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Refractory Acute LeukemiaPhase 1NCT06466395

A Phase I Study of Hyper-CVAD In Combination With Venetoclax In Pediatric Patients With Relapsed or Refractory Acute Leukemias That Are of the Lymphoid Lineage Including Bi-Phenotypic or Undifferentiated Leukemias

A Phase 1 study testing Venetoclax and Hyper-CVAD in people with Refractory Acute Leukemia and Relapsed Acute Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

To find the recommended dose of hyper-CVAD in combination with venetoclax that can be given to participants with relapsed or refractory leukemia. Read more about this study

Who is it for?
Ages 2 to 21 years
What is being tested?
Venetoclax and Hyper-CVAD
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
1 siteUnited States
Study dates
18 Feb 2025to 31 Dec 2031
Phase
Phase 1
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 2 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Relapsed/refractory leukemias as defined as:
  • Pediatric, adolescent, or young adult patients with relapsed or refractory acute leukemias that are of the lymphoid lineage including bi-phenotypic or undifferentiated leukemias as per NCCN v2.2021 and W.H.O. classification in relapse or primary refractory.
  • Participants must have ≥5% blasts in the bone marrow as assessed by morphology. However, if an adequate bone marrow sample cannot be obtained, patients may be enrolled if there is unequivocal evidence of leukemia with ≥5% blasts in the peripheral blood.
  • Participants have adequate performance status (ECOG ≤2) for patients ≥16 years old, Lansky score >50 for patients <16 years old.
  • Participants must be ≥ 2 years old or less than or equal to 21 years of age at time of signing/or having proxy sign the informed consent to be enrolled on study.
  • Participants with asymptomatic CNS disease are eligible.
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  • These conditions are allowed on study: conditions requiring chronic systemic glucocorticoid use, such as autoimmune disease, graft versus host disease (GVHD) (well controlled on a stable dose of steroid or alternative therapy) or severe asthma. Participants are also allowed up to 5 days of glucocorticoids as cytoreduction, the use of hydroxyurea and the usage of cytarabine up to 2gm/m2. This should also be discussed with PI.
  • For participants on chronic glucocorticoid therapy: Participants should be on stable systemic steroid doses less than or equal to 11.6mg/m2 (20 mg max) of prednisone daily
  • During times of dexamethasone dosing (cycles 1,3,5 and 7): Hold chronic steroids on days dexamethasone is given then resume normally scheduled chronic steroid dosing the following day.
  • Participants on chronic steroids > 11.6mg/m2 (20 mg max) prednisone equivalent will be excluded from the study.
  • The use of topical steroids for cutaneous graft-versus-host disease (GVHD) is allowed.
  • Participants should be at least 2 weeks or 5 half-lives (whichever is longer) from prior therapy, other than hydroxyurea, glucocorticoids or low dose cytarabine (as mentioned above) to maintain blast count prior to initiation of study therapy.
  • Participants must have adequate organ function and laboratory results (obtained within 14 days of enrollment):
  • 6.1. Total serum bilirubin ≤1.5 x upper limit of normal (ULN). Participants with known Gilbert's syndrome may have a total bilirubin up to ≤3 x ULN.
  • 6.2. Adequate renal function (creatinine clearance >30mL/min) unless related to the disease.
  • 6.3. Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤3 x ULN; ≤5 x ULN unless in case of suspected leukemic liver involvement Females of childbearing potential must have a negative serum or urine beta-human chorionic gonadotropin (beta-hcg) pregnancy test result within 14 days prior to the first dose of study drugs and must agree to use one of the following effective contraception methods during the study and for 3 months following the last dose of study drug.
  • The effects of these investigational agents on the developing human fetus are unknown. For this reason and because chemotherapeutic and inhibiting agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:
  • Postmenopausal (no menses in greater than or equal to 12 consecutive months).
  • History of hysterectomy or bilateral salpingo-oophorectomy.
  • Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
  • History of bilateral tubal ligation or another surgical sterilization procedure.
  • Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
  • Males need to inform the doctor right away if the partner becomes pregnant or suspects pregnancy. While in this study and for 30 days after the last treatment the patient should not donate sperm for the purposes of reproduction. He will need to use a condom while in this study and for 30 days after the last treatment.
  • Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of all study drug administration. Participants must have had at least 30 days between prior hematopoietic stem cell transplant and first dose of study drug.
  • For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
  • Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
  • Ability to understand and the willingness to sign a written informed consent document.
  • Participants with CD19+ B-ALL need to have received CD19-directed therapy prior to being considered for enrollment on this study.

