Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT05703542

BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome

Led by Eric Stephen Winer, MD · Updated on 2025-07-18

24

Participants Needed

1

Research Sites

182 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 and older
  • Diagnosis of relapsed AML with 5% or more myeloblasts in bone marrow or reappearance of blasts in blood
  • Diagnosis of refractory AML with 2 or fewer prior induction treatments
  • Diagnosis of WHO-defined myelodysplastic syndrome with excess blasts-2 (MDS-EB-2) with specified blast counts and refractory or relapsed after at least 4 cycles of hypomethylating agents
  • ECOG performance status 0 to 2 (Karnofsky score 60% or higher)
  • Adequate organ and marrow function including creatinine clearance of 30 mL/min or higher, total bilirubin less than or equal to 1.5 times upper limit of normal, ALT and AST less than or equal to 3 times upper limit of normal, and ejection fraction above 35%
  • White blood cell count less than 25,000/µL on day 1 of cycle 1; hydroxyurea allowed for cytoreduction
  • HIV-positive participants on effective therapy with undetectable viral load within 6 months
  • Participants with chronic hepatitis B with undetectable viral load on suppressive therapy
  • Participants with treated hepatitis C virus infection who are cured or on treatment with undetectable viral load
  • Participants with treated central nervous system disease with no progression on follow-up imaging
  • Male participants agree to use contraception and avoid sperm donation from first dose until 90 days post last dose
  • Females of childbearing potential must have negative pregnancy test within 7 days before treatment and agree to use contraception until 90 days after last dose
  • Ability to understand and sign informed consent
Not Eligible

You will not qualify if you...

  • Diagnosis of Acute Promyelocytic Leukemia
  • Active central nervous system involvement with AML
  • Recent chemotherapy, immunotherapy, radiotherapy, or investigational therapy within 2 weeks or 5 half-lives before first dose (except hydroxyurea)
  • Use of oral tyrosine kinase inhibitors within 2 weeks or 5 half-lives before first dose
  • Less than 100 days from allogeneic bone marrow transplant
  • Active graft-versus-host disease or use of calcineurin inhibitors within 28 days before treatment
  • Unresolved severe side effects from prior cancer treatments (except hair loss)
  • Use of other investigational agents
  • Use of strong CYP3A4 inducers/inhibitors without adjustment and use of gliptins
  • History of orthostatic hypotension with systolic blood pressure under 100 or uncontrolled hypertension
  • Cardiovascular disability of NYHA class 2 or higher
  • Active cancers within past 2 years except certain treated skin, cervical, breast, or low-grade prostate cancers
  • Active hepatitis B or C infection without controlled viral load
  • Uncontrolled illnesses including infections, heart failure, unstable angina, arrhythmias, or psychiatric/social conditions limiting study compliance
  • Pregnant or breastfeeding women

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Dana Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Actively Recruiting

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Research Team

E

Eric S Winer, MD

CONTACT

E

Eric S Winter, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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