LeukemiaPhase 1NCT06021600
A Phase 1, Open-Label, Sequential Cross-over, Bioavailability/Bioequivalence Study to Compare the Pharmacokinetics of Oral Cladribine With the Reference Listed Drug, Intravenous Cladribine
A Phase 1 study testing Cladribine in people with Leukemia and Myelodysplastic Syndromes, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To compare an investigational oral form of the drug cladribine to the FDA approved form of the drug when it is given by vein (IV). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes, Acute Myeloid Leukemia
- What is being tested?
- 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
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Oct 2023to 24 Mar 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Study participants must meet the following inclusion criteria
- Patients, both men and women of all races and ethnic groups, aged ≥ 18 years are eligible for enrollment.
- Provision of written informed consent prior to any study related procedures.
- Patients with newly diagnosed or previously treated HCL who have indication for therapy and are candidates for cladribine therapy (all 3 cohorts).
- Patients with previously treated T-cell prolymphocytic leukemia who have received at least one course of alemtuzumab-based treatment, who have documented indication for therapy and who are candidates for cladribine therapy (Cohort 1 and 2) only.
- Adequate renal and hepatic organ function as indicated by the following laboratory values:
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- Creatinine clearance ≥ 60 mL/min
- Serum total bilirubin ≤1.5×ULN (with the exception of patients with known Gilbert's syndrome: serum total bilirubin must be <3×ULN in these patients)
- Aspartate aminotransferase (serum glutamic oxaloacetic transaminase) and alanine aminotransferase (serum glutamic pyruvic transaminase) ≤2.5×ULN or ≤5×ULN if due to leukemic involvement).
- Adequate cardiac function with a left ventricular ejection fraction ≥45%.
- Female patients are eligible to enter and participate in the study if they are of non-childbearing potential. Female patients of childbearing age must have a negative pregnancy test at screening and must not be breastfeeding and also agree to use at least 2 forms of effective birth control during the study treatment period and for at least 3 months after the last dose of investigational product. (See definitions in Appendix 2)
- Male patients 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 3 months after the last dose of investigational product. (See definitions in Appendix 2)
Who cannot join
- Study participants must not meet any of the following study exclusion criteria:
- Uncontrolled intercurrent illness including, but not limited to ongoing or active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, unstable cardiac arrhythmia, malabsorption, 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.
- Receipt of live or live-attenuated vaccines within 4-6 weeks preceding oral cladribine treatment, planned during cladribine treatment or after cladribine treatment until the participant's immune system is no longer weakened (e.g., white blood cell counts are within normal limits).
- Active hepatitis infection, positive hepatitis C antibody, hepatitis B virus surface antigen or hepatitis B virus core antibody at screening. Patients who are hepatitis C antibody positive will need to have a negative PCR result prior to enrollment. Those who are hepatitis C PCR positive will be excluded. Patients who are hepatitis B virus surface antigen positive or hepatitis B PCR positive will be excluded. Patients who are anti-HBc antibody positive and who are surface antigen negative will need to have a negative PCR result before enrollment.
- Human immunodeficiency virus (HIV) positive with a viral load >400 copies/mL and a CD4+ T-cell count of <350 cells/µL or with a history of an acquired immunodeficiency syndrome (AIDS) opportunistic infection within the past 12 months.
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- 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 cohort.
- Patients with baseline QTc >470 msec will not be enrolled in Cohort 1. If QTc changes are observed in response to cladribine in Cohort 1, then the protocol will be amended to exclude patients with baseline QTc >470 msec from Cohorts 2 and 3, as well
Trial locations and contacts
1 site in 1 country.
About this study
To compare an investigational oral form of the drug cladribine to the FDA approved form of the drug when it is given by vein (IV).
Read the detailed description
Objectives:
- To compare the pharmacokinetic (PK) properties of oral cladribine (investigational product) and intravenous (IV) cladribine (reference standard drug) when administered to patients with hairy-cell leukemia (HCL) or T-cell prolymphocytic leukemia (T-PLL).
Primary:
- To identify a dose of oral cladribine that is bioequivalent to IV cladribine when the IV dose is administered via a 2-hour infusion to patients with HCL or T-PLL. Area under the curve (AUC) and maximum concentration (Cmax) will be measured and relative exposure of oral and IV cladribine will be assessed based on the geometric mean ratio and associated 90% confidence interval for dose-normalized AUCs and Cmax for oral-to-IV cladribine, as estimated from an appropriate linear mixed effects model.
Secondary:
- To assess the plasma PK of oral cladribine following single-dose and multiple-dose administration.
- To identify a dose of oral cladribine that provides comparable exposure (AUC) to IV cladribine when the IV dose is administered via a 2-hour infusion to patients with HCL or T-PLL.
