BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorEric Stephen Winer, MD
About this trial
The goal of this research study is to find the safest and most effective dose of the study drug, BXCL701, for the treatment of Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS).
The names of the study drugs involved in this study are/is:
* BXCL701
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18 and older
Relapsed AML: as evidence by ≥5% myeloblasts in the bone marrow, or reappearance of blasts in the peripheral blood
Refractory AML: ≤2 prior induction regimens (example: patients who receive 7 + 3 followed by 5 + 2 would count as one induction regimen) OR
Subjects with WHO defined myelodysplastic syndrome with excess blasts-2 (MDS-EB-2) as defined by blast count between 10% - 19% in the bone marrow or peripheral blood blasts 5% - 19% or Auer rods noted and who are refractory or relapsed after at least 4 cycles of a hypomethylating agent (azacitidine, decitabine, or oral decitabine/cedazuridine)
Disqualifiers
Subjects who have known Acute Promyelocytic Leukemia.
Subjects with active CNS involvement with AML.
Participants who have had chemotherapy, other investigational therapy, immunotherapy, or radiotherapy within 2 weeks or 5 half-lives from prior therapy, whichever is longer, prior to the first dose of study medication. Hydroxyurea is allowed with no required washout, and hydroxyurea may be administered for the first cycle of the protocol for patients who have proliferative disease (WBC <25K) with a maximum allowed dose of 6 g per day.
Participants who have received oral tyrosine kinase inhibitors (TKIs) within two weeks or 5 half-lives (whichever is longer) of the first dose of study medication
Trial design
Design model
Single group
Treatments tested in this trial
BXCL701
DrugTablet, taken Orally
Treatment groups
24 Participants
are divided into 1 treatment groupGroup A: Dose Escalation BXCL701Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Maximum Tolerated Dose (MTD)
Defined as the highest dose at which fewer than one-third of patients experience a dose-level toxicity (DLT). MTD will be used to inform recommended Phase II dose. If no DLTs are observed, the MTD is not reached.
Time frame
From initiation of therapy to day 28, up to 35 days2
Number of Participants with treatment related Adverse Events per CTCAE 5.0
Assessed using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame
4 weeks up to 1 yearSecondary outcomes
1
Complete Remission (CR) Rate
Proportion of patients who have the response, criteria are based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for Acute Myeloid Leukemia (APPENDIX D) and Myelodysplastic Syndrome (APPENDIX F).
Time frame
Disease assesment at baseline, cycle 1, 3 , 8, 15 , 22, and day 1 of every cycle and up to 12 cycles, corresponding to a treatment duration up to one year (each cycle is 28 days).2
Complete Response with Incomplete Count Recovery (CRi) Rate
Proportion of patient who has the response, criteria are based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for Acute Myeloid Leukemia (APPENDIX D) and Myelodysplastic Syndrome (APPENDIX F).
Time frame
Disease assesment at baseline, cycle 1, 3 , 8, 15 , 22, and day 1 of every cycle and up to 12 cycles, corresponding to a treatment duration up to one year (each cycle is 28 days).3
Partial Response (PR) Rate
Based upon International Working Group (IWG) and European LeukemiaNet (ELN) criteria for AML (APPENDIX D) and MDS (APPENDIX F). Defined as decrease by \>= 50% in blast percentage to 5-25% or to \<= 5% with auer rods present in marrow; normalization neutrophil count \>= 1000μL and platelet count \>= 100000μL.
Time frame
Disease assesment at baseline, cycle 1, 3 , 8, 15 , 22, and day 1 of every cycle and up to 12 cycles, corresponding to a treatment duration up to one year (each cycle is 28 days)..4
Morphologic Leukemia-free State (MLFS) Rate
Evaluated only in AML patients per the 2017 ELN criteria (Dohner, Blood 2017, PMID: 27895058), defined as: Bone marrow blasts \<5%; absence of blasts with Auer rods; absence of extramedullary disease; no hematologic recovery required.
Time frame
Disease assesment at baseline, cycle 1, 3 , 8, 15 , 22, and day 1 of every cycle and up to 12 cycles, corresponding to a treatment duration up to one year (each cycle is 28 days).Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Eric Stephen Winer, MD
Lead sponsor
Dana-Farber Cancer Institute
Sponsor institution
Source ClinicalTrials.gov