Testing CTX-712 for patients with relapsed or refractory acute myeloid leukemia and higher risk myelodysplastic syndromes

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a phase 1/2 multicenter, open-label, single-arm study designed to evaluate the safety, tolerability, and pharmacokinetic and pharmacodynamic profile of CTX-712 in patients with relapsed or refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (HR-MDS). The study will begin with a dose escalation phase to determine the recommended phase 2 dose (RP2D) of CTX-712, followed by an expansion cohort to further assess this dose. Once the RP2D is established, the phase 2 portion will evaluate the therapeutic activity of CTX-712 in the target patient population.

Why it matters

Who can join: the full rules

Inclusion criteria:

1. Age ≥18 years.
2. Diagnosis of AML, HR-MDS, or high marrow blast MDS/MPN (including CMML). Note: Only patients with AML and HR-MDS are eligible in the expansion cohorts.
3. Prior treatment history must include 1-4 prior lines of therapy. Note: 1-3 prior lines are allowed for patients in the expansion cohorts.
4. Adequate organ function evidenced by the following laboratory values:

   Creatinine clearance (CL) ≥60 mL/min
   * Total serum bilirubin \< 1.5 × upper limit of normal (ULN)
   * Alanine aminotransferase (ALT)
   * Aspartate aminotransferase(AST) \< 2.5 × ULN
   * White blood cell count at the time of the first dose \<10 k/μL
5. Eastern Cooperative Oncology Group performance status ≤2.
6. Female patients of childbearing potential must have a negative pregnancy test within 7 days before study treatment initiation and if sexually active, agree to use a highly effective form of contraception throughout their participation during study treatment and up to 4 months after the last dose of study drug.
7. Male patients with female partners of childbearing potential must, even if surgically sterilized, agree to practice effective barrier contraception during the entire study treatment period and through four months after the last dose of study drug, or practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant.

Exclusion criteria:

1. Diagnosis of acute promyelocytic leukemia.
2. Isolated extramedullary relapse (phase 2 only).
3. Active central nervous system (CNS) leukemia.
4. History of other malignancy.
5. Any of the following cardiopulmonary abnormalities:

   1. Myocardial infarction within six months prior to registration.
   2. New York Heart Association Class III or IV heart failure or known left ventricular ejection fraction \< 50%.
   3. A history of familial long QT syndrome.
   4. Symptomatic atrial or ventricular arrhythmias not controlled by medications.
   5. QTcF ≥ 470 msec calculated according to institutional guidelines, unless due to underlying bundle branch block and/or pacemaker and with approval of the medical monitor.
   6. Known moderate to severe and clinically significant chronic obstructive pulmonary disease, interstitial lung disease and/or pulmonary fibrosis (e.g., requiring home oxygen therapy).
6. Pregnancy and/or lactation.
7. Major surgery (excluding placement of vascular access) within 4 weeks prior to first dose of CTX-712.
8. History of allogeneic organ transplantation (excluding cornea).
9. History of allogenic hematopoietic stem cell transplantation within 6 months of planned study treatment initiation and/or graft-versus host disease grade ≥ 1 following allogenic hematopoietic stem cell transplantation.
10. History of or chimeric antigen receptor T-cell therapy or other modified T cell therapy.
11. Active, uncontrolled bacterial, fungal, or viral infection, including hepatitis B virus, hepatitis C virus, known human immunodeficiency virus, or acquired immunodeficiency syndrome related illness. Infections controlled with oral anti-infective agents, including prophylactic treatments, are allowed. Patient must be viral load negative.
12. Psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol and/or follow-up procedures outlined in the protocol.

Where it's running

• Mayo Clinic Arizona
  Phoenix, Arizona, United States Recruiting
• Mayo Clinic Florida
  Jacksonville, Florida, United States Recruiting
• Northwestern University
  Chicago, Illinois, United States Recruiting
• Mayo Clinic Comprehensive Cancer Center
  Rochester, Minnesota, United States Recruiting
• The University of Rochester
  Rochester, New York, United States Recruiting
• The University of Texas MD Anderson Cancer Center
  Houston, Texas, United States Recruiting

Study contacts

• Principal investigator: Guillermo Garcia-Manero, MD — M.D. Anderson Cancer Center
• Study coordinator: Laurie F Graham, RN, MSN
• Email: lgraham@theradex.com
• Phone: (609) 608-2152

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.