Back to resultsA Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Primary Purpose
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
BTX-A51
Sponsored by
About this trial
This is an interventional treatment trial for Acute Myeloid Leukemia
Eligibility Criteria
Inclusion Criteria:
- Demonstration of understanding and voluntarily signing of an informed consent form
- Age ≥ 18 years
- Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to the World Health Organization classification and, with respect to MDS, that is high risk; participants must have refractory or relapsed disease and be ineligible for or have exhausted standard therapeutic options that would otherwise be likely to provide clinical benefit
- Eastern Cooperative Oncology Group performance status ≤ 2 and life expectancy of ≥ 6 weeks
- Adequate organ function (Grade 1 serum creatinine; Grade 1 total bilirubin; aspartate aminotransferase and/or alanine transaminase ≤ 2 × ULN)
- Females of childbearing age must not be pregnant at time of Screening/beginning of treatment and agree to either abstain from sexual intercourse or use highly effective methods of contraception (for up to 3 months after last dose of study drug)
- Males sexually active with a woman of childbearing age must agree to use barrier method of birth control during and after the study (up to 3 months after last dose of study drug)
Exclusion Criteria:
- Diagnosis of acute promyelocytic leukemia
- White blood cell count > 20 x 10^9/L
- Receipt of cancer chemotherapy (other than hydroxyurea) within 2 weeks prior to the start of study drug
- In participants who have undergone autologous or allogeneic stem cell transplantation: transplantation within the 3 months prior to Screening; active graft-versus-host disease requiring anything other than topical corticosteroids and budesonide; treatment with systemic immunosuppressive medications including high-dose steroids (≥ 20 mg prednisolone or equivalent per day), or calcineurin inhibitors (e.g., cyclosporine, tacrolimus) for at least 1 week prior to Screening, and sirolimus, mycophenylate mofetil, azathioprine, or ruxolitinib for at least 2 weeks prior to Screening
- Immediate life-threatening severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
- Persistent toxicities from prior treatment of Grade 2 or higher
- Active uncontrolled systemic fungal, bacterial, mycobacterial, or viral infection
- Clinically significant cardiac disease
- Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally
- Any other concurrent medical condition or disease that is likely to interfere with study procedures or results, or that, in the opinion of the Investigator, would constitute a hazard for participating in this study
- If female, pregnant or breastfeeding
Sites / Locations
- City of Hope National Medical CenterRecruiting
- Memorial Sloan-Kettering Cancer CenterRecruiting
- The University of Texas MD Anderson Cancer CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
Phase 1a (Dose Escalation Phase)
Phase 1b (Cohort Expansion Phase)
Continued Treatment Phase
Arm Description
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). The BTX-A51 starting dose for Cohort 1 is 1 mg, to be given 5 days per week (maximum weekly dose of 5 mg). Beginning with Cohort 2, doses are intended to be administered 3 days per week. Barring dose-limiting toxicity (DLT), sequential dose escalation of BTX-A51 is planned with up to a total of eight dose levels to a maximum of 20 mg (60 mg/week); on the basis of these an MTD will be identified. The numbers of participants and actual doses administered will be determined using a Bayesian optimal interval (BOIN) design to determine the DLTs and MTD of BTX-A51.
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). Phase 1b will continue at the MTD or the highest dose achieved in Phase 1a.
Participants who complete one cycle of BTX-A51 treatment in either Phase 1a or Phase 1b will be offered continued access to treatment for up to eight 28-day cycles if the Investigator determines that the benefit outweighs the risk. Dosing will continue at the assigned dose or may be increased (not to exceed a level already tolerated by at least one participant if Phase 1a is ongoing or the MTD/recommended Phase 2 dose if already established).
