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Cladribine + Cytarabine vs. HMA + Venetoclax for Acute Myeloid Leukemia

SA
JW
Overseen ByJudy Walsh
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial compares two treatment plans for individuals with acute myeloid leukemia (AML) or severe myelodysplastic syndrome (MDS), particularly those who may not tolerate intense treatments. One plan combines cladribine (a chemotherapy drug), cytarabine, and decitabine. The other uses azacitidine or decitabine with venetoclax, the current standard treatment. The trial aims to determine which treatment is more effective. It is suitable for those diagnosed with AML or high-grade MDS who experience symptoms such as frequent blood transfusions, significant weight loss, or night sweats. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor to get specific guidance based on your situation.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that the combination of cladribine, low-dose cytarabine, and decitabine is generally well-tolerated by patients. Studies have found this treatment safe and effective for certain blood cancers. For instance, one study reported a 68% positive response rate in patients unable to undergo more intense treatments.

Cladribine's FDA approval for other uses supports its safety. While some side effects occurred, they were manageable, and the treatment was deemed safe.

Participants should understand that this trial phase focuses on collecting more detailed information about safety and effectiveness. So far, the treatment has shown promising safety results.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments for Acute Myeloid Leukemia (AML) because they combine innovative approaches that differ from the standard care. Unlike most AML treatments that typically use hypomethylating agents like azacitidine or decitabine with venetoclax, the investigational treatment includes cladribine and cytarabine, which target cancer cells in a unique way. Cladribine and cytarabine disrupt DNA synthesis differently, which can potentially enhance the effectiveness against leukemia cells. This combination may offer new hope for patients by possibly improving treatment outcomes compared to existing options.

What evidence suggests that this trial's treatments could be effective for acute myeloid leukemia?

Research has shown that a combination of three drugs—cladribine, low-dose cytarabine, and decitabine—may be promising for treating acute myeloid leukemia (AML). Participants in the investigational arm of this trial will receive this combination. In earlier studies, this treatment led to a high response rate of 68% in patients unable to handle intensive treatment. Another study found that 93% of newly diagnosed AML patients showed no signs of cancer after treatment. These results suggest that this treatment could be effective for AML, particularly for patients who cannot undergo more aggressive therapies.12356

Who Is on the Research Team?

PC

Paul Crispen, MD

Principal Investigator

University of Florida

Are You a Good Fit for This Trial?

This trial is for patients aged 60 or older with acute myeloid leukemia (AML) or high-grade myelodysplastic syndrome (MDS), who are unfit for intensive induction. It's also open to younger patients if they prefer a non-intensive regimen. Participants must be treatment-naive, have an ECOG Performance status of 0-2, and adequate kidney function. Prior HMA therapy is okay unless it led to AML progression.

Inclusion Criteria

I am 60 years old or older.
I am using birth control and have a negative pregnancy test.
Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures.
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are randomized to receive either cladribine/low-dose cytarabine alternating with decitabine or hypomethylating agents plus venetoclax

18 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

  • Cladribine
  • Cytarabine
  • Decitabine
  • Venetoclax
Trial Overview The study compares a new treatment combo of Cladribine/LoDAC with Decitabine against the standard Hypomethylating Agents plus Venetoclax in elderly/unfit patients with AML/MDS. Patients will randomly receive either the novel regimen (Arm A) or the current standard care (Arm B).
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Active Control
Group I: Arm A (investigational arm): cladribine, cytarabine, and decitabineExperimental Treatment3 Interventions
Group II: Arm B (control arm): azacitidine with venetoclax or decitabine with venetoclaxActive Control2 Interventions

Cladribine is already approved in United States, European Union for the following indications:

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Approved in United States as Leustatin for:
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Approved in European Union as Litak for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials
1,428
Recruited
987,000+

Citations

1.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/30115541/
Cladribine and low-dose cytarabine alternating with ... - PubMedThe combination of cladribine and low-dose cytarabine alternating with decitabine appears to be a safe and highly effective regimen for the treatment of ...
NCT01515527 | Cladribine Plus Low Dose Cytarabine ...Cladribine is FDA approved and commercially available for use in patients with hairy cell leukemia. Its use in patients with AML is investigational. Cytarabine ...
Efficacy and safety of cladribine, low-dose cytarabine and ...A phase II study demonstrated a composite complete remission (CRc) rate of 93% with CLAD, LDAC and venetoclax in ND AML.
Cladribine and low-dose cytarabine alternating with ...The combination of cladribine and low-dose cytarabine alternating with decitabine appears to be a safe and highly effective regimen for the treatment of ...
Cladribine with Low Dose Cytarabine for Unfit Patients: A Safe ...Frontline cladribine (Clad) with low dose cytarabine (LDAC) alternating with decitabine yielded an overall response rate (ORR) of 68%, a median ...
Relapsed/Refractory Acute Myeloid Leukemia Pipeline ...Relapsed/Refractory Acute Myeloid Leukemia Pipeline Outlook Report 2025: Novel Antiviral Drugs Advance with Strong MOA Data | DelveInsight · APG- ...
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