Who cannot join

  • Past or current history of a secondary or other primary tumor or a chronic myeloid leukemia (CML) blast crisis with exception of:
  • curatively treated non-melanomatous skin cancer,
  • other primary solid tumor treated with curative intent and no known active disease present, and no treatment administered during the last 2 years.
  • Presence of clinically significant uncontrolled CNS pathology such as epilepsy, paresis, aphasia, stroke, severe brain injuries, organic brain syndrome, or psychosis.
  • Presence of the following are allowed: headaches, vomiting, nerve palsy.
  • Significant traumatic injury or major surgery (major surgery means opening of a body cavity, e.g., thoracotomy, laparotomy, laparoscopic organ resection, and major orthopedic procedures, e.g. joint replacement, open reduction and internal fixation) within 14 days of scheduled dosing day 1.
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  • Participants with uncontrolled infections (viral, bacterial, or fungal) per PI's discretion. Infections controlled on concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxis per institutional guidelines are acceptable.
  • Medical history of cardiovascular disease such as:
  • Clinically significant cardiac disease defined as congestive heart failure (NYHA class III or IV), arrhythmia or conduction abnormality requiring medication, or cardiomyopathy. This will be reviewed during screening EKG/ECHO as well as prior documentation.
  • Females who are pregnant or lactating.
  • Participants may be excluded if they are currently enrolled in another ongoing clinical trial with investigational products.
  • Liver cirrhosis or other active severe liver disease or with suspected active alcohol abuse.
  • Participants who are unable or unwilling to comply with all study requirements for clinical visits, examinations, tests, and procedures.
  • If participant has not recovered from previous chemotherapy, surgery, radiation before the start of study drugs.
  • Other severe, uncontrolled acute or chronic medical or psychiatric condition or laboratory abnormality that in the opinion of the Investigator may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and/or would make the patient inappropriate for enrollment into this study.
  • Participants with active/uncontrolled HIV infection, AIDS, or currently taking contraindicated medications for HIV control
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to all agents used in study. Since each reaction can vary from mild pruritis to anaphylaxis, the reaction will be discussed with PI prior to excluding participant.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

David McCall, MD contact

(713) 792-6604

dmccall1@mdanderson.org

United States1 site, 1 recruiting

  • MD Anderson Cancer Center

    Houston, Texas, 77030

    David McCall, MD, 713-792-6604, dmccall1@mdanderson.org

    David McCall, MD

    Recruiting

Other trials in United States

About this study

To find the recommended dose of hyper-CVAD in combination with venetoclax that can be given to participants with relapsed or refractory leukemia.

Read the detailed description

Primary Objectives

  • To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D) of venetoclax in combination with hyper-CVAD in patients with relapsed or refractory acute leukemias that are of the lymphoid lineage including bi-phenotypic or undifferentiated leukemias.
  • To characterize the safety and tolerability of hyper-CVAD in combination with venetoclax.

Secondary Objectives

  • To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with incomplete blood count recovery (CRi) and partial response (PR).
  • Evaluate additional measures of clinical benefit including overall survival (OS), event-free survival (EFS), progression-free survival (PFS) minimal residual disease (MRD) rate, and duration of response (DOR).

Exploratory Objectives

. To evaluate the pharmacodynamics (PD) and biological effects of hyper-CVAD in combination with venetoclax through molecular and cellular markers that may be predictive of antitumor activity and/or resistance.

Being tested
Venetoclax, Hyper-CVAD

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VenetoclaxDrug

    Also called ABT-199, GDC-0199

    Given by PO

  • Hyper-CVADDrug

    Given by IV

Study groups in NCT06466395
Study groupRoleWhat they receive
Phase 1aExperimentalParticipants enrolled in Phase 1a, the dose of venetoclax the participant receive will depend on when the participant joins this study. The first group of participants will receive the lowest dose level of venetoclax. Each new group will receive a higher dose of venetoclax than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of venetoclax is found. The study doctor will discuss with you the doses the participants will receive as part of hyper-CVAD based on standard of care.
Receives: Drug: Venetoclax; Drug: Hyper-CVAD
Phase 1bExperimentalParticipants enrolled in Phase 1b, will receive venetoclax at the recommended dose that was found in Phase 1. The study doctor will discuss with you the doses the participants will receive as part of hyper-CVAD based on standard of care.
Receives: Drug: Venetoclax; Drug: Hyper-CVAD

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    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.

  1. First posted20 Jun 2024
  2. Study start18 Feb 2025
  3. Primary completion31 Dec 2027 (estimated)
  4. Study completion31 Dec 2031 (estimated)

Resources

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
Single group
Masking
None (open label)
Enrolment
22 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06466395
Sponsor's protocol ID
2023-1044
Other IDs
NCI-2024-05154
Status last verified
Sep 2026
First submitted
14 Jun 2024
Last updated
15 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Venetoclax and Hyper-CVAD in people with Refractory Acute Leukemia and Relapsed Acute Leukemia, 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 28 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 15 Sep 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?

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 David McCall, MD ((713) 792-6604, dmccall1@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?

On the ClinicalTrials.gov record for NCT06466395.

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 15 Sep 2026.

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