Outcomes
Primary Outcome Measures
Incidence of dose-limiting toxicities (DLTs)
A DLT is defined as a severe or clinically significant adverse event (AE) or abnormal laboratory value (Grade 3 or greater, unless otherwise specified) starting with the first dose on Cycle 1 Day 1, unless it is clearly related to disease progression, intercurrent illness, preexisting condition, or concomitant medications. DLTs are based on National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Number of participants with non-serious AEs and serious AEs (SAEs)
The severity/intensity of AEs will be graded based upon the participant's symptoms according to the NCI CTCAE Version 5.0
Number of participants with laboratory abnormalities and/or AEs
Number of participants with potentially clinically significant laboratory values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs
Number of participants with potentially clinically significant 12-lead ECG findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Number of participants with echocardiogram (ECHO) abnormalities and/or AEs
Number of participants with potentially clinically significant ECHO abnormalities and/or AEs, such as elevated or abnormal left ventricular ejection fraction (LVEF) or abnormal Global Longitudinal Strain (GLS)
Number of participants with vital sign abnormalities and/or AEs
Number of participants with potentially clinically significant vital sign values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Number of participants with physical examination abnormalities and/or AEs
Number of participants with potentially clinically significant physical examination findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Maximum tolerated dose (MTD)
The DLTs are to be evaluated for determination of the MTD. The MTD will be the dose for which the isotonic estimate of the toxicity rate is closest to the target toxicity rate of 0.3. If there are ties, the higher dose level when the isotonic estimate is lower than the target toxicity rate will be identified and the lower dose level selected when the isotonic estimate is greater than or equal to the target toxicity rate.
Recommended Phase 2 dose (RP2D)
The DLTs are to be evaluated based on cumulative safety/PK data in participants treated in Phase 1b for determination of the RP2D (which may or may not differ from the MTD)
Secondary Outcome Measures
Complete remission (CR) for participants with acute myeloid leukemia (AML)
CR is defined as free of all symptoms related to leukemia and with an absolute neutrophil count (ANC) ≥ 1.0 × 10^9/L (1000/µL), platelet count ≥ 100 × 10^9/L (100,000/µL), and normal bone marrow (BM) with < 5 percent blasts; absence of circulating blasts and blasts with Auer rods
Complete remission with incomplete blood count recovery (CRi) for participants with AML
CRi is defined as all CR criteria except for residual neutropenia (ANC < 1.0 × 10^9/L [1000/µL]) or thrombocytopenia (platelet count < 100 × 10^9/L [100,000/µL])
Morphologic leukemia-free state (MLFS) for participants with AML
MLFS is defined as BM blasts < 5 percent; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required. Marrow should not barely be "aplastic;" at least 200 cells should be enumerated or cellularity should be at least 10 percent.
Partial remission (PR) for participants with AML
PR is defined as all hematologic criteria of CR; decrease of BM blast percentage to 5 - 25 percent; and decrease of pretreatment BM blast percentage by at least 50 percent
Complete remission (CR) for participants with high-risk myelodysplastic syndrome (MDS)
CR is defined as free of all symptoms related to leukemia and with an ANC ≥ 1.0 × 10^9/L, platelet count ≥ 100 × 10^9/L, BM ≤ 5 percent myeloblasts, with normal maturation of all cell lines, hemoglobin ≥ 11 g/dL, and no blasts in peripheral blood (PB)
Partial remission (PR) for participants with high-risk MDS
PR is defined as all CR criteria with ≥ 50 percent decrease in BM blasts over pre-treatment (but still > 5 percent)
Hematologic improvement (HI) for participants with high-risk MDS
The International Working Group criteria for HI defining specific responses of cytopenias in the three hematopoietic lineages: erythroid (HI-E), platelet (HI-P), and neutrophil (HI-N)
Overall survival and event-free survival in participants with AML or high-risk MDS
Follow-up will occur by telephone contact once every 3 months for assessment of survival status and bone marrow transplant (BMT) conditioning or other new antineoplastic therapies since discontinuation of study drug; the cause of death will be documented. Disease assessment will be collected for participants who discontinue study medication due to any reason other than progression or death. If a participant has not progressed or died, progression-free survival is censored at the date of last follow up. The distribution of time-to-event endpoints will be estimated using the method of Kaplan and Meier.