- To assess the safety of oral cladribine in patients with HCL or T PLL. No formal efficacy evaluations will be performed in this study. All patients will be observed for complete remission (CR), complete remission without minimal residual disease (MRD), partial response (PR), progressive disease (PD) or stable disease (SD) at the end of the study, although response is not a study endpoint.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Being tested
- Cladribine
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CladribineDrug
Also called Leustatin®, 2-CdA
Given by PO and Given by IV (vein)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Pilot) | Experimental | Participants will only receive 1 dose of oral cladribine, followed by 4 daily doses of cladribine by vein Receives: Drug: Cladribine |
| Cohort 2 (PK) | Experimental | Participants will only receive 1 dose of oral cladribine, followed by 4 daily doses of cladribine by vein Receives: Drug: Cladribine |
| Cohort 3 (All-Oral HCL) | Experimental | Participants will all receive the same dose of oral cladribine over the course of 5 days. Receives: Drug: Cladribine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 posted1 Sep 2023
- Study start9 Oct 2023
- Primary completion24 Mar 2026
- Study completion24 Mar 2026
Why it stopped: Slow participant enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Farhad Ravandi-Kashani, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06021600
- Sponsor's protocol ID
- 2023-0134
- Other IDs
- NCI-2023-06805
- Status last verified
- Apr 2026
- First submitted
- 28 Aug 2023
- Last updated
- 30 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Sequential Cross-over, Bioavailability/Bioequivalence Study to Compare the Pharmacokinetics of Oral Cladribine With the Reference Listed Drug, Intravenous Cladribine
- ClinicalTrials.gov ID
- NCT06021600
- Acronym / sponsor ID
- 2023-0134
- Conditions
- Leukemia, Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow participant enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 9 Oct 2023 (actual)
- Primary completion
- 24 Mar 2026 (actual)
- Study completion
- 24 Mar 2026 (actual)
- First posted
- 1 Sep 2023 (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.
To compare an investigational oral form of the drug cladribine to the FDA approved form of the drug when it is given by vein (IV).
Detailed description
Objectives:
- To compare the pharmacokinetic (PK) properties of oral cladribine (investigational product) and intravenous (IV) cladribine (reference standard drug) when administered to patients with hairy-cell leukemia (HCL) or T-cell prolymphocytic leukemia (T-PLL).
Primary:
- To identify a dose of oral cladribine that is bioequivalent to IV cladribine when the IV dose is administered via a 2-hour infusion to patients with HCL or T-PLL. Area under the curve (AUC) and maximum concentration (Cmax) will be measured and relative exposure of oral and IV cladribine will be assessed based on the geometric mean ratio and associated 90% confidence interval for dose-normalized AUCs and Cmax for oral-to-IV cladribine, as estimated from an appropriate linear mixed effects model.
Secondary:
- To assess the plasma PK of oral cladribine following single-dose and multiple-dose administration.
- To identify a dose of oral cladribine that provides comparable exposure (AUC) to IV cladribine when the IV dose is administered via a 2-hour infusion to patients with HCL or T-PLL.
- To assess the safety of oral cladribine in patients with HCL or T PLL. No formal efficacy evaluations will be performed in this study. All patients will be observed for complete remission (CR), complete remission without minimal residual disease (MRD), partial response (PR), progressive disease (PD) or stable disease (SD) at the end of the study, although response is not a study endpoint.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Pilot) | Experimental | Drug: CladribineParticipants will only receive 1 dose of oral cladribine, followed by 4 daily doses of cladribine by vein |
| Cohort 2 (PK) | Experimental | Drug: CladribineParticipants will only receive 1 dose of oral cladribine, followed by 4 daily doses of cladribine by vein |
| Cohort 3 (All-Oral HCL) | Experimental | Drug: CladribineParticipants will all receive the same dose of oral cladribine over the course of 5 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Study participants must meet the following inclusion criteria
- Patients, both men and women of all races and ethnic groups, aged ≥ 18 years are eligible for enrollment.
- Provision of written informed consent prior to any study related procedures.
- Patients with newly diagnosed or previously treated HCL who have indication for therapy and are candidates for cladribine therapy (all 3 cohorts).
- Patients with previously treated T-cell prolymphocytic leukemia who have received at least one course of alemtuzumab-based treatment, who have documented indication for therapy and who are candidates for cladribine therapy (Cohort 1 and 2) only.
Key exclusion criteria (10 listed)
- Uncontrolled intercurrent illness including, but not limited to ongoing or active uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, unstable cardiac arrhythmia, malabsorption, 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.
- Receipt of live or live-attenuated vaccines within 4-6 weeks preceding oral cladribine treatment, planned during cladribine treatment or after cladribine treatment until the participant's immune system is no longer weakened (e.g., white blood cell counts are within normal limits).
- Active hepatitis infection, positive hepatitis C antibody, hepatitis B virus surface antigen or hepatitis B virus core antibody at screening. Patients who are hepatitis C antibody positive will need to have a negative PCR result prior to enrollment. Those who are hepatitis C PCR positive will be excluded. Patients who are hepatitis B virus surface antigen positive or hepatitis B PCR positive will be excluded. Patients who are anti-HBc antibody positive and who are surface antigen negative will need to have a negative PCR result before enrollment.
- Human immunodeficiency virus (HIV) positive with a viral load >400 copies/mL and a CD4+ T-cell count of <350 cells/µL or with a history of an acquired immunodeficiency syndrome (AIDS) opportunistic infection within the past 12 months.
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 |
|---|---|---|
| CladribineAlso: Leustatin®, 2-CdAGiven by PO and Given by IV (vein) | Drug | Cohort 1 (Pilot), Cohort 2 (PK), Cohort 3 (All-Oral HCL) |
Primary outcome
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Time frame: through study completion; an average of 1 year
Results and safety
The study was stopped early (reason given: Slow participant enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2026.
- M D Anderson Cancer CenterLink 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. This study is not recruiting at the moment.
Farhad Ravandi-Kashani, MD 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 study testing Cladribine in people with Leukemia and Myelodysplastic Syndromes, 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 12 inclusion and 10 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: stopped early, as of the sponsor's last update on 30 Jul 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Slow participant enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0 (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 30 Jul 2026; AzTrials last refreshed its copy on 30 Oct 2026.