A Phase 1b/2 Open-label, Dose-ranging Safety and Efficacy Study of Oral Cladribine in Patients With Acute Myeloid Leukemia (AML)
2 other identifiers
interventional
58
1 country
1
Brief Summary
The goal of Part 1 of this clinical research study is to find the highest tolerable dose of cladribine that can be given in combination with low dose cytarabine (LDAC) and venetoclax to patients who have AML. The goal of Part 2 of this clinical research study is to learn if the dose of cladribine found in Part 1, when combined with LDAC and venetoclax, can help to control the disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Dec 2027
Typical duration for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 30, 2025
CompletedFirst Posted
Study publicly available on registry
July 8, 2025
CompletedStudy Start
First participant enrolled
December 1, 2027
ExpectedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2028
Study Completion
Last participant's last visit for all outcomes
June 30, 2030
March 4, 2026
March 1, 2026
7 months
June 30, 2025
March 2, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Adverse Events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Through study completion; an average of 1 year
Study Arms (1)
Part 1/2 Treatment with Oral Cladribine in Patients with Acute Myeloid Leukemia
EXPERIMENTALParticipants may be in the hospital up to the first 5 days on study to all monitoring for tumor lysis syndrome (TLS) and toxicities.
Interventions
Given by intravenous adminstration
Given Orally
Eligibility Criteria
You may qualify if:
- Provision of written informed consent prior to any study related procedures.
- Disease characteristics, defined as:
- Part 1:
- Dose-escalation cohorts: Patients with relapsed and/or refractory AML.
- Part 2:
- Cohort A: Patients aged .18 years with newly diagnosed ts-AML, defined as patients with prior diagnosis of MDS, treated with hypomethylating agents who have then progressed to AML. Prior therapy with hydroxyurea, hematopoietic growth factors, HMA, ATRA, or a total dose of cytarabine up to 2g (for emergency use for stabilization) is allowed.
- Cohort B: Patients aged . 60 years with newly diagnosed AML with monocytic phenotype, defined as AML-M5 by FAB or flow cytometry, and/or patients . 60 years with newly diagnosed AML with RAS mutations. Patients aged \< 60 years who are unsuitable for standard induction therapy may be eligible after discussion with primary investigator.
- Adequate renal and hepatic organ function as indicated by the following laboratory values:
- Serum creatinine . 2.0xupper limit of normal (ULN)
- Serum total bilirubin .2xULN (with the exception of patients with known Gilbert's syndrome: serum total bilirubin must be \<3xULN in these patients)
- Aspartate aminotransferase (serum glutamic oxaloacetic transaminase) and alanine aminotransferase (serum glutamic pyruvic transaminase) .2.5xULN or .5xULN if due to leukemic involvement)
- Adequate cardiac function with a left ventricular ejection fraction .50%
- Female participants are eligible to enter and participate in the study if they are of nonchildbearing potential. Female participants of childbearing age must use at least 2 forms of effective birth control during the study treatment period and for at least 90 days after the last dose of investigational product.
- Male participants are eligible to enter and participate in the study if they agree to use effective methods of contraception during the study treatment period and for at least 90 days after the last dose of investigational product.
You may not qualify if:
- Uncontrolled intercurrent illness including, but not limited to ongoing or active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to any of the compounds in the study.
- Legal incapacity or limited legal capacity.
- Inability to swallow oral medications (capsules and tablets) without chewing, breaking, crushing, opening or otherwise altering the product formulation.
- Patients unwilling to comply with protocol requirements related to the assigned part.
- Patients with psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because the agents used in this study have the potential for teratogenic or abortifacient effects. Because there is a potential risk for adverse events in nursing infants secondary to treatment of the mother with chemotherapy agents, breastfeeding should also be avoided.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Gautam Borthakur, MBBS
M.D. Anderson Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 30, 2025
First Posted
July 8, 2025
Study Start (Estimated)
December 1, 2027
Primary Completion (Estimated)
June 30, 2028
Study Completion (Estimated)
June 30, 2030
Last Updated
March 4, 2026
Record last verified: 2026-03