PK parameter: Maximum observed plasma concentration (Cmax)
Maximum observed plasma drug concentration after the first dose
PK parameter: Time of maximum plasma concentration (Tmax)
Time to reach Cmax
PK parameter: Plasma CmaxD5
Maximum observed plasma drug concentration after the dose on Day 5
PK parameter: Plasma TmaxD5
Time to reach CmaxD5
PK parameter: C0
Pre-dose concentrations in PK blood samples on dosing days
PK parameter: AUC0-24
Area under the curve from time 0 to 24 hours after the first dose
PK parameter: AUC0-24D5
AUC from time 0 to 24 hours after the dose on Day 5
PK parameter: Terminal elimination rate constant (Kel)
Calculated by linear least-squares regression analysis from a semi-log plot of the plasma concentration versus time curve after the Day 5 dose
PK parameter: Terminal elimination phase half-life (t1/2)
Estimated after the Day 5 dose, calculated as 0.693/Kel
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04243785
Brief Title
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Official Title
A First-In-Human, Open-Label, Escalating Multiple-Dose Study to Evaluate the Safety, Toxicity, and Pharmacokinetics of BTX-A51 Capsules in Patients With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Recruiting
Study Start Date
January 6, 2020 (Actual)
Primary Completion Date
March 2023 (Anticipated)
Study Completion Date
March 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioTheryX, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).
The study will be done in two phases. Phase 1a of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants will be enrolled in Phase 1b of this study for additional experience with safety and efficacy, and to determine the recommended Phase 2 dose (RP2D) which may or may not be different from the MTD.
Continued treatment will be available under this study protocol for up to eight 28-day cycles (Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk.
Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukemia, Myelodysplastic Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Phase 1a (Dose Escalation Phase)
Arm Type
Experimental
Arm Description
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). The BTX-A51 starting dose for Cohort 1 is 1 mg, to be given 5 days per week (maximum weekly dose of 5 mg). Beginning with Cohort 2, doses are intended to be administered 3 days per week. Barring dose-limiting toxicity (DLT), sequential dose escalation of BTX-A51 is planned with up to a total of eight dose levels to a maximum of 20 mg (60 mg/week); on the basis of these an MTD will be identified. The numbers of participants and actual doses administered will be determined using a Bayesian optimal interval (BOIN) design to determine the DLTs and MTD of BTX-A51.
Arm Title
Phase 1b (Cohort Expansion Phase)
Arm Type
Experimental
Arm Description
Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). Phase 1b will continue at the MTD or the highest dose achieved in Phase 1a.
Arm Title
Continued Treatment Phase
Arm Type
Experimental
Arm Description
Participants who complete one cycle of BTX-A51 treatment in either Phase 1a or Phase 1b will be offered continued access to treatment for up to eight 28-day cycles if the Investigator determines that the benefit outweighs the risk. Dosing will continue at the assigned dose or may be increased (not to exceed a level already tolerated by at least one participant if Phase 1a is ongoing or the MTD/recommended Phase 2 dose if already established).
Intervention Type
Drug
Intervention Name(s)
BTX-A51
Intervention Description
Orally administered capsules available in strengths of 0.5 mg, 1.0 mg, and 2.0 mg.
