Acute Myeloid Leukemia (AML)Phase 1/2NCT07053020
A Phase 1 Open-Label, Dose-Ranging Safety and Pharmacokinetics Study of Oral Cladribine in Patients With Acute Myeloid Leukemia (AML)
A Phase 1/2 study testing Cytarabine and Oral Cladribine in people with Acute Myeloid Leukemia (AML), run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- Cytarabine and Oral Cladribine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2027to 30 Jun 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030
Gautam Borthakur, MBBS, 713-563-1586, gborthak@mdanderson.org
Gautam Borthakur, MBBS
About this study
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.
Read the detailed description
Primary Objective:
The primary objective of Phase 1 is to determine safety and tolerability of oral cladribine in patients with AML and to identify a RP2D.
Secondary Objective:
The secondary objectives of Phase 1 are to provide a qualitative assessment of systemic exposure based on plasma cladribine concentrations after administration of oral cladribine to patients with AML and to explore the activity of cladribine/LDAC/venetoclax in patients with relapsed or refractory AML.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- Cytarabine, Oral Cladribine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CytarabineDrug
Given by intravenous adminstration
Oral CladribineDrug
Given Orally
| Study group | Role | What they receive |
|---|---|---|
| Part 1/2 Treatment with Oral Cladribine in Patients with Acute Myeloid Leukemia | Experimental | Participants may be in the hospital up to the first 5 days on study to all monitoring for tumor lysis syndrome (TLS) and toxicities. Receives: Drug: Cytarabine; Drug: Oral Cladribine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2025
- Study start1 Dec 2027 (estimated)
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Jun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 58 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Gautam Borthakur, MBBS, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07053020
- Sponsor's protocol ID
- 2024-1896
- Other IDs
- NCI-2025-04733
- Status last verified
- Aug 2026
- First submitted
- 30 Jun 2025
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label, Dose-Ranging Safety and Pharmacokinetics Study of Oral Cladribine in Patients With Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT07053020
- Acronym / sponsor ID
- 2024-1896
- Conditions
- Acute Myeloid Leukemia (AML)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 58 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 1 Dec 2027 (estimated)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Jun 2030 (estimated)
- First posted
- 8 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Primary Objective:
The primary objective of Phase 1 is to determine safety and tolerability of oral cladribine in patients with AML and to identify a RP2D.
Secondary Objective:
The secondary objectives of Phase 1 are to provide a qualitative assessment of systemic exposure based on plasma cladribine concentrations after administration of oral cladribine to patients with AML and to explore the activity of cladribine/LDAC/venetoclax in patients with relapsed or refractory AML.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Part 1/2 Treatment with Oral Cladribine in Patients with Acute Myeloid Leukemia | Experimental | Drug: Cytarabine, Drug: Oral CladribineParticipants may be in the hospital up to the first 5 days on study to all monitoring for tumor lysis syndrome (TLS) and toxicities. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Provision of written informed consent prior to any study related procedures.
- Disease characteristics, defined as:
- Dose-escalation cohorts: Patients with relapsed and/or refractory AML.
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CytarabineGiven by intravenous adminstration | Drug | Part 1/2 Treatment with Oral Cladribine in Patients with Acute Myeloid Leukemia |
| Oral CladribineGiven Orally | Drug | Part 1/2 Treatment with Oral Cladribine in Patients with Acute Myeloid Leukemia |
Primary outcome
Safety and Adverse Events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Gautam Borthakur, MBBS principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cytarabine and Oral Cladribine in people with Acute Myeloid Leukemia (AML), run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gautam Borthakur, MBBS ((713) 563-1586, gborthak@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Adverse Events (AEs) (time frame: Through study completion; an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2026.