Status:
UNKNOWN
Phase 2, Open-Label, Single Arm Study, With BST-236 in Adults With R/R AML or Higher-Risk MDS
Lead Sponsor:
BioSight Ltd.
Conditions:
AML, Adult
MDS
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
An open label multi center study to assess the safety and efficacy of BST-236 as single agent in adult patients unfit for standard therapy with Acute Myeloid Leukemia (AML) or higher-risk (HR) Myelody...
Eligibility Criteria
Inclusion
- Documented diagnosis of MDS, according to World Health Organization (WHO) classification and Revised International Prognostic Scoring System (IPSS-R) overall score ≥ 4.5 Or
- Diagnosed AML according to the 2016 revision to the WHO classification of myeloid neoplasms and acute leukemia: ≥20% blasts in peripheral blood or bone marrow
- Adult ≥18 years of age
- MDS relapse following treatment with azacitidine or decitabine Or
- MDS failure to achieve complete or partial response or stable disease with hematologic improvement after at least 4 cycles of azacitidine or decitabine, all within the last 1 year Or
- MDS progression while on azacitidine or decitabine treatment irrespective of the number of cycles the patient has received Or
- AML relapse after initial CR/CRi/CRh following treatment with: azacitidine, decitabine, Low-Dose Ara-C (LDAC) , venetoclax+HMA, or venetoclax+LDAC Or
- AML failure to achieve CR, CRh or CRi following at least 4 cycles of azacitidine or decitabine or 2 cycles of venetoclax+HMA or venetoclax+LDAC within the last 1 year.
- Or
- AML progression while on azacitidine, decitabine, LDAC, venetoclax+HMA, venetoclax+LDAC, irrespective of the number of cycles the patient has received.
- Not able to receive an allogeneic bone marrow transplantation (BMT) at the time of study enrolment.
- Not eligible for intensive chemotherapy;
- Age ≥75 years Or
- Age ≥18 years with at least one of the following comorbidities:
- Significant heart or lung comorbidities, as reflected by at least one of the following:
- Left ventricular ejection fraction (LVEF) ≤50%
- Lung diffusing capacity for carbon monoxide (DLCO) ≤65% of expected
- Forced expiratory volume in 1 second (FEV1) ≤65% of expected
- Chronic stable angina or congestive heart failure controlled with medication
- Other comorbidity or conditions that the Investigator judges as incompatible with intensive chemotherapy, which must be documented
- ECOG=2
- Creatinine clearance (estimated by the Modification of Diet in Renal Disease (MDRD) equation or measured by 24 hours urine collection) ≥45 mL/min
- Liver enzymes (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤2.5 times the upper limits of normal (ULN), unless attributed to leukemia (in AML patients)
- Total bilirubin ≤3 XULN unless due to Gilbert disease
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Women of reproductive potential must have a negative serum pregnancy test within 48 hours prior to the first day of any BST-236 treatment course
- Women of reproductive potential must use two forms of effective birth control methods starting from at least 1 month prior to BST-236 first dose and until 3 months following the last BST-236 administration day (acceptable methods of birth control in this study include: surgical sterilization, intrauterine devices, oral contraceptives, contraceptive patch, long-acting injectable contraceptives, or double-barrier method condom or diaphragm with spermicide)
- Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 3 months following the last dose of study drug
- Subject must voluntarily sign and date an informed consent, approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB), prior to the initiation of any screening or study-specific procedures
- Patient must be able to adhere to the study visit schedule and other protocol requirements
- Exclusion Criteria:
- MDS or AML evolving from a pre-existing myeloproliferative neoplasm (MPN)
- MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN
- Acute promyelocytic leukemia
- Previous treatment for AML or MDS with drugs other than HMA or LDAC or combinations of venetoclax with either HMA or LDAC
- Previous allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation
- Participation in a previous clinical trial involving use of an investigational drug within 30 days or at least 5 half-lives of tested drug (whichever is shorter) of study day 1
- Peripheral White Blood Cell (WBC) count \>30,000 /µL in the 48 hours prior to first BST-236 dose administration. Hydroxyurea administration or leukapheresis is permitted to meet this criterion
- Administration of HMA, LDAC, or venetoclax within 14 days prior to Study Day 1
- Previous treatment with cytarabine at a dose higher than 20 mg/ m2/d
- Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment)
- Any medical or surgical condition, presence of laboratory abnormalities or psychiatric illness that may preclude safe and complete study participation based on the Investigator's judgment
- Diagnosis of malignant disease (other than AML) within the previous 12 months (excluding basal cell carcinoma of the skin without complications, "in-situ" carcinoma, or other local malignancy excised or irradiated with a high probability of cure and not treated with systemic or topical chemotherapy)
- Surgical procedure, excluding central venous catheter placement or other minor procedures (e.g. skin biopsy) in the 14 days prior to first BST-236 dose administration
- History of allergic reactions attributed to compounds of similar chemical composition as BST-236 and/or cytarabine
- Life expectancy shorter than 3 months attributed to any known medical condition other than AML/MDS
- In 12 leads ECG, corrected QT interval (QTc)\>480msec or history of QT prolongation or Torsades de pointes
Exclusion
Key Trial Info
Start Date :
November 14 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 14 2024
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT04749355
Start Date
November 14 2021
End Date
March 14 2024
Last Update
March 9 2023
Active Locations (4)
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1
Ochsner LSU Health Shreveport - Academic Medical Center
Shreveport, Louisiana, United States, 71104
2
The University of Texas MD Anderson Cancer center
Houston, Texas, United States, 77030
3
University of Virginia Cancer Center
Charlottesville, Virginia, United States, 22903
4
Seattle Cancer Care Alliance
Seattle, Washington, United States, 98109