Primary Outcome Measure Information:
Title
Incidence of dose-limiting toxicities (DLTs)
Description
A DLT is defined as a severe or clinically significant adverse event (AE) or abnormal laboratory value (Grade 3 or greater, unless otherwise specified) starting with the first dose on Cycle 1 Day 1, unless it is clearly related to disease progression, intercurrent illness, preexisting condition, or concomitant medications. DLTs are based on National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with non-serious AEs and serious AEs (SAEs)
Description
The severity/intensity of AEs will be graded based upon the participant's symptoms according to the NCI CTCAE Version 5.0
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with laboratory abnormalities and/or AEs
Description
Number of participants with potentially clinically significant laboratory values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with 12-lead electrocardiogram (ECG) abnormalities and/or AEs
Description
Number of participants with potentially clinically significant 12-lead ECG findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with echocardiogram (ECHO) abnormalities and/or AEs
Description
Number of participants with potentially clinically significant ECHO abnormalities and/or AEs, such as elevated or abnormal left ventricular ejection fraction (LVEF) or abnormal Global Longitudinal Strain (GLS)
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with vital sign abnormalities and/or AEs
Description
Number of participants with potentially clinically significant vital sign values; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Number of participants with physical examination abnormalities and/or AEs
Description
Number of participants with potentially clinically significant physical examination findings; toxicity will be graded and reported according to the NCI CTCAE Version 5.0
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Maximum tolerated dose (MTD)
Description
The DLTs are to be evaluated for determination of the MTD. The MTD will be the dose for which the isotonic estimate of the toxicity rate is closest to the target toxicity rate of 0.3. If there are ties, the higher dose level when the isotonic estimate is lower than the target toxicity rate will be identified and the lower dose level selected when the isotonic estimate is greater than or equal to the target toxicity rate.
Time Frame
Up to 28 days (one cycle) for each dosing cohort in Phase 1a
Title
Recommended Phase 2 dose (RP2D)
Description
The DLTs are to be evaluated based on cumulative safety/PK data in participants treated in Phase 1b for determination of the RP2D (which may or may not differ from the MTD)
Time Frame
Up to 28 days (one cycle) for each dosing cohort in Phase 1b
Secondary Outcome Measure Information:
Title
Complete remission (CR) for participants with acute myeloid leukemia (AML)
Description
CR is defined as free of all symptoms related to leukemia and with an absolute neutrophil count (ANC) ≥ 1.0 × 10^9/L (1000/µL), platelet count ≥ 100 × 10^9/L (100,000/µL), and normal bone marrow (BM) with < 5 percent blasts; absence of circulating blasts and blasts with Auer rods
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Complete remission with incomplete blood count recovery (CRi) for participants with AML
Description
CRi is defined as all CR criteria except for residual neutropenia (ANC < 1.0 × 10^9/L [1000/µL]) or thrombocytopenia (platelet count < 100 × 10^9/L [100,000/µL])
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Morphologic leukemia-free state (MLFS) for participants with AML
Description
MLFS is defined as BM blasts < 5 percent; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required. Marrow should not barely be "aplastic;" at least 200 cells should be enumerated or cellularity should be at least 10 percent.
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Partial remission (PR) for participants with AML
Description
PR is defined as all hematologic criteria of CR; decrease of BM blast percentage to 5 - 25 percent; and decrease of pretreatment BM blast percentage by at least 50 percent
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Complete remission (CR) for participants with high-risk myelodysplastic syndrome (MDS)
Description
CR is defined as free of all symptoms related to leukemia and with an ANC ≥ 1.0 × 10^9/L, platelet count ≥ 100 × 10^9/L, BM ≤ 5 percent myeloblasts, with normal maturation of all cell lines, hemoglobin ≥ 11 g/dL, and no blasts in peripheral blood (PB)
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Partial remission (PR) for participants with high-risk MDS
Description
PR is defined as all CR criteria with ≥ 50 percent decrease in BM blasts over pre-treatment (but still > 5 percent)
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Hematologic improvement (HI) for participants with high-risk MDS
Description
The International Working Group criteria for HI defining specific responses of cytopenias in the three hematopoietic lineages: erythroid (HI-E), platelet (HI-P), and neutrophil (HI-N)
Time Frame
Up to a total of eight 28-day cycles (approximately 224 days)
Title
Overall survival and event-free survival in participants with AML or high-risk MDS
Description
Follow-up will occur by telephone contact once every 3 months for assessment of survival status and bone marrow transplant (BMT) conditioning or other new antineoplastic therapies since discontinuation of study drug; the cause of death will be documented. Disease assessment will be collected for participants who discontinue study medication due to any reason other than progression or death. If a participant has not progressed or died, progression-free survival is censored at the date of last follow up. The distribution of time-to-event endpoints will be estimated using the method of Kaplan and Meier.
Time Frame
Up to 2 years after participant's last dose of BTX-A51 or upon death, whichever occurs first
Title
PK parameter: Maximum observed plasma concentration (Cmax)
Description
Maximum observed plasma drug concentration after the first dose
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: Time of maximum plasma concentration (Tmax)
Description
Time to reach Cmax
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: Plasma CmaxD5
Description
Maximum observed plasma drug concentration after the dose on Day 5
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: Plasma TmaxD5
Description
Time to reach CmaxD5
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: C0
Description
Pre-dose concentrations in PK blood samples on dosing days
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: AUC0-24
Description
Area under the curve from time 0 to 24 hours after the first dose
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: AUC0-24D5
Description
AUC from time 0 to 24 hours after the dose on Day 5
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: Terminal elimination rate constant (Kel)
Description
Calculated by linear least-squares regression analysis from a semi-log plot of the plasma concentration versus time curve after the Day 5 dose
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
Title
PK parameter: Terminal elimination phase half-life (t1/2)
Description
Estimated after the Day 5 dose, calculated as 0.693/Kel
Time Frame
During Cycle 1 for each dosing cohort in Phase 1a on Days 1, 3, and 5 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose); Days 2, 4, 6, 7, 8; and Day 15 (pre-dose and approximately 1, 2, 3, 5, 8, and 12 hours post-dose)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Demonstration of understanding and voluntarily signing of an informed consent form
Age ≥ 18 years
Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to the World Health Organization classification and, with respect to MDS, that is high risk; participants must have refractory or relapsed disease and be ineligible for or have exhausted standard therapeutic options that would otherwise be likely to provide clinical benefit
Eastern Cooperative Oncology Group performance status ≤ 2 and life expectancy of ≥ 6 weeks
Adequate organ function (Grade 1 serum creatinine; Grade 1 total bilirubin; aspartate aminotransferase and/or alanine transaminase ≤ 2 × ULN)
Females of childbearing age must not be pregnant at time of Screening/beginning of treatment and agree to either abstain from sexual intercourse or use highly effective methods of contraception (for up to 3 months after last dose of study drug)
Males sexually active with a woman of childbearing age must agree to use barrier method of birth control during and after the study (up to 3 months after last dose of study drug)
Exclusion Criteria:
Diagnosis of acute promyelocytic leukemia
White blood cell count > 20 x 10^9/L
Receipt of cancer chemotherapy (other than hydroxyurea) within 2 weeks prior to the start of study drug
In participants who have undergone autologous or allogeneic stem cell transplantation: transplantation within the 3 months prior to Screening; active graft-versus-host disease requiring anything other than topical corticosteroids and budesonide; treatment with systemic immunosuppressive medications including high-dose steroids (≥ 20 mg prednisolone or equivalent per day), or calcineurin inhibitors (e.g., cyclosporine, tacrolimus) for at least 1 week prior to Screening, and sirolimus, mycophenylate mofetil, azathioprine, or ruxolitinib for at least 2 weeks prior to Screening
Immediate life-threatening severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
Persistent toxicities from prior treatment of Grade 2 or higher
Active uncontrolled systemic fungal, bacterial, mycobacterial, or viral infection
Clinically significant cardiac disease
Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally
Any other concurrent medical condition or disease that is likely to interfere with study procedures or results, or that, in the opinion of the Investigator, would constitute a hazard for participating in this study
If female, pregnant or breastfeeding
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Tracy Lawhon, JD
Phone
858-699-2239
Email
tlawhon@biotheryx.com
First Name & Middle Initial & Last Name or Official Title & Degree
Edgar Bautista
Email
ebautista@biotheryx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tracy Lawhon, JD
Organizational Affiliation
BioTheryX, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
City of Hope National Medical Center
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brian Ball, MD
Phone
626-218-4784
Email
brball@coh.org
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eytan Stein, MD
Phone
646-608-3749
Email
SteinE@mskcc.org
Facility Name
The University of Texas MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gautam Borthakur, MD
Phone
713-563-1586
Email
Gborthak@mdanderson.org
12. IPD Sharing Statement
Learn more about this trial
A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
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