Actively Recruiting

Phase 1
Age: 18Years +
All Genders
ID03896269

Phase 1 Dose Escalation Study of CPX-351 for Patients With Intermediate-2 or High Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia After Failure to Hypomethylating Agents

Led by M.D. Anderson Cancer Center ยท Updated on 2026-05-20

38

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

M

M.D. Anderson Cancer Center

Lead Sponsor

N

National Cancer Institute (NCI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying the use of liposome-encapsulated daunorubicin-cytarabine (CPX-351) to find the best dose and understand side effects in treating patients with intermediate-2 or high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) that has returned or not responded to other treatments. This phase I trial focuses on safety, tolerability, maximum tolerated dose, and preliminary effectiveness of CPX-351, a chemotherapy drug that works by stopping cancer cells from growing or spreading. Patients receive CPX-351 intravenously over 90 minutes during an induction phase on days 1, 3, and 5. If the disease is stable and side effects are acceptable, patients may receive additional doses on days 1 and 3 after 2 to 5 weeks. Those who respond with at least hematological improvement enter a consolidation phase, receiving treatment every 28 days for up to 12 cycles, with possible extension for 12 more cycles if no significant disease progression or toxicity occurs. During the study, participants will be monitored for side effects, response to treatment, and disease progression. Follow-up visits occur 30 days after treatment and then every six months. Researchers will measure adverse events, response rates, survival outcomes, and relapse-free survival over a period of up to 1.5 years to evaluate the drug's safety and preliminary efficacy.

CONDITIONS

Brief Title

CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) according to World Health Organization criteria
  • Patients not eligible for or choosing not to proceed with stem cell transplant at enrollment
  • Classified as intermediate-2 or high-risk by International Prognostic Scoring System with excess blasts over 5% or 10-19% bone marrow blasts
  • No response after at least 4 cycles of azacitidine, decitabine, guadecitabine, or ASTX727 or relapse/progression after any number of cycles
  • Signed informed consent indicating understanding of the study and willingness to participate
  • Total bilirubin less than 3 mg/dL (up to 5 times upper limit if Gilbert's syndrome present)
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) less than or equal to 3 times upper limit of normal
  • Serum creatinine clearance greater than 30 mL/min and no end-stage renal disease
  • Use of hydroxyurea or hematopoietic growth factors allowed if in patient's best interest
Not Eligible

You will not qualify if you...

  • New York Heart Association class III or IV congestive heart failure or left ventricular ejection fraction less than 50%
  • History of myocardial infarction within 6 months, unstable angina, or severe uncontrolled arrhythmias
  • Uncontrolled infection not responding to antibiotics
  • Pregnant or lactating females
  • Patients with reproductive potential unwilling to follow contraception requirements
  • Positive pregnancy test at screening
  • Receiving other investigational agents, chemotherapy, radiotherapy, or immunotherapy within 14 days before study treatment
  • Prior cumulative anthracycline exposure exceeding 550 mg/m2 daunorubicin or equivalent, or over 400 mg/m2 with mediastinal radiation

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 1.5 years

Participants receive liposome-encapsulated daunorubicin-cytarabine intravenously as induction therapy followed by consolidation therapy if appropriate.

Treatment cycles with IV infusions on specific days repeated every 28 days for up to 24 cycles; initial induction therapy includes infusions on days 1, 3, and 5; subsequent cycles have infusions on days 1 and 3

Follow-up

Duration - Up to 1.5 years after treatment completion

Participants are followed up after completion of study treatment to monitor safety and outcomes.

Visits at 30 days post-treatment and then every 6 months

Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

G

Guillermo M Bravo

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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Published Research Related To This Trial

Phase 1/2 study of CPX-351 for patients with Int-2 or high risk International Prognostic Scoring System myelodysplastic syndromes and chronic myelomonocytic leukaemia after failure to hypomethylating agents.

Guillermo Montalban-Bravo, Elias Jabbour, Gautam Borthakur...

https://pubmed.ncbi.nlm.nih.gov/37946611