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Acute Promyelocytic Leukemia (APL)NCT07187505

Venetoclax Combined With ATRA and ATO in Hyperleukocytic Acute Promyelocytic Leukemia

A study testing Venetoclax, All-trans retinoic acid and Arsenic Trioxide (ATO) in people with Acute Promyelocytic Leukemia (APL), run by Anhui Medical University at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study aims to evaluate the safety and effectiveness of combining venetoclax with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) in patients with newly diagnosed acute promyelocytic leukemia (APL) who have very high white blood cell counts. Read more about this study

Who is it for?
Ages 14 years and older
Conditions the study is for
Acute Promyelocytic Leukemia (APL)
What is being tested?
Venetoclax, All-trans retinoic acid and Arsenic Trioxide (ATO)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
28 participants
Where
1 siteChina
Study dates
1 Jul 2025to 1 Jul 2027
Phase
Interventional
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 14 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients diagnosed with acute promyelocytic leukemia (APL) according to bone marrow morphology and immunophenotyping, consistent with the WHO 2016 diagnostic criteria.
  • Age ≥14 years, both male and female patients are eligible.
  • Adequate organ function, defined as:
  • 3.1 Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3 × upper limit of normal (ULN);
  • 3.2 Total serum bilirubin ≤1.5 × ULN;
  • 3.3 Creatinine clearance ≥30 mL/min;
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  • 3.4 Serum cardiac enzymes <2.0 × ULN.
  • Signed informed consent obtained from the patient or a legally authorized representative.
  • White blood cell (WBC) count >10 × 10⁹/L at initial diagnosis, or WBC >10 × 10⁹/L during treatment.

Who cannot join

  • Diagnosis of acute non-promyelocytic leukemia, myeloid sarcoma, or chronic myeloid leukemia in accelerated or blast phase.
  • Known hypersensitivity to any drug included in the study regimen.
  • Pregnant or breastfeeding women, and women of childbearing potential who are unwilling to use effective contraception during the study treatment period.
  • Presence of organic heart disease, such as uncontrolled or symptomatic arrhythmia, congestive heart failure, or myocardial infarction within 6 months prior to screening that resulted in clinical symptoms or impaired cardiac function (NYHA class ≥III).
  • Concurrent malignancies, except for:
  • 5.1 Malignancies treated with curative intent (e.g., hematopoietic stem cell transplantation) and with no known active disease for ≥5 years before enrollment;
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  • 5.2 Adequately treated non-melanoma skin cancer or malignant lentigo without evidence of disease, even if diagnosed <3 years before enrollment;
  • 5.3 Adequately treated carcinoma in situ without evidence of disease, even if diagnosed <3 years before enrollment.
  • Patients with acquired immunodeficiency syndrome (AIDS) or syphilis, or those with active hepatitis B (detectable HBV DNA) or active hepatitis C infection.
  • Any concurrent medical condition or disease that may interfere with study procedures or outcomes, or that may pose an unacceptable risk to the participant as determined by the investigator (e.g., active systemic infection).
  • Inability to understand or comply with the study protocol.
  • Participation in another clinical study within 1 month prior to enrollment.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Department of Hematology, The First Affiliated Hospital of Anhui Medical University, 218 Jixi Road

    Hefei, Anhui, 230022

Other trials in China

About this study

Official title: Venetoclax Plus All-Trans Retinoic Acid and Arsenic Trioxide in Newly Diagnosed Acute Promyelocytic Leukemia With Hyperleukocytosis: A Prospective Single-Arm Study

This study aims to evaluate the safety and effectiveness of combining venetoclax with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) in patients with newly diagnosed acute promyelocytic leukemia (APL) who have very high white blood cell counts. APL is a rare type of blood cancer, and patients with high white blood cell levels often face serious complications. Current treatments with ATRA and ATO are effective, but the outcomes for patients with high white blood cells remain poor. This study will test whether adding venetoclax, a drug that helps leukemia cells die, can improve treatment results.

Read the detailed description

PRIMARY OBJECTIVE 1. To evaluate the efficacy of the venetoclax + ATRA + ATO regimen, as defined by complete remission (CR), complete remission with incomplete hematologic recovery (CRi), and morphological leukemia-free state (MLFS).

________________________________________ SECONDARY OBJECTIVES

  1. To evaluate long-term effectiveness and durability of the regimen, as defined by 1-year overall survival (OS), 1-year event-free survival (EFS), and overall response rate (ORR).
  2. To evaluate the safety of the regimen, as defined by Grade 3-4 clinical adverse events (AEs), incidence of laboratory abnormalities, differentiation syndrome, and treatment-related mortality (TRM).
  3. To assess transfusion requirements (red blood cells and platelets) during induction. ________________________________________ OUTLINE
  • Newly diagnosed hyperleukocytosis group: Patients with baseline WBC >10 × 10⁹/L will receive venetoclax + ATRA + ATO as induction therapy.
  • Secondary hyperleukocytosis group: Patients who develop WBC >10 × 10⁹/L for ≥3 consecutive days during therapy will receive venetoclax added dynamically to ATRA + ATO. Induction regimen:
  • Venetoclax (VEN): 100 mg orally once daily on days 1-7. For patients with WBC >100 × 10⁹/L, administer 50 mg on days 1-2, then escalate to 100 mg on days 3-7.
  • All-Trans Retinoic Acid (ATRA): 25 mg/m² orally per day (divided doses), on days 1-28.
  • Arsenic Trioxide (ATO): 0.15 mg/kg intravenously once daily, on days 1-28. Assessment:
  • Complete blood counts monitored regularly.
  • Bone marrow evaluation on days 14 and 28.
  • One treatment cycle lasts 28 days. Response evaluation:
  • On day 21 of the first cycle, bone marrow assessment will be performed.
  • If CR, CRi, or MLFS is not achieved, induction with the same regimen will continue for one additional cycle.
  • Patients failing to achieve remission after two cycles will be withdrawn from the study.
  • Patients achieving remission will proceed to consolidation and maintenance therapy according to investigator's discretion.
Being tested
Venetoclax, All-trans retinoic acid, Arsenic Trioxide (ATO)
Keywords
Venetoclax, All-Trans Retinoic Acid (ATRA), Arsenic Trioxide (ATO), Acute Promyelocytic Leukemia
Medical subject headings
Leukemia, Promyelocytic, Acute

What participants receive

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

  • VenetoclaxDrug

    Also called Venclexta

    Venetoclax is a selective BCL-2 inhibitor administered orally once daily. The dose will be adjusted according to the study protocol and patient tolerance.

  • All-trans retinoic acidDrug

    Also called Tretinoin, Vesanoid

    All-Trans Retinoic Acid (ATRA) is administered orally, twice daily, as part of standard induction and consolidation therapy for acute promyelocytic leukemia.

  • Arsenic Trioxide (ATO)Drug

    Also called Trisenox

    Arsenic Trioxide (ATO) is administered intravenously once daily, in combination with ATRA and venetoclax, during induction and consolidation therapy.

Study groups in NCT07187505
Study groupRoleWhat they receive
Experimental Arm: Venetoclax + ATRA + ATOExperimentalPatients in this arm will receive a combination regimen consisting of venetoclax, all-trans retinoic acid (ATRA), and arsenic trioxide (ATO) as induction therapy, followed by consolidation according to protocol. The regimen is designed for newly diagnosed acute promyelocytic leukemia (APL) with hyperleukocytosis.
Receives: Drug: Venetoclax; Drug: All-trans retinoic acid; Drug: Arsenic Trioxide (ATO)

What is being measured

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

  1. Early Mortality (Day 0-30)

    Proportion of patients who die from any cause within 30 days after treatment initiation.

    Measured: 30 days

Secondary outcomes (3)

  1. Incidence of Complications

    Proportion of patients experiencing severe complications related to induction therapy.

    Measured: 30 days

  2. Event-Free Survival (EFS)

    Time from enrollment to treatment failure, relapse, or death from any cause.

    Measured: Up to 2 years

  3. Overall Response Rate (ORR)

    Proportion of patients achieving overall response, defined as complete remission (CR) plus partial remission (PR).

    Measured: At 3 months after induction therapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Sep 2025
  2. Study start1 Jul 2025
  3. Primary completion1 Jul 2027 (estimated)
  4. Study completion1 Jul 2027 (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 N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
28 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07187505
Sponsor's protocol ID
PJ 2025-03-57(1)
Status last verified
Sep 2025
First submitted
6 Sep 2025
Last updated
23 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: De-identified individual participant data (IPD), including demographic information, baseline characteristics, efficacy outcomes, and safety data, will be shared. Data will be provided to qualified researchers for the purpose of academic research. Access will require submission of a research proposal and approval by the study steering committee, as well as signing a data use agreement.

Questions about this trial

What is this clinical trial studying?

A study testing Venetoclax, All-trans retinoic acid and Arsenic Trioxide (ATO) in people with Acute Promyelocytic Leukemia (APL), run by Anhui Medical University at 1 site in China.

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 9 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 23 Sep 2025. 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 China. 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.

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?

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?

Early Mortality (Day 0-30) (time frame: 30 days). 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 NCT07187505.

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 23 Sep 2025.

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