Secondary Outcomes

Description: Cytogenetic response (CyR) is based on the prevalence of Philadelphia positive cells in metaphase from Bone Marrow sample.

Measure: Estimate cumulative probability of Percentage of Participants with Major Cytogenetic Response in Chronic Phase and Advanced Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Week 52

Description: OHR includes Complete Hematological Response (CHR) or return to chronic phase (RCP).

Measure: Estimate cumulative probability of Percentage of Participants with Overall Hematologic Response in the Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient population by number of lines of prior therapy.

Time: Week 52

Measure: Characterize distribution of best response (molecular, cytogenetic, or hematologic) in the Chronic Phase, Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Week 52

Measure: Estimating probability of Percentage of Participants with Major Cytogenetic Response at 3, 6, 12, 18, and 24 months in the Chronic Phase, Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Month 3, 6, 12, 18, and 24

Measure: Estimating the probability of confirmed Overall Hematologic Response at 3, 6, 9, 12, 18, and 24 months in the Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Month 3, 6, 9, 12, 18, and 24

Measure: Estimating the probability of cumulative confirmed Complete Hematologic Response in the Chronic Phase, Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Week 52

Measure: Estimating the probability of cumulative major molecular response in the Chronic Phase, Accelerated Phase and Blast Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia patient populations.

Time: Week 52

5 Dynamics of ABL Mutations in Imatinib Failed Ph Positive or Bcr-Abl Positive CML CP or AP Patients Who Treated With Nilotinib as Second-line TKI Therapy (AMICAN-Prospective)in Asia

The purposes of this study are to investigate expression and frequency of ABL point mutations, a major cause of resistance in imatinib failed CML Asian patients and to find causes of Asian-specific resistance to cancer-targeting therapies through a prospective investigation of dynamics of point mutations and expression of new point mutations during nilotinib treatment.

NCT01562847 Chronic Myeloid Leukemia Drug: Nilotinib
MeSH: Leukemia, Myelogenous, Chronic, BCR-ABL Positive
HPO: Chronic myelogenous leukemia

With regard to peculiar point mutations, V299L, F317L, and E25K/V show relative resistance to dasatinib, and p-loop mutations including G250E, Q252H, Y253F/H and E255K/V and F359C/V show relative resistance to nilotinib. --- V299L ---

Primary Outcomes

Measure: To confirm the patterns of resistance including point mutations which are newly expressed during the nilotinib treatment

Time: 5 years

Secondary Outcomes

Measure: To analyze and evaluate the overall survival and disease free survival in the nilotinib treatment according to progression of the disease and types of point mutations

Time: 5 years

6 An Open Label, Randomized (2:1) Phase IIb Study of Dasatinib Versus Imatinib in Patients With Chronic Phase Chronic Myeloid Leukemia Who Have Not Achieved an Optimal Response to 3 Months of Therapy With 400 mg Imatinib

The purpose of this study is to test the hypothesis that patients with CML who have not achieved optimal response after 3 months of treatment with imatinib will have a better response by switching to dasatinib compared to staying on their original imatinib regimen.

NCT01593254 Chronic Phase Chronic Myeloid Leukemia Drug: Imatinib Drug: Dasatinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Chronic-Phase
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

Patients with no evidence of clonal evolution, including those patients whose cytogenetic testing fails or bone marrow aspiration is a dry tap at 3 months, are eligible for the study - Subjects with less than CHR after 3 months of imatinib treatment or lost CHR after initial achievement - Documented T315I/A, F317L, or V299L mutations (if already available - not required for screening) - A serious uncontrolled medical disorder or active infection that would impair the ability of the subject to receive protocol therapy For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com. --- T315I --- --- F317L --- --- V299L ---

Patients with no evidence of clonal evolution, including those patients whose cytogenetic testing fails or bone marrow aspiration is a dry tap at 3 months, are eligible for the study - Subjects with less than CHR after 3 months of imatinib treatment or lost CHR after initial achievement - Documented T315I/A, F317L, or V299L mutations (if already available - not required for screening) - A serious uncontrolled medical disorder or active infection that would impair the ability of the subject to receive protocol therapy Chronic Phase Chronic Myeloid Leukemia Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Chronic-Phase null --- T315I --- --- F317L --- --- V299L ---

Primary Outcomes

Description: Major Molecular Response, is defined as a 3-log reduction in BCR-ABL transcripts from the standardized baseline, which represents 100% on the international scale, so a 3-log reduction is fixed at 0.1% for MMR; N/A = not applicable. 95% CI is Clopper-Pearson(Exact) two-sided 95% confidence intervals. P-value is based on Cochran-Mantel-Haenszel (CMH) test stratified by Sokal score(high, intermediate, low, and unknown) and time between 3 month molecular analysis and randomization (<=4 weeks vs >4 weeks). Month 12 is calculated fro

Measure: Percentage of Patients Achieving Major Molecular Response (MMR) After 12 Months of CML Treatment

Time: At 12 months after Day 1 initiation of 1st line treatment with imatinib or imatinib at any dose, after less than optimal response to first-line imatinib.

Secondary Outcomes

Description: Time to MMR is how fast patients achieve optimal response. It is the time between randomization date and first date that MMR criteria are satisfied.

Measure: Median Time to Major Molecular Response (MMR)

Time: Up to 10 years

Description: Time to MR^4.5 is how fast patients achieve a deeper response. It is the time between randomization date and first date that MR^4.5 criteria are satisfied.

Measure: Time to Molecular Response (MR)^4.5

Time: Up to 10 years

Description: PFS is how long patients are likely to live without progression of their disease. It is the time from randomization date to progression date or death date, whichever occurs first.

Measure: Progression Free Survival (PFS)

Time: Up to 10 years

Description: OS is how long patients are likely to remain alive. It is the time from randomization date to death date.

Measure: Overall Survival (OS)

Time: Up to 10 years

7 Discontinuation of Dasatinib in Patients With Chronic Myeloid Leukemia in Chronic Phase Who Have Maintained Complete Molecular Remission for Two Years; Dasatinib Stop Trial

The purpose of this study is to assess whether dasatinib can be discontinued without occurrence of molecular relapse in patients with chronic myeloid leukemia in chronic phase in complete molecular remission (CMR) while on dasatinib.

NCT01627132 Chronic Myeloid Leukemia Drug: Dasatinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

- Patients with mutation of T315I, F317L, V299L. --- T315I --- --- F317L --- --- V299L ---

Primary Outcomes

Measure: The overall probability of maintenance of complete molecular remission at 12 months after stopping dasatinib.

Time: at 12 months

Secondary Outcomes

Measure: Rate of complete molecular remission that will be sustained after dasatinib rechallenge.

Time: at 12 months

8 A Phase 3, Multi-center, Open-label, Randomized Study of Oral ABL001 Versus Bosutinib in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP), Previously Treated With 2 or More Tyrosine Kinase Inhibitors

The purpose of this pivotal study is to compare the efficacy of ABL001 with that of bosutinib in the treatment of patients with CML-CP having previously been treated with a minimum of two prior ATP-binding site TKIs. Patients intolerant to the most recent TKI therapy must have BCR-ABL1 ratio > 0.1% IS at screening and patients failing their most recent TKI therapy must meet the definition of treatment failure as per the 2013 ELN guidelines. Patients with documented treatment failure while on bosutinib treatment will have the option to switch to asciminib treatment within 96 weeks after the last patient has been randomized on study.

NCT03106779 Chronic Myelogenous Leukemia Drug: ABL001 Drug: Bosutinib
MeSH: Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive
HPO: Chronic myelogenous leukemia Leukemia Myeloid leukemia

- Three months after the initiation of therapy: No CHR or > 95% Ph+ metaphases - Six months after the initiation of therapy: BCR-ABL1 ratio > 10% IS and/or > 65% Ph+ metaphases - Twelve months after initiation of therapy: BCR-ABL1 ratio > 10% IS and/or > 35% Ph+ metaphases - At any time after the initiation of therapy, loss of CHR, CCyR or PCyR - At any time after the initiation of therapy, the development of new BCR-ABL1 mutations which potentially cause resistance to study treatment - At any time after the initiation of therapy, confirmed loss of MMR in 2 consecutive tests, of which one must have a BCR-ABL1 ratio ≥ 1% IS - At any time after the initiation of therapy, new clonal chromosome abnormalities in Ph+ cells: CCA/Ph+ - Intolerance is defined as: - Non-hematologic intolerance: Patients with grade 3 or 4 toxicity while on therapy, or with persistent grade 2 toxicity, unresponsive to optimal management, including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) - Hematologic intolerance: Patients with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses recommended by manufacturer Exclusion Criteria: Known presence of the T315I or V299L mutation at any time prior to study entry Known second chronic phase of CML after previous progression to AP/BC Previous treatment with a hematopoietic stem-cell transplantation Patient planning to undergo allogeneic hematopoietic stem cell transplantation Cardiac or cardiac repolarization abnormality, including any of the following: - History within 6 months prior to starting study treatment of myocardial infarction (MI), angina pectoris, coronary artery bypass graft (CABG) - Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block) - QTcF at screening ≥450 msec (male patients), ≥460 msec (female patients) - Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following: - Risk factors for Torsades de Pointes (TdP) including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/symptomatic bradycardia - Concomitant medication(s) with a known risk of Torsades de Pointes per www.crediblemeds.org that cannot be discontinued or replaced 7 days prior to starting study drug by safe alternative medication. --- T315I --- --- V299L ---

Primary Outcomes

Description: To compare the MMR rate of ABL001 versus bosutinib

Measure: Major Molecular Response (MMR) rate

Time: at 24 weeks

Secondary Outcomes

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Major Molecular Response (MMR) rate

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib. Cytogenic response will include Complete, Partial, Major, Minor, Minimal and no response.

Measure: Complete Cytogenetic response rate

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Time to MMR

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Duration of MMR

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Time to CCyR

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Duration of CCyR

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Time to treatment failure

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Progression free survival

Time: 96 weeks after the last patient received the first study dose

Description: To compare additional parameters of the efficacy of ABL001 versus bosutinib

Measure: Overall survival

Time: 96 weeks after the last patient received the first study dose

Description: To characterize the PK of ABL001 in the CML-CP population

Measure: Trough plasma concentrations

Time: 96 weeks after the last patient received the first study dose

Description: To characterize the PK of ABL001 in the CML-CP population

Measure: PK parameter: Cmax,

Time: 96 weeks after the last patient received the first study dose

Description: To characterize the PK of ABL001 in the CML-CP population

Measure: PK parameter: Tmax

Time: 96 weeks after the last patient received the first study dose

Description: To characterize the PK of ABL001 in the CML-CP population

Measure: PK parameter: AUC0-12h

Time: 96 weeks after the last patient received the first study dose

Description: To characterize the PK of ABL001 in the CML-CP population

Measure: PK parameter: CL/F

Time: 96 weeks after the last patient received the first study dose

9 Phase I Trial Adding Ruxolitinib to a Combination of Dasatinib Plus Dexamethasone in Remission Induction Therapy in Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Patients Aged 40 Years or Older.

The purpose of this study is to test the safety of a new combination of three oral drugs in Ph+ ALL. These drugs are dexamethasone, dasatinib, and ruxolitinib. All three drugs have been studied before in humans. This is a phase I study in which ruxolitinib dose will start low for the first patient together with dexamethasone plus dasatinib. If this dose does not cause a bad side effect, the ruxolitinib dose will slowly be made higher as new patients take part in the study. This will help the investigators find the right dose of ruxolitinib to give together with dexamethasone and dasatinib that will be used in future studies

NCT02494882 Acute Lymphoblastic Leukemia Drug: Ruxolitinib Drug: Dasatinib Drug: Dexamethasone
MeSH: Leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Lymphoid Philadelphia Chromosome
HPO: Leukemia Lymphoid leukemia

L248R, L248V, Q252H, E255K, V299L, T315A, T315I, F317C, F317L, F317S, F317V) - Relapsed or refractory Ph-like ALL without prior exposure to dasatibin and with mutations or rearrangements of genes conferring sensitivity to dasatibin (ABL, CSF1R, PDGFRB) or ruxolitinib (CRLF2, JAK3, EPOR, TSLP) - Newly diagnosed or relapsed CML in lymphoid blast crisis - Confirmation of Philadelphia chromosome positivity by cytogenetics (karyotype/FISH) and/or molecular tests (BCR-ABL1 transcripts) - Acceptable end-organ function, except for documented exclusions for organ function compromise due to ALL itself - ECOG performance status ≤ 2 - Men and women of childbearing potential must be willing to practice an effective method of birth control during treatment and for at least 4 months following treatment on study Exclusion Criteria: - Ph-negative ALL - Patients with dominant leukemic clone bearing documented bcr-abl mutations enabling bcr-abl TKI resistance at diagnosis - Mature B-cell (Burkitt's) ALL - Serum creatinine > 1.5x ULN and calculated creatinine clearance, based on a 24-hour urine collection, < 30 mL/min--unless related to ALL/tumor lysis syndrome and able to be corrected - Direct Bilirubin > 2x ULN; AST/ALT > 10x ULN, unless related to ALL liver infiltration. --- L248R --- --- L248V --- --- Q252H --- --- E255K --- --- V299L ---

Primary Outcomes

Description: is to be evaluated by using a combination of criteria. Molecular remissions will be defined by standard criteria for BCR-ABL1 and IGH qRT-PCR. Flow cytometric assessment of MRD will be defined by standard flow cytometry criteria

Measure: Clinical response

Time: 2 years

Secondary Outcomes

Description: Molecular remission status will be defined by undetectable BCR-ABL1 transcripts and/or IGH clonal gene rearrangement in bone marrow aspirate (BMA) examination as determined by qRT-PCR in CLIA laboratory.

Measure: Complete Molecular Remission (CMR) rate

Time: 2 years


HPO Nodes


Chronic myelogenous leukemia
Genes 5
MPL BCR JAK2 KIT THPO
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
Myeloid leukemia
Genes 12
GATA2 F13A1 CBL ARHGAP26 F13B KRAS PTPN11 SAMD9L KIT SETBP1 NF1 NRAS
Lymphoid